Intravitreal sirolimus proves effective in reducing noninfectious uveitis inflammation

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Intravitreal sirolimus 440 mcg or 880 mcg administered on days 1, 60, and 120, was shown to significantly improve ocular inflammation with preservation of best-corrected visual acuity in patients with noninfectious uveitis of the posterior segment, a phase III study has shown.

In the multinational SAKURA (Study Assessing Double-masked Uveitis Treatment) study, 346 study eyes were analyzed in this randomly assigned, double-masked, actively controlled study. In the study arm given intravitreal sirolimus 440 mcg, 22.8% (P = .025) met the primary endpoint of no vitreous haze (VH) at month 5 in the study eye without the aid of rescue therapy. In the group given intravitreal sirolimus 880 mcg, 16.4% (P = .182) met the primary endpoint, compared with 10.3% of active controls who were given 44 mcg intravitreal sirolimus.

For the secondary outcome, 52.6% (P = .008) of the intravitreal sirolimus 440 mcg arm had VH scores of 0 or a 0.5+ response rate at month 5. In the intravitreal sirolimus 880 mcg, 43.1% (P = .228) achieved a VH score of 0 or a 0.5+ response rate, compared with 35% of the 44 mcg active control group. Mean best-corrected visual acuity was maintained throughout the study in each study arm, with 76.9% of those who received corticosteroids at baseline in the 440 mcg study arm successfully tapering them to 5 mg per day or less by month 5, and 66.7% of those receiving corticosteroids in the 880 mcg group doing so. This was in comparison with 63.6% of those using corticosteroids in the active control group. Adverse events were similar across the study, and all doses were well tolerated. The study was funded by Santen.

Read the full study in Ophthalmology (2016;23[11]:2413-23).
 

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Intravitreal sirolimus 440 mcg or 880 mcg administered on days 1, 60, and 120, was shown to significantly improve ocular inflammation with preservation of best-corrected visual acuity in patients with noninfectious uveitis of the posterior segment, a phase III study has shown.

In the multinational SAKURA (Study Assessing Double-masked Uveitis Treatment) study, 346 study eyes were analyzed in this randomly assigned, double-masked, actively controlled study. In the study arm given intravitreal sirolimus 440 mcg, 22.8% (P = .025) met the primary endpoint of no vitreous haze (VH) at month 5 in the study eye without the aid of rescue therapy. In the group given intravitreal sirolimus 880 mcg, 16.4% (P = .182) met the primary endpoint, compared with 10.3% of active controls who were given 44 mcg intravitreal sirolimus.

For the secondary outcome, 52.6% (P = .008) of the intravitreal sirolimus 440 mcg arm had VH scores of 0 or a 0.5+ response rate at month 5. In the intravitreal sirolimus 880 mcg, 43.1% (P = .228) achieved a VH score of 0 or a 0.5+ response rate, compared with 35% of the 44 mcg active control group. Mean best-corrected visual acuity was maintained throughout the study in each study arm, with 76.9% of those who received corticosteroids at baseline in the 440 mcg study arm successfully tapering them to 5 mg per day or less by month 5, and 66.7% of those receiving corticosteroids in the 880 mcg group doing so. This was in comparison with 63.6% of those using corticosteroids in the active control group. Adverse events were similar across the study, and all doses were well tolerated. The study was funded by Santen.

Read the full study in Ophthalmology (2016;23[11]:2413-23).
 

 

Intravitreal sirolimus 440 mcg or 880 mcg administered on days 1, 60, and 120, was shown to significantly improve ocular inflammation with preservation of best-corrected visual acuity in patients with noninfectious uveitis of the posterior segment, a phase III study has shown.

In the multinational SAKURA (Study Assessing Double-masked Uveitis Treatment) study, 346 study eyes were analyzed in this randomly assigned, double-masked, actively controlled study. In the study arm given intravitreal sirolimus 440 mcg, 22.8% (P = .025) met the primary endpoint of no vitreous haze (VH) at month 5 in the study eye without the aid of rescue therapy. In the group given intravitreal sirolimus 880 mcg, 16.4% (P = .182) met the primary endpoint, compared with 10.3% of active controls who were given 44 mcg intravitreal sirolimus.

For the secondary outcome, 52.6% (P = .008) of the intravitreal sirolimus 440 mcg arm had VH scores of 0 or a 0.5+ response rate at month 5. In the intravitreal sirolimus 880 mcg, 43.1% (P = .228) achieved a VH score of 0 or a 0.5+ response rate, compared with 35% of the 44 mcg active control group. Mean best-corrected visual acuity was maintained throughout the study in each study arm, with 76.9% of those who received corticosteroids at baseline in the 440 mcg study arm successfully tapering them to 5 mg per day or less by month 5, and 66.7% of those receiving corticosteroids in the 880 mcg group doing so. This was in comparison with 63.6% of those using corticosteroids in the active control group. Adverse events were similar across the study, and all doses were well tolerated. The study was funded by Santen.

Read the full study in Ophthalmology (2016;23[11]:2413-23).
 

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Amyloid PET scans may have clinical utility in patients with cognitive impairment

Many pitfalls and unaddressed uses of amyloid PET
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Amyloid PET scans can affect diagnostic thinking and patient management when used alongside routine diagnostic work up in adults suspected of having Alzheimer’s disease, results from the multicenter, open-label Assessment of the Incremental Diagnostic Value of Florbetapir 18F Imaging in Patients with Cognitive Impairment (INDIA-FBP) study reveal.

The investigators, led by Marina Boccardi, PhD, of the LANVIE-Laboratory of Neuroimaging of Aging at the University of Geneva, conducted the study to provide evidence of whether amyloid PET imaging has diagnostic utility in patients suspected of having Alzheimer’s disease because amyloid PET scanning in its current state has debatable clinical value because of a lack of disease-modifying drugs available to treat Alzheimer’s and the expense of the scans.

The trial was published in this week’s edition of JAMA Neurology and Dr. Boccardi and her colleagues found that amyloid PET scans in 228 cognitively impaired Italian adults improved diagnostic confidence and led to a change in diagnosis in 28% of patients whose prescan diagnosis was Alzheimer’s disease and in 25% (P = .02) of patients who had a non–Alzheimer’s disease–related dementia diagnosis. A positive scan resulted in a change in diagnosis to Alzheimer’s disease 72% of the time in patients with a prescan diagnosis of frontotemporal lobar degeneration (FTLD). Cognition-specific medications were initiated in 66% of previously untreated patients and were withdrawn in 33% of previously treated patients, results from the study showed (JAMA Neurol. 2016 Oct 31. doi: 10.1001/jamaneurol.2016.3751).

The scans’ ability to provide additional information to change a diagnosis justifies their use, especially when considering “reports of adverse events in patients with FTLD treated with cholinesterase inhibitors and of ineffectiveness for cognitive impairment of vascular etiology,” the investigators wrote.

The use of amyloid PET scans to change a diagnosis to Alzheimer’s disease or confirm its diagnosis also allowed for earlier detection and intervention, gave patients an opportunity to be included in clinical trials, and allowed patients to make residential and financial arrangements at a time when they were still able to express their preference.

Anticipated results from the Imaging Dementia—Evidence for Amyloid Scanning (IDEAS) and Amyloid Imaging to Prevent Alzheimer’s Disease (AMYPAD) studies will expand on the current observations on a larger scale as well as help to quantify the cost-effectiveness of amyloid PET in clinical routine practice, they said.

In the current study, a prescan diagnosis was made by clinicians who, at the time of the work-up, estimated diagnostic confidence and provided drug treatment. At the same time, an amyloid PET/CT scan was performed and the result was communicated to the clinicians upon work-up completion. Clinicians relied on the scan results for their final clinical diagnoses and identification of the most-appropriate treatment.

Amyloid PET scans are currently not reimbursed by the Centers for Medicare & Medicaid Services outside of their use in clinical trials.

This study was sponsored by Istituto di Ricovero e Cura a Carattere Scientifico Centro San Giovanni di Dio Fatebenefratelli in Brescia, Italy, and Avid Radiopharmaceuticals. Several of the authors reported serving on the scientific advisory boards of pharmaceutical companies.

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While amyloid PET scans have a clear role in research and in the right clinical setting they can support a suspected diagnosis of Alzheimer’s disease or help a clinician to choose between Alzheimer’s and a non-Alzheimer’s condition, their use can have pitfalls even at a diagnostically simplified level.

Dr. Richard J. Caselli
We know that amyloid positivity increases with age even in the cognitively unimpaired population, and it does a poor job of marking patients with actual cognitive deficits, unlike tau PET. And even if amyloid scan results are positive, patients may still harbor significant comorbid abnormalities that contribute to clinical severity. Amyloid PET scans also do not assess cholinergic integrity, and some patients with non-Alzheimer’s dementia also have cholinergic deficiencies that may derive the same benefit from treatment.

There are at least four patient populations in whom amyloid PET scans could potentially have clinical utility, but these are not addressed in the current debate over their use or in this study: patients with early-onset dementia, patients with normal pressure hydrocephalus, patients with rapidly progressive dementia in which the differential diagnosis includes some reversible conditions that may require a diagnostic brain biopsy, and rare patients with cerebral amyloid angiopathy who develop tumorlike accumulations that necessitate a brain biopsy for diagnosis.

Dr. Bryan K. Woodruff
Regardless of the current debate, the use of molecular imaging to identify the therapeutic target through either amyloid PET for amyloid-targeted therapies or tau PET for tau-targeted therapies will be unquestioned when disease-modifying therapy arrives.

Richard J. Caselli, MD, and Bryan K. Woodruff, MD, are with the department of neurology at the Mayo Clinic Arizona, Scottsdale. Their comments are derived from an editorial accompanying Dr. Boccardi and colleagues’ report (JAMA Neurol. 2016 Oct 31. doi: 10.1001/jamaneurol.2016.3792). Dr. Caselli reported receiving research funding from Merck, and Dr. Woodruff reported receiving research funding from Genentech and Avid Pharmaceuticals.

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While amyloid PET scans have a clear role in research and in the right clinical setting they can support a suspected diagnosis of Alzheimer’s disease or help a clinician to choose between Alzheimer’s and a non-Alzheimer’s condition, their use can have pitfalls even at a diagnostically simplified level.

Dr. Richard J. Caselli
We know that amyloid positivity increases with age even in the cognitively unimpaired population, and it does a poor job of marking patients with actual cognitive deficits, unlike tau PET. And even if amyloid scan results are positive, patients may still harbor significant comorbid abnormalities that contribute to clinical severity. Amyloid PET scans also do not assess cholinergic integrity, and some patients with non-Alzheimer’s dementia also have cholinergic deficiencies that may derive the same benefit from treatment.

There are at least four patient populations in whom amyloid PET scans could potentially have clinical utility, but these are not addressed in the current debate over their use or in this study: patients with early-onset dementia, patients with normal pressure hydrocephalus, patients with rapidly progressive dementia in which the differential diagnosis includes some reversible conditions that may require a diagnostic brain biopsy, and rare patients with cerebral amyloid angiopathy who develop tumorlike accumulations that necessitate a brain biopsy for diagnosis.

Dr. Bryan K. Woodruff
Regardless of the current debate, the use of molecular imaging to identify the therapeutic target through either amyloid PET for amyloid-targeted therapies or tau PET for tau-targeted therapies will be unquestioned when disease-modifying therapy arrives.

Richard J. Caselli, MD, and Bryan K. Woodruff, MD, are with the department of neurology at the Mayo Clinic Arizona, Scottsdale. Their comments are derived from an editorial accompanying Dr. Boccardi and colleagues’ report (JAMA Neurol. 2016 Oct 31. doi: 10.1001/jamaneurol.2016.3792). Dr. Caselli reported receiving research funding from Merck, and Dr. Woodruff reported receiving research funding from Genentech and Avid Pharmaceuticals.

Body

 

While amyloid PET scans have a clear role in research and in the right clinical setting they can support a suspected diagnosis of Alzheimer’s disease or help a clinician to choose between Alzheimer’s and a non-Alzheimer’s condition, their use can have pitfalls even at a diagnostically simplified level.

Dr. Richard J. Caselli
We know that amyloid positivity increases with age even in the cognitively unimpaired population, and it does a poor job of marking patients with actual cognitive deficits, unlike tau PET. And even if amyloid scan results are positive, patients may still harbor significant comorbid abnormalities that contribute to clinical severity. Amyloid PET scans also do not assess cholinergic integrity, and some patients with non-Alzheimer’s dementia also have cholinergic deficiencies that may derive the same benefit from treatment.

There are at least four patient populations in whom amyloid PET scans could potentially have clinical utility, but these are not addressed in the current debate over their use or in this study: patients with early-onset dementia, patients with normal pressure hydrocephalus, patients with rapidly progressive dementia in which the differential diagnosis includes some reversible conditions that may require a diagnostic brain biopsy, and rare patients with cerebral amyloid angiopathy who develop tumorlike accumulations that necessitate a brain biopsy for diagnosis.

Dr. Bryan K. Woodruff
Regardless of the current debate, the use of molecular imaging to identify the therapeutic target through either amyloid PET for amyloid-targeted therapies or tau PET for tau-targeted therapies will be unquestioned when disease-modifying therapy arrives.

Richard J. Caselli, MD, and Bryan K. Woodruff, MD, are with the department of neurology at the Mayo Clinic Arizona, Scottsdale. Their comments are derived from an editorial accompanying Dr. Boccardi and colleagues’ report (JAMA Neurol. 2016 Oct 31. doi: 10.1001/jamaneurol.2016.3792). Dr. Caselli reported receiving research funding from Merck, and Dr. Woodruff reported receiving research funding from Genentech and Avid Pharmaceuticals.

Title
Many pitfalls and unaddressed uses of amyloid PET
Many pitfalls and unaddressed uses of amyloid PET

 

Amyloid PET scans can affect diagnostic thinking and patient management when used alongside routine diagnostic work up in adults suspected of having Alzheimer’s disease, results from the multicenter, open-label Assessment of the Incremental Diagnostic Value of Florbetapir 18F Imaging in Patients with Cognitive Impairment (INDIA-FBP) study reveal.

The investigators, led by Marina Boccardi, PhD, of the LANVIE-Laboratory of Neuroimaging of Aging at the University of Geneva, conducted the study to provide evidence of whether amyloid PET imaging has diagnostic utility in patients suspected of having Alzheimer’s disease because amyloid PET scanning in its current state has debatable clinical value because of a lack of disease-modifying drugs available to treat Alzheimer’s and the expense of the scans.

The trial was published in this week’s edition of JAMA Neurology and Dr. Boccardi and her colleagues found that amyloid PET scans in 228 cognitively impaired Italian adults improved diagnostic confidence and led to a change in diagnosis in 28% of patients whose prescan diagnosis was Alzheimer’s disease and in 25% (P = .02) of patients who had a non–Alzheimer’s disease–related dementia diagnosis. A positive scan resulted in a change in diagnosis to Alzheimer’s disease 72% of the time in patients with a prescan diagnosis of frontotemporal lobar degeneration (FTLD). Cognition-specific medications were initiated in 66% of previously untreated patients and were withdrawn in 33% of previously treated patients, results from the study showed (JAMA Neurol. 2016 Oct 31. doi: 10.1001/jamaneurol.2016.3751).

The scans’ ability to provide additional information to change a diagnosis justifies their use, especially when considering “reports of adverse events in patients with FTLD treated with cholinesterase inhibitors and of ineffectiveness for cognitive impairment of vascular etiology,” the investigators wrote.

The use of amyloid PET scans to change a diagnosis to Alzheimer’s disease or confirm its diagnosis also allowed for earlier detection and intervention, gave patients an opportunity to be included in clinical trials, and allowed patients to make residential and financial arrangements at a time when they were still able to express their preference.

Anticipated results from the Imaging Dementia—Evidence for Amyloid Scanning (IDEAS) and Amyloid Imaging to Prevent Alzheimer’s Disease (AMYPAD) studies will expand on the current observations on a larger scale as well as help to quantify the cost-effectiveness of amyloid PET in clinical routine practice, they said.

In the current study, a prescan diagnosis was made by clinicians who, at the time of the work-up, estimated diagnostic confidence and provided drug treatment. At the same time, an amyloid PET/CT scan was performed and the result was communicated to the clinicians upon work-up completion. Clinicians relied on the scan results for their final clinical diagnoses and identification of the most-appropriate treatment.

Amyloid PET scans are currently not reimbursed by the Centers for Medicare & Medicaid Services outside of their use in clinical trials.

This study was sponsored by Istituto di Ricovero e Cura a Carattere Scientifico Centro San Giovanni di Dio Fatebenefratelli in Brescia, Italy, and Avid Radiopharmaceuticals. Several of the authors reported serving on the scientific advisory boards of pharmaceutical companies.

 

Amyloid PET scans can affect diagnostic thinking and patient management when used alongside routine diagnostic work up in adults suspected of having Alzheimer’s disease, results from the multicenter, open-label Assessment of the Incremental Diagnostic Value of Florbetapir 18F Imaging in Patients with Cognitive Impairment (INDIA-FBP) study reveal.

The investigators, led by Marina Boccardi, PhD, of the LANVIE-Laboratory of Neuroimaging of Aging at the University of Geneva, conducted the study to provide evidence of whether amyloid PET imaging has diagnostic utility in patients suspected of having Alzheimer’s disease because amyloid PET scanning in its current state has debatable clinical value because of a lack of disease-modifying drugs available to treat Alzheimer’s and the expense of the scans.

The trial was published in this week’s edition of JAMA Neurology and Dr. Boccardi and her colleagues found that amyloid PET scans in 228 cognitively impaired Italian adults improved diagnostic confidence and led to a change in diagnosis in 28% of patients whose prescan diagnosis was Alzheimer’s disease and in 25% (P = .02) of patients who had a non–Alzheimer’s disease–related dementia diagnosis. A positive scan resulted in a change in diagnosis to Alzheimer’s disease 72% of the time in patients with a prescan diagnosis of frontotemporal lobar degeneration (FTLD). Cognition-specific medications were initiated in 66% of previously untreated patients and were withdrawn in 33% of previously treated patients, results from the study showed (JAMA Neurol. 2016 Oct 31. doi: 10.1001/jamaneurol.2016.3751).

The scans’ ability to provide additional information to change a diagnosis justifies their use, especially when considering “reports of adverse events in patients with FTLD treated with cholinesterase inhibitors and of ineffectiveness for cognitive impairment of vascular etiology,” the investigators wrote.

The use of amyloid PET scans to change a diagnosis to Alzheimer’s disease or confirm its diagnosis also allowed for earlier detection and intervention, gave patients an opportunity to be included in clinical trials, and allowed patients to make residential and financial arrangements at a time when they were still able to express their preference.

Anticipated results from the Imaging Dementia—Evidence for Amyloid Scanning (IDEAS) and Amyloid Imaging to Prevent Alzheimer’s Disease (AMYPAD) studies will expand on the current observations on a larger scale as well as help to quantify the cost-effectiveness of amyloid PET in clinical routine practice, they said.

In the current study, a prescan diagnosis was made by clinicians who, at the time of the work-up, estimated diagnostic confidence and provided drug treatment. At the same time, an amyloid PET/CT scan was performed and the result was communicated to the clinicians upon work-up completion. Clinicians relied on the scan results for their final clinical diagnoses and identification of the most-appropriate treatment.

Amyloid PET scans are currently not reimbursed by the Centers for Medicare & Medicaid Services outside of their use in clinical trials.

This study was sponsored by Istituto di Ricovero e Cura a Carattere Scientifico Centro San Giovanni di Dio Fatebenefratelli in Brescia, Italy, and Avid Radiopharmaceuticals. Several of the authors reported serving on the scientific advisory boards of pharmaceutical companies.

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Key clinical point: Amyloid PET improved diagnostic confidence but also led to changes in diagnosis and therapeutic plan if results were inconsistent with a previous diagnosis.

Main finding: Amyloid PET scans led to a change in diagnosis in 28% of patients whose prescan diagnosis was Alzheimer’s disease (AD) and in 25% of patients who had a non–AD related dementia diagnosis.

Source: Multicenter, open-label study of the incremental diagnostic value of Florbetapir 18F imaging in 228 adult patients with cognitive impairment.

Disclosures: This study was sponsored by Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) Centro San Giovanni di Dio Fatebenefratelli in Brescia, Italy, and Avid Radiopharmaceuticals. Several of the authors reported serving on the scientific advisory boards of pharmaceutical companies.

Drug enhances carboplatin efficacy against ovarian cancer

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The WEE1 tyrosine kinase inhibitor AZD1775 enhanced carboplatin’s antitumor activity in a phase II proof-of-concept trial of refractory or resistant TP53-mediated ovarian cancer, according to a report in the Journal of Clinical Oncology.

The manufacturer-sponsored open-label nonrandomized trial involved 23 women (median age, 58 years; range, 25-74 years) who had epithelial ovarian cancer and TP53 mutations and whose disease didn’t respond to or recurred after first-line platinum plus paclitaxel therapy. All the participants also underwent either primary or interval debulking surgery, said Suzanne Leijen, MD, PhD, of The Netherlands Cancer Institute, Amsterdam, and her associates.

The patients took 225 mg AZD1775 orally twice a day for 3 days along with IV carboplatin in 21-day cycles, until the cancer progressed. The primary outcome measure, overall response rate, was 43% in the 21 evaluable patients. After two treatment cycles, one patient had a complete response, eight had a partial response, seven had stable disease, and five had progressive disease.

“This response rate exceeds the effect that could be expected with second-line single-agent treatment options, including paclitaxel, pegylated liposomal doxorubicin, bevacizumab, and topotecan, which have reported response rates of 11%-21%,” the investigators said (J Clin Oncol. 2016 Oct. 31. doi: 10.1200/JCO.2016.67.5942).

The median progression-free survival was 5.3 months and median overall survival was 12.6 months. One patient showed a partial response for 31 months and another showed a complete response for 42 months, and both continue to receive the treatment.

AZD1775 plus carboplatin “was generally well tolerated and demonstrated manageable toxicity,” including nausea, vomiting, diarrhea, fatigue, bone marrow suppression, thrombocytopenia, and neutropenia.

This study was supported by Merck, which was involved in the data analysis and interpretation. Dr. Leijen reported having no relevant financial disclosures; her associates reported ties to Merck, AstraZeneca, Roche, Novartis, Advaxis, and Modra Pharmaceuticals.

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The WEE1 tyrosine kinase inhibitor AZD1775 enhanced carboplatin’s antitumor activity in a phase II proof-of-concept trial of refractory or resistant TP53-mediated ovarian cancer, according to a report in the Journal of Clinical Oncology.

The manufacturer-sponsored open-label nonrandomized trial involved 23 women (median age, 58 years; range, 25-74 years) who had epithelial ovarian cancer and TP53 mutations and whose disease didn’t respond to or recurred after first-line platinum plus paclitaxel therapy. All the participants also underwent either primary or interval debulking surgery, said Suzanne Leijen, MD, PhD, of The Netherlands Cancer Institute, Amsterdam, and her associates.

The patients took 225 mg AZD1775 orally twice a day for 3 days along with IV carboplatin in 21-day cycles, until the cancer progressed. The primary outcome measure, overall response rate, was 43% in the 21 evaluable patients. After two treatment cycles, one patient had a complete response, eight had a partial response, seven had stable disease, and five had progressive disease.

“This response rate exceeds the effect that could be expected with second-line single-agent treatment options, including paclitaxel, pegylated liposomal doxorubicin, bevacizumab, and topotecan, which have reported response rates of 11%-21%,” the investigators said (J Clin Oncol. 2016 Oct. 31. doi: 10.1200/JCO.2016.67.5942).

The median progression-free survival was 5.3 months and median overall survival was 12.6 months. One patient showed a partial response for 31 months and another showed a complete response for 42 months, and both continue to receive the treatment.

AZD1775 plus carboplatin “was generally well tolerated and demonstrated manageable toxicity,” including nausea, vomiting, diarrhea, fatigue, bone marrow suppression, thrombocytopenia, and neutropenia.

This study was supported by Merck, which was involved in the data analysis and interpretation. Dr. Leijen reported having no relevant financial disclosures; her associates reported ties to Merck, AstraZeneca, Roche, Novartis, Advaxis, and Modra Pharmaceuticals.

 

The WEE1 tyrosine kinase inhibitor AZD1775 enhanced carboplatin’s antitumor activity in a phase II proof-of-concept trial of refractory or resistant TP53-mediated ovarian cancer, according to a report in the Journal of Clinical Oncology.

The manufacturer-sponsored open-label nonrandomized trial involved 23 women (median age, 58 years; range, 25-74 years) who had epithelial ovarian cancer and TP53 mutations and whose disease didn’t respond to or recurred after first-line platinum plus paclitaxel therapy. All the participants also underwent either primary or interval debulking surgery, said Suzanne Leijen, MD, PhD, of The Netherlands Cancer Institute, Amsterdam, and her associates.

The patients took 225 mg AZD1775 orally twice a day for 3 days along with IV carboplatin in 21-day cycles, until the cancer progressed. The primary outcome measure, overall response rate, was 43% in the 21 evaluable patients. After two treatment cycles, one patient had a complete response, eight had a partial response, seven had stable disease, and five had progressive disease.

“This response rate exceeds the effect that could be expected with second-line single-agent treatment options, including paclitaxel, pegylated liposomal doxorubicin, bevacizumab, and topotecan, which have reported response rates of 11%-21%,” the investigators said (J Clin Oncol. 2016 Oct. 31. doi: 10.1200/JCO.2016.67.5942).

The median progression-free survival was 5.3 months and median overall survival was 12.6 months. One patient showed a partial response for 31 months and another showed a complete response for 42 months, and both continue to receive the treatment.

AZD1775 plus carboplatin “was generally well tolerated and demonstrated manageable toxicity,” including nausea, vomiting, diarrhea, fatigue, bone marrow suppression, thrombocytopenia, and neutropenia.

This study was supported by Merck, which was involved in the data analysis and interpretation. Dr. Leijen reported having no relevant financial disclosures; her associates reported ties to Merck, AstraZeneca, Roche, Novartis, Advaxis, and Modra Pharmaceuticals.

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FROM THE JOURNAL OF CLINICAL ONCOLOGY

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Key clinical point: The WEE1 tyrosine kinase inhibitor AZD1775 enhanced carboplatin’s antitumor activity in refractory or resistant TP53-mutated ovarian cancer.

Major finding: The overall response rate was 43% in 21 evaluable patients: One patient had a complete response, eight had a partial response, seven had stable disease, and five had progressive disease.

Data source: A proof-of-concept open-label nonrandomized phase II study involving 23 patients.

Disclosures: This study was supported by Merck, which was involved in the data analysis and interpretation. Dr. Leijen reported having no relevant financial disclosures; her associates reported ties to Merck, AstraZeneca, Roche, Novartis, Advaxis, and Modra Pharmaceuticals.

Causes of recurrent pediatric pancreatitis start to emerge

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– Once children have a first bout of acute pancreatitis, a second, separate episode of acute pancreatitis most often occurs in patients with genetically triggered pancreatitis, those who are taller or weigh more than average, and patients with pancreatic necrosis, based on multicenter, prospective data collected from 83 patients.

This is the first reported study to prospectively follow pediatric cases of acute pancreatitis, and additional studies with more patients are needed to better identify the factors predisposing patients to recurrent episodes of acute pancreatitis and to quantify the amount of risk these factors pose, Katherine F. Sweeny, MD, said at the annual meeting of the Federation of the International Societies of Pediatric Gastroenterology, Hepatology, and Nutrition.

 



Mitchel L. Zoler/Frontline Medical News
Dr. Katherine F. Sweeny
Dr. Sweeny and her associates enrolled 85 pediatric patients into the study who were diagnosed with an initial episode of acute pancreatitis at a center participating in the International Study Group of Pediatric Pancreatitis: In Search for a Cure (INSPPIRE) during the 37 months from March 2013 to April 2016. The average age of the patients was 14 years. They came from the 14 centers participating in INSPPIRE, including 10 U.S. based locations. Nearly a third of the pancreatitis cases had an idiopathic cause, a toxin or drug was implicated in 18%, a virus or other systemic cause in 18%, a biliary or gallstone problem in 17%, trauma in 9%, a genetic cause in six patients (mutations in PRSS1, SPNK-1, and CFTR), 7%, and one patient had a metabolic etiology.

The analysis focused on the 83 patients with at least 3 months of follow-up. During observation, 17 (20%) of the patients developed a second episode of acute pancreatitis that was distinguished from the initial episode by either at least 1 pain-free month or by complete normalization of amylase and lipase levels between the two episodes. Thirteen of the 17 recurrences occurred within 5 months of the first episode, with 11 of these occurring within the first 3 months after the first attack, a subgroup Dr. Sweeny called the “rapid progressors.”

Comparison of the 11 rapid progressors with the other 72 patients showed that the rapid progressors were significantly taller and weighed more. In addition, two of the 11 rapid progressors had pancreatic necrosis while none of the other patients had this complication.

The pancreatitis etiologies of the 11 rapid progressors also highlighted the potent influence a mutation can have on producing recurrent acute pancreatitis. Four of the 11 rapid progressors had a genetic mutation linked to pancreatitis susceptibility, and five of the six patients with a genetic cause for their index episode of pancreatitis developed a second acute episode during follow-up, said Dr. Sweeny, a pediatrician at Cincinnati Children’s Hospital Medical Center. In contrast, the next most effective cause of recurrent pancreatitis was a toxin or drug, which resulted in about a 25% incidence rate of a second episode. All of the other pancreatitis etiologies had recurrence rates of 10% or less.

Collecting better information on the causes of recurrent pancreatitis and chronic pancreatitis is especially important because of the rising incidence of acute pediatric pancreatitis, currently about one case in every 10,000 children and adolescents. Prior to formation of the INSPPIRE consortium, studies of pediatric pancreatitis had largely been limited to single-center retrospective reviews. The limitations of these data have made it hard to predict which patients with a first episode of acute pancreatitis will progress to a second episode or beyond, Dr. Sweeny said.

Dr. Sweeny had no disclosures.

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– Once children have a first bout of acute pancreatitis, a second, separate episode of acute pancreatitis most often occurs in patients with genetically triggered pancreatitis, those who are taller or weigh more than average, and patients with pancreatic necrosis, based on multicenter, prospective data collected from 83 patients.

This is the first reported study to prospectively follow pediatric cases of acute pancreatitis, and additional studies with more patients are needed to better identify the factors predisposing patients to recurrent episodes of acute pancreatitis and to quantify the amount of risk these factors pose, Katherine F. Sweeny, MD, said at the annual meeting of the Federation of the International Societies of Pediatric Gastroenterology, Hepatology, and Nutrition.

 



Mitchel L. Zoler/Frontline Medical News
Dr. Katherine F. Sweeny
Dr. Sweeny and her associates enrolled 85 pediatric patients into the study who were diagnosed with an initial episode of acute pancreatitis at a center participating in the International Study Group of Pediatric Pancreatitis: In Search for a Cure (INSPPIRE) during the 37 months from March 2013 to April 2016. The average age of the patients was 14 years. They came from the 14 centers participating in INSPPIRE, including 10 U.S. based locations. Nearly a third of the pancreatitis cases had an idiopathic cause, a toxin or drug was implicated in 18%, a virus or other systemic cause in 18%, a biliary or gallstone problem in 17%, trauma in 9%, a genetic cause in six patients (mutations in PRSS1, SPNK-1, and CFTR), 7%, and one patient had a metabolic etiology.

The analysis focused on the 83 patients with at least 3 months of follow-up. During observation, 17 (20%) of the patients developed a second episode of acute pancreatitis that was distinguished from the initial episode by either at least 1 pain-free month or by complete normalization of amylase and lipase levels between the two episodes. Thirteen of the 17 recurrences occurred within 5 months of the first episode, with 11 of these occurring within the first 3 months after the first attack, a subgroup Dr. Sweeny called the “rapid progressors.”

Comparison of the 11 rapid progressors with the other 72 patients showed that the rapid progressors were significantly taller and weighed more. In addition, two of the 11 rapid progressors had pancreatic necrosis while none of the other patients had this complication.

The pancreatitis etiologies of the 11 rapid progressors also highlighted the potent influence a mutation can have on producing recurrent acute pancreatitis. Four of the 11 rapid progressors had a genetic mutation linked to pancreatitis susceptibility, and five of the six patients with a genetic cause for their index episode of pancreatitis developed a second acute episode during follow-up, said Dr. Sweeny, a pediatrician at Cincinnati Children’s Hospital Medical Center. In contrast, the next most effective cause of recurrent pancreatitis was a toxin or drug, which resulted in about a 25% incidence rate of a second episode. All of the other pancreatitis etiologies had recurrence rates of 10% or less.

Collecting better information on the causes of recurrent pancreatitis and chronic pancreatitis is especially important because of the rising incidence of acute pediatric pancreatitis, currently about one case in every 10,000 children and adolescents. Prior to formation of the INSPPIRE consortium, studies of pediatric pancreatitis had largely been limited to single-center retrospective reviews. The limitations of these data have made it hard to predict which patients with a first episode of acute pancreatitis will progress to a second episode or beyond, Dr. Sweeny said.

Dr. Sweeny had no disclosures.

 

– Once children have a first bout of acute pancreatitis, a second, separate episode of acute pancreatitis most often occurs in patients with genetically triggered pancreatitis, those who are taller or weigh more than average, and patients with pancreatic necrosis, based on multicenter, prospective data collected from 83 patients.

This is the first reported study to prospectively follow pediatric cases of acute pancreatitis, and additional studies with more patients are needed to better identify the factors predisposing patients to recurrent episodes of acute pancreatitis and to quantify the amount of risk these factors pose, Katherine F. Sweeny, MD, said at the annual meeting of the Federation of the International Societies of Pediatric Gastroenterology, Hepatology, and Nutrition.

 



Mitchel L. Zoler/Frontline Medical News
Dr. Katherine F. Sweeny
Dr. Sweeny and her associates enrolled 85 pediatric patients into the study who were diagnosed with an initial episode of acute pancreatitis at a center participating in the International Study Group of Pediatric Pancreatitis: In Search for a Cure (INSPPIRE) during the 37 months from March 2013 to April 2016. The average age of the patients was 14 years. They came from the 14 centers participating in INSPPIRE, including 10 U.S. based locations. Nearly a third of the pancreatitis cases had an idiopathic cause, a toxin or drug was implicated in 18%, a virus or other systemic cause in 18%, a biliary or gallstone problem in 17%, trauma in 9%, a genetic cause in six patients (mutations in PRSS1, SPNK-1, and CFTR), 7%, and one patient had a metabolic etiology.

The analysis focused on the 83 patients with at least 3 months of follow-up. During observation, 17 (20%) of the patients developed a second episode of acute pancreatitis that was distinguished from the initial episode by either at least 1 pain-free month or by complete normalization of amylase and lipase levels between the two episodes. Thirteen of the 17 recurrences occurred within 5 months of the first episode, with 11 of these occurring within the first 3 months after the first attack, a subgroup Dr. Sweeny called the “rapid progressors.”

Comparison of the 11 rapid progressors with the other 72 patients showed that the rapid progressors were significantly taller and weighed more. In addition, two of the 11 rapid progressors had pancreatic necrosis while none of the other patients had this complication.

The pancreatitis etiologies of the 11 rapid progressors also highlighted the potent influence a mutation can have on producing recurrent acute pancreatitis. Four of the 11 rapid progressors had a genetic mutation linked to pancreatitis susceptibility, and five of the six patients with a genetic cause for their index episode of pancreatitis developed a second acute episode during follow-up, said Dr. Sweeny, a pediatrician at Cincinnati Children’s Hospital Medical Center. In contrast, the next most effective cause of recurrent pancreatitis was a toxin or drug, which resulted in about a 25% incidence rate of a second episode. All of the other pancreatitis etiologies had recurrence rates of 10% or less.

Collecting better information on the causes of recurrent pancreatitis and chronic pancreatitis is especially important because of the rising incidence of acute pediatric pancreatitis, currently about one case in every 10,000 children and adolescents. Prior to formation of the INSPPIRE consortium, studies of pediatric pancreatitis had largely been limited to single-center retrospective reviews. The limitations of these data have made it hard to predict which patients with a first episode of acute pancreatitis will progress to a second episode or beyond, Dr. Sweeny said.

Dr. Sweeny had no disclosures.

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Key clinical point: Recurrent episodes of acute pancreatitis in children and adolescents were linked with above-average weight and height, pancreatic necrosis, and genetic mutations causing the pancreatitis.

Major finding: Overall, 17 of 83 patients (20%) had recurrent acute pancreatitis, but among six patients with a genetic cause, five had recurrences.

Data source: Eighty-three patients enrolled in INSPPIRE, an international consortium formed to prospectively study pediatric pancreatitis.

Disclosures: Dr. Sweeny had no disclosures.

Statins Are Underused in Recent-Onset Parkinson’s Disease

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Many patients with recent-onset Parkinson’s disease have increased cardiovascular risk, but statin therapy is underused in this population, according to research published online ahead of print September 26 in the Journal of Neurology, Neurosurgery and Psychiatry. Increased cardiovascular risk is associated with greater motor and cognitive severity and greater axial impairment. Increased use of statins may reduce chronic vascular damage, and thereby slow the progression of motor and cognitive decline in this population, said the authors.

Cardiovascular disease influences phenotypic variation in Parkinson’s disease, but data are unclear about whether cardiovascular risk factors influence Parkinson’s disease phenotype, said Diane M. A. Swallow, MB, ChB, a clinical research fellow in movement disorders at Queen Elizabeth University Hospital in Glasgow. She and her colleagues studied prospectively enrolled participants in the UK Tracking Parkinson’s study and the Oxford Discovery study to quantify vascular risk and statin use in patients with recent-onset Parkinson’s disease. The investigators also sought to clarify the relationship between vascular risk and the severity and phenotype of Parkinson’s disease.

Cardiovascular risk was quantified using the QRISK2-2015 prediction algorithm, which computes risk based on traditional risk factors (eg, age, systolic blood pressure, smoking status, and ratio of total serum cholesterol to high-density lipoprotein cholesterol) together with BMI, ethnicity, measures of deprivation, family history, chronic kidney disease, rheumatoid arthritis, atrial fibrillation, diabetes mellitus, and antihypertensive treatment.

In all, 2,909 patients were analyzed. The population’s mean age was 67.5, mean disease duration was 1.3 years, and 65.3% of the sample was male. As calculated by the QRISK2-2015, 10-year cardiovascular risk was low for 22.3%, medium for 28.7%, and high for 33.8% of the sample, respectively. Statins were prescribed in 15.1% of patients with medium vascular risk and in 37.2% of patients with high vascular risk. In contrast, statins were prescribed in 75.3% of participants with established vascular disease.

Increasing vascular risk was associated with worsening scores on Part 3 of the Unified Parkinson’s Disease Rating Scale when adjusted for age, sex, disease duration, and coffee consumption. Increasing vascular risk was also associated with increasing cognitive impairment, a worsening score on the Montreal Cognitive Assessment (MoCA), and an increasing proportion of patients with mild cognitive impairment and dementia. The motor and cognitive characteristics of participants with high vascular risk were similar to those of participants with established vascular disease.

Among individuals with established vascular disease and a QRISK2 score of 10% or greater, people who were treated with statins were less likely to have the postural instability and gait difficulty subtype of Parkinson’s disease, but had lower total MoCA scores and were more likely to have cognitive impairment. Among patients with established cardiovascular disease, people treated with statins had better cognition, but did not significantly differ in their likelihood of having postural instability and gait difficulty.

“In assessing our findings, we need to consider that the patients are likely to have been recommended for statins based on their clinical findings, so we cannot compare these groups as if they had been randomized to statin therapy. It is clear that increased vascular risk adds to the burden of gait and cognitive impairment that we see in patients with Parkinson’s disease, and that this [risk] is undertreated,” said Dr. Swallow.

Erik Greb

Suggested Reading

Swallow DM, Lawton MA, Grosset KA, et al. Statins are underused in recent-onset Parkinson’s disease with increased vascular risk: findings from the UK Tracking Parkinson’s and Oxford Parkinson’s Disease Centre (OPDC) discovery cohorts. J Neurol Neurosurg Psychiatry. 2016 Sep 26 [Epub ahead of print].

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Many patients with recent-onset Parkinson’s disease have increased cardiovascular risk, but statin therapy is underused in this population, according to research published online ahead of print September 26 in the Journal of Neurology, Neurosurgery and Psychiatry. Increased cardiovascular risk is associated with greater motor and cognitive severity and greater axial impairment. Increased use of statins may reduce chronic vascular damage, and thereby slow the progression of motor and cognitive decline in this population, said the authors.

Cardiovascular disease influences phenotypic variation in Parkinson’s disease, but data are unclear about whether cardiovascular risk factors influence Parkinson’s disease phenotype, said Diane M. A. Swallow, MB, ChB, a clinical research fellow in movement disorders at Queen Elizabeth University Hospital in Glasgow. She and her colleagues studied prospectively enrolled participants in the UK Tracking Parkinson’s study and the Oxford Discovery study to quantify vascular risk and statin use in patients with recent-onset Parkinson’s disease. The investigators also sought to clarify the relationship between vascular risk and the severity and phenotype of Parkinson’s disease.

Cardiovascular risk was quantified using the QRISK2-2015 prediction algorithm, which computes risk based on traditional risk factors (eg, age, systolic blood pressure, smoking status, and ratio of total serum cholesterol to high-density lipoprotein cholesterol) together with BMI, ethnicity, measures of deprivation, family history, chronic kidney disease, rheumatoid arthritis, atrial fibrillation, diabetes mellitus, and antihypertensive treatment.

In all, 2,909 patients were analyzed. The population’s mean age was 67.5, mean disease duration was 1.3 years, and 65.3% of the sample was male. As calculated by the QRISK2-2015, 10-year cardiovascular risk was low for 22.3%, medium for 28.7%, and high for 33.8% of the sample, respectively. Statins were prescribed in 15.1% of patients with medium vascular risk and in 37.2% of patients with high vascular risk. In contrast, statins were prescribed in 75.3% of participants with established vascular disease.

Increasing vascular risk was associated with worsening scores on Part 3 of the Unified Parkinson’s Disease Rating Scale when adjusted for age, sex, disease duration, and coffee consumption. Increasing vascular risk was also associated with increasing cognitive impairment, a worsening score on the Montreal Cognitive Assessment (MoCA), and an increasing proportion of patients with mild cognitive impairment and dementia. The motor and cognitive characteristics of participants with high vascular risk were similar to those of participants with established vascular disease.

Among individuals with established vascular disease and a QRISK2 score of 10% or greater, people who were treated with statins were less likely to have the postural instability and gait difficulty subtype of Parkinson’s disease, but had lower total MoCA scores and were more likely to have cognitive impairment. Among patients with established cardiovascular disease, people treated with statins had better cognition, but did not significantly differ in their likelihood of having postural instability and gait difficulty.

“In assessing our findings, we need to consider that the patients are likely to have been recommended for statins based on their clinical findings, so we cannot compare these groups as if they had been randomized to statin therapy. It is clear that increased vascular risk adds to the burden of gait and cognitive impairment that we see in patients with Parkinson’s disease, and that this [risk] is undertreated,” said Dr. Swallow.

Erik Greb

Suggested Reading

Swallow DM, Lawton MA, Grosset KA, et al. Statins are underused in recent-onset Parkinson’s disease with increased vascular risk: findings from the UK Tracking Parkinson’s and Oxford Parkinson’s Disease Centre (OPDC) discovery cohorts. J Neurol Neurosurg Psychiatry. 2016 Sep 26 [Epub ahead of print].

Many patients with recent-onset Parkinson’s disease have increased cardiovascular risk, but statin therapy is underused in this population, according to research published online ahead of print September 26 in the Journal of Neurology, Neurosurgery and Psychiatry. Increased cardiovascular risk is associated with greater motor and cognitive severity and greater axial impairment. Increased use of statins may reduce chronic vascular damage, and thereby slow the progression of motor and cognitive decline in this population, said the authors.

Cardiovascular disease influences phenotypic variation in Parkinson’s disease, but data are unclear about whether cardiovascular risk factors influence Parkinson’s disease phenotype, said Diane M. A. Swallow, MB, ChB, a clinical research fellow in movement disorders at Queen Elizabeth University Hospital in Glasgow. She and her colleagues studied prospectively enrolled participants in the UK Tracking Parkinson’s study and the Oxford Discovery study to quantify vascular risk and statin use in patients with recent-onset Parkinson’s disease. The investigators also sought to clarify the relationship between vascular risk and the severity and phenotype of Parkinson’s disease.

Cardiovascular risk was quantified using the QRISK2-2015 prediction algorithm, which computes risk based on traditional risk factors (eg, age, systolic blood pressure, smoking status, and ratio of total serum cholesterol to high-density lipoprotein cholesterol) together with BMI, ethnicity, measures of deprivation, family history, chronic kidney disease, rheumatoid arthritis, atrial fibrillation, diabetes mellitus, and antihypertensive treatment.

In all, 2,909 patients were analyzed. The population’s mean age was 67.5, mean disease duration was 1.3 years, and 65.3% of the sample was male. As calculated by the QRISK2-2015, 10-year cardiovascular risk was low for 22.3%, medium for 28.7%, and high for 33.8% of the sample, respectively. Statins were prescribed in 15.1% of patients with medium vascular risk and in 37.2% of patients with high vascular risk. In contrast, statins were prescribed in 75.3% of participants with established vascular disease.

Increasing vascular risk was associated with worsening scores on Part 3 of the Unified Parkinson’s Disease Rating Scale when adjusted for age, sex, disease duration, and coffee consumption. Increasing vascular risk was also associated with increasing cognitive impairment, a worsening score on the Montreal Cognitive Assessment (MoCA), and an increasing proportion of patients with mild cognitive impairment and dementia. The motor and cognitive characteristics of participants with high vascular risk were similar to those of participants with established vascular disease.

Among individuals with established vascular disease and a QRISK2 score of 10% or greater, people who were treated with statins were less likely to have the postural instability and gait difficulty subtype of Parkinson’s disease, but had lower total MoCA scores and were more likely to have cognitive impairment. Among patients with established cardiovascular disease, people treated with statins had better cognition, but did not significantly differ in their likelihood of having postural instability and gait difficulty.

“In assessing our findings, we need to consider that the patients are likely to have been recommended for statins based on their clinical findings, so we cannot compare these groups as if they had been randomized to statin therapy. It is clear that increased vascular risk adds to the burden of gait and cognitive impairment that we see in patients with Parkinson’s disease, and that this [risk] is undertreated,” said Dr. Swallow.

Erik Greb

Suggested Reading

Swallow DM, Lawton MA, Grosset KA, et al. Statins are underused in recent-onset Parkinson’s disease with increased vascular risk: findings from the UK Tracking Parkinson’s and Oxford Parkinson’s Disease Centre (OPDC) discovery cohorts. J Neurol Neurosurg Psychiatry. 2016 Sep 26 [Epub ahead of print].

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How Much MS Disease Activity Is Acceptable?

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LONDON—No evidence of disease activity (NEDA) is a worthy but potentially problematic goal in the care of patients with multiple sclerosis (MS), according to an overview provided at the 32nd Congress of the European Committee for Treatment and Research in MS. The definition of NEDA is evolving as neurologists adopt new metrics in clinical practice, and NEDA is a difficult outcome to sustain in the long term.

Studies of interferon beta suggest that a minimal amount of clinical or MRI activity is not necessarily associated with poor long-term outcomes. Such research raises the possibility that minimal evidence of disease activity (MEDA) could be an appropriate goal of treatment, said Mar Tintoré, MD, PhD, neurologist at the MS Centre of Catalonia in Barcelona.

Mar Tintoré, MD, PhD

The Emergence of NEDA

A patient receiving treatment who has no sustained disability progression, no relapses, and no new T2 or gadolinium-enhancing lesions has NEDA, as the term was originally conceived. The outcome was rapidly adopted in clinical trials. NEDA also has been applied in the clinical setting. In one study of patients with MS treated in clinical practice, 54% had NEDA at one year. Researchers also found that NEDA may predict long-term outcomes. In one study, 78% of patients with NEDA at year two did not have disease progression at year seven.

A drawback of NEDA is that disease activity may be diffuse and may not manifest itself in relapses, new lesions, or disability progression. “It also may give a false sense of security to the doctor and to the patient,” said Dr. Tintoré.

A fourth factor, brain volume loss, recently was incorporated into NEDA, which has come to be called NEDA-4. Two studies have determined that a 0.4% annual rate of brain volume loss is the best threshold for distinguishing between people with MS and healthy controls.

The Difficulties of NEDA

The new conception of NEDA still omits several factors that neurologists may consider important. NEDA does not take cognition into account, but adding the MS Functional Composite to the NEDA criteria could change that. Patient-reported outcomes such as fatigue, depression, and quality of life also might be appropriate outcomes to incorporate into NEDA, said Dr. Tintoré. In addition, biomarkers such as CSF levels of neurofilament light indicate treatment response and could be added to NEDA.

Current clinical practice may make it difficult to assess whether a patient has NEDA. Many neurologists perform an MRI on a patient before initiating treatment and again at 12 months. “Many of the drugs that we are currently using are not active from baseline,” said Dr. Tintoré. Because changes in the first six months of treatment do not necessarily indicate treatment response, it may be necessary to perform another MRI after several months of treatment as a second baseline measurement, said Dr. Tintoré.

Furthermore, agreement between neuroradiologists is not high. Researchers have observed great variability between readers who had been asked to assess the presence of new T2 or gadolinium-enhancing lesions. And although standardized MRI protocols have been established, they may not be followed uniformly, perhaps because of difficulties specific to given facilities.

Is MEDA an Appropriate Goal?

One potential argument in favor of adopting NEDA as a goal of treatment is that disease activity often predicts poor long-term outcomes. Dr. Tintoré and colleagues assessed the treatment effect in patients with relapsing-remitting MS after two years of interferon therapy. They followed patients for 12 years. Clinical activity during the first two years of treatment was associated with increased risks of developing secondary progressive MS, of reaching an Expanded Disability Status Scale (EDSS) score of 7, and of having a five-point increase on the EDSS at 12 years.

Yet patients with little disease activity may still have good long-term outcomes. In a separate study, Dr. Tintoré and colleagues assessed the presence of MRI lesions after one year in patients with relapsing-remitting MS who were receiving interferon treatment. Participants were followed for eight years, and the primary end point was an increase of at least two points on EDSS after eight years. The investigators found no difference in outcome at eight years between patients who had one gadolinium-enhancing lesion at one year and those who had none at one year. Patients who had two or more gadolinium-enhancing lesions at one year, however, were more likely to reach the primary end point than patients who had fewer than two gadolinium-enhancing lesions at one year.

Results were similar for new T2 lesions. There was no difference in outcome at eight years between participants with no new T2 lesions at one year and those with one or two new T2 lesions at one year. Patients with three or more new T2 lesions at one year, however, were more likely to reach the primary end point. The Rio score was able to predict which patients would have more disability progression, but NEDA was not able to do so, said Dr. Tintoré. These results are similar to those of the MRI in MS (MAGNIMS) data set, in which a low level of disease activity was not associated with poor long-term outcome.

“Combined clinical and MRI scores allow us to integrate disease activity data into individual therapeutic decisions for our patients with MS,” said Dr. Tintoré. Newer therapies have made NEDA attainable for more patients, but not for all patients. Because minimal clinical or MRI activity is not necessarily associated with a poor response in the long term, neurologists may debate whether or when MEDA is an acceptable goal of treatment.

Erik Greb

 

 

Suggested Reading

Giovannoni G, Turner B, Gnanapavan S, et al. Is it time to target no evident disease activity (NEDA) in multiple sclerosis? Mult Scler Relat Disord. 2015;4(4):329-333.

Kappos L, De Stefano N, Freedman MS, et al. Inclusion of brain volume loss in a revised measure of ‘no evidence of disease activity’ (NEDA-4) in relapsing-remitting multiple sclerosis. Mult Scler. 2016;22(10):1297-1305.

Río J, Castilló J, Rovira A, et al. Measures in the first year of therapy predict the response to interferon beta in MS. Mult Scler. 2009;15(7):848-853.

Uher T, Havrdova E, Sobisek L, et al. Is no evidence of disease activity an achievable goal in MS patients on intramuscular interferon beta-1a treatment over long-term follow-up? Mult Scler. 2016 May 26 [Epub ahead of print].

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LONDON—No evidence of disease activity (NEDA) is a worthy but potentially problematic goal in the care of patients with multiple sclerosis (MS), according to an overview provided at the 32nd Congress of the European Committee for Treatment and Research in MS. The definition of NEDA is evolving as neurologists adopt new metrics in clinical practice, and NEDA is a difficult outcome to sustain in the long term.

Studies of interferon beta suggest that a minimal amount of clinical or MRI activity is not necessarily associated with poor long-term outcomes. Such research raises the possibility that minimal evidence of disease activity (MEDA) could be an appropriate goal of treatment, said Mar Tintoré, MD, PhD, neurologist at the MS Centre of Catalonia in Barcelona.

Mar Tintoré, MD, PhD

The Emergence of NEDA

A patient receiving treatment who has no sustained disability progression, no relapses, and no new T2 or gadolinium-enhancing lesions has NEDA, as the term was originally conceived. The outcome was rapidly adopted in clinical trials. NEDA also has been applied in the clinical setting. In one study of patients with MS treated in clinical practice, 54% had NEDA at one year. Researchers also found that NEDA may predict long-term outcomes. In one study, 78% of patients with NEDA at year two did not have disease progression at year seven.

A drawback of NEDA is that disease activity may be diffuse and may not manifest itself in relapses, new lesions, or disability progression. “It also may give a false sense of security to the doctor and to the patient,” said Dr. Tintoré.

A fourth factor, brain volume loss, recently was incorporated into NEDA, which has come to be called NEDA-4. Two studies have determined that a 0.4% annual rate of brain volume loss is the best threshold for distinguishing between people with MS and healthy controls.

The Difficulties of NEDA

The new conception of NEDA still omits several factors that neurologists may consider important. NEDA does not take cognition into account, but adding the MS Functional Composite to the NEDA criteria could change that. Patient-reported outcomes such as fatigue, depression, and quality of life also might be appropriate outcomes to incorporate into NEDA, said Dr. Tintoré. In addition, biomarkers such as CSF levels of neurofilament light indicate treatment response and could be added to NEDA.

Current clinical practice may make it difficult to assess whether a patient has NEDA. Many neurologists perform an MRI on a patient before initiating treatment and again at 12 months. “Many of the drugs that we are currently using are not active from baseline,” said Dr. Tintoré. Because changes in the first six months of treatment do not necessarily indicate treatment response, it may be necessary to perform another MRI after several months of treatment as a second baseline measurement, said Dr. Tintoré.

Furthermore, agreement between neuroradiologists is not high. Researchers have observed great variability between readers who had been asked to assess the presence of new T2 or gadolinium-enhancing lesions. And although standardized MRI protocols have been established, they may not be followed uniformly, perhaps because of difficulties specific to given facilities.

Is MEDA an Appropriate Goal?

One potential argument in favor of adopting NEDA as a goal of treatment is that disease activity often predicts poor long-term outcomes. Dr. Tintoré and colleagues assessed the treatment effect in patients with relapsing-remitting MS after two years of interferon therapy. They followed patients for 12 years. Clinical activity during the first two years of treatment was associated with increased risks of developing secondary progressive MS, of reaching an Expanded Disability Status Scale (EDSS) score of 7, and of having a five-point increase on the EDSS at 12 years.

Yet patients with little disease activity may still have good long-term outcomes. In a separate study, Dr. Tintoré and colleagues assessed the presence of MRI lesions after one year in patients with relapsing-remitting MS who were receiving interferon treatment. Participants were followed for eight years, and the primary end point was an increase of at least two points on EDSS after eight years. The investigators found no difference in outcome at eight years between patients who had one gadolinium-enhancing lesion at one year and those who had none at one year. Patients who had two or more gadolinium-enhancing lesions at one year, however, were more likely to reach the primary end point than patients who had fewer than two gadolinium-enhancing lesions at one year.

Results were similar for new T2 lesions. There was no difference in outcome at eight years between participants with no new T2 lesions at one year and those with one or two new T2 lesions at one year. Patients with three or more new T2 lesions at one year, however, were more likely to reach the primary end point. The Rio score was able to predict which patients would have more disability progression, but NEDA was not able to do so, said Dr. Tintoré. These results are similar to those of the MRI in MS (MAGNIMS) data set, in which a low level of disease activity was not associated with poor long-term outcome.

“Combined clinical and MRI scores allow us to integrate disease activity data into individual therapeutic decisions for our patients with MS,” said Dr. Tintoré. Newer therapies have made NEDA attainable for more patients, but not for all patients. Because minimal clinical or MRI activity is not necessarily associated with a poor response in the long term, neurologists may debate whether or when MEDA is an acceptable goal of treatment.

Erik Greb

 

 

Suggested Reading

Giovannoni G, Turner B, Gnanapavan S, et al. Is it time to target no evident disease activity (NEDA) in multiple sclerosis? Mult Scler Relat Disord. 2015;4(4):329-333.

Kappos L, De Stefano N, Freedman MS, et al. Inclusion of brain volume loss in a revised measure of ‘no evidence of disease activity’ (NEDA-4) in relapsing-remitting multiple sclerosis. Mult Scler. 2016;22(10):1297-1305.

Río J, Castilló J, Rovira A, et al. Measures in the first year of therapy predict the response to interferon beta in MS. Mult Scler. 2009;15(7):848-853.

Uher T, Havrdova E, Sobisek L, et al. Is no evidence of disease activity an achievable goal in MS patients on intramuscular interferon beta-1a treatment over long-term follow-up? Mult Scler. 2016 May 26 [Epub ahead of print].

LONDON—No evidence of disease activity (NEDA) is a worthy but potentially problematic goal in the care of patients with multiple sclerosis (MS), according to an overview provided at the 32nd Congress of the European Committee for Treatment and Research in MS. The definition of NEDA is evolving as neurologists adopt new metrics in clinical practice, and NEDA is a difficult outcome to sustain in the long term.

Studies of interferon beta suggest that a minimal amount of clinical or MRI activity is not necessarily associated with poor long-term outcomes. Such research raises the possibility that minimal evidence of disease activity (MEDA) could be an appropriate goal of treatment, said Mar Tintoré, MD, PhD, neurologist at the MS Centre of Catalonia in Barcelona.

Mar Tintoré, MD, PhD

The Emergence of NEDA

A patient receiving treatment who has no sustained disability progression, no relapses, and no new T2 or gadolinium-enhancing lesions has NEDA, as the term was originally conceived. The outcome was rapidly adopted in clinical trials. NEDA also has been applied in the clinical setting. In one study of patients with MS treated in clinical practice, 54% had NEDA at one year. Researchers also found that NEDA may predict long-term outcomes. In one study, 78% of patients with NEDA at year two did not have disease progression at year seven.

A drawback of NEDA is that disease activity may be diffuse and may not manifest itself in relapses, new lesions, or disability progression. “It also may give a false sense of security to the doctor and to the patient,” said Dr. Tintoré.

A fourth factor, brain volume loss, recently was incorporated into NEDA, which has come to be called NEDA-4. Two studies have determined that a 0.4% annual rate of brain volume loss is the best threshold for distinguishing between people with MS and healthy controls.

The Difficulties of NEDA

The new conception of NEDA still omits several factors that neurologists may consider important. NEDA does not take cognition into account, but adding the MS Functional Composite to the NEDA criteria could change that. Patient-reported outcomes such as fatigue, depression, and quality of life also might be appropriate outcomes to incorporate into NEDA, said Dr. Tintoré. In addition, biomarkers such as CSF levels of neurofilament light indicate treatment response and could be added to NEDA.

Current clinical practice may make it difficult to assess whether a patient has NEDA. Many neurologists perform an MRI on a patient before initiating treatment and again at 12 months. “Many of the drugs that we are currently using are not active from baseline,” said Dr. Tintoré. Because changes in the first six months of treatment do not necessarily indicate treatment response, it may be necessary to perform another MRI after several months of treatment as a second baseline measurement, said Dr. Tintoré.

Furthermore, agreement between neuroradiologists is not high. Researchers have observed great variability between readers who had been asked to assess the presence of new T2 or gadolinium-enhancing lesions. And although standardized MRI protocols have been established, they may not be followed uniformly, perhaps because of difficulties specific to given facilities.

Is MEDA an Appropriate Goal?

One potential argument in favor of adopting NEDA as a goal of treatment is that disease activity often predicts poor long-term outcomes. Dr. Tintoré and colleagues assessed the treatment effect in patients with relapsing-remitting MS after two years of interferon therapy. They followed patients for 12 years. Clinical activity during the first two years of treatment was associated with increased risks of developing secondary progressive MS, of reaching an Expanded Disability Status Scale (EDSS) score of 7, and of having a five-point increase on the EDSS at 12 years.

Yet patients with little disease activity may still have good long-term outcomes. In a separate study, Dr. Tintoré and colleagues assessed the presence of MRI lesions after one year in patients with relapsing-remitting MS who were receiving interferon treatment. Participants were followed for eight years, and the primary end point was an increase of at least two points on EDSS after eight years. The investigators found no difference in outcome at eight years between patients who had one gadolinium-enhancing lesion at one year and those who had none at one year. Patients who had two or more gadolinium-enhancing lesions at one year, however, were more likely to reach the primary end point than patients who had fewer than two gadolinium-enhancing lesions at one year.

Results were similar for new T2 lesions. There was no difference in outcome at eight years between participants with no new T2 lesions at one year and those with one or two new T2 lesions at one year. Patients with three or more new T2 lesions at one year, however, were more likely to reach the primary end point. The Rio score was able to predict which patients would have more disability progression, but NEDA was not able to do so, said Dr. Tintoré. These results are similar to those of the MRI in MS (MAGNIMS) data set, in which a low level of disease activity was not associated with poor long-term outcome.

“Combined clinical and MRI scores allow us to integrate disease activity data into individual therapeutic decisions for our patients with MS,” said Dr. Tintoré. Newer therapies have made NEDA attainable for more patients, but not for all patients. Because minimal clinical or MRI activity is not necessarily associated with a poor response in the long term, neurologists may debate whether or when MEDA is an acceptable goal of treatment.

Erik Greb

 

 

Suggested Reading

Giovannoni G, Turner B, Gnanapavan S, et al. Is it time to target no evident disease activity (NEDA) in multiple sclerosis? Mult Scler Relat Disord. 2015;4(4):329-333.

Kappos L, De Stefano N, Freedman MS, et al. Inclusion of brain volume loss in a revised measure of ‘no evidence of disease activity’ (NEDA-4) in relapsing-remitting multiple sclerosis. Mult Scler. 2016;22(10):1297-1305.

Río J, Castilló J, Rovira A, et al. Measures in the first year of therapy predict the response to interferon beta in MS. Mult Scler. 2009;15(7):848-853.

Uher T, Havrdova E, Sobisek L, et al. Is no evidence of disease activity an achievable goal in MS patients on intramuscular interferon beta-1a treatment over long-term follow-up? Mult Scler. 2016 May 26 [Epub ahead of print].

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Delaying cancer treatment for fertility preservation did not affect outcomes

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– Delaying cancer treatment to allow women to undergo fertility preservation did not affect long-term cancer outcomes, suggest the findings from a retrospective study of more than 500 patients.

Over a median of 3.7 years of follow-up, 2.3% of patients who elected fertility preservation developed recurrent cancer, compared with 5.3% of patients who did not undergo fertility preservation (P = .09), according to Molly Moravek, MD, of the University of Michigan, Ann Arbor, and her associates.

Survival rates were 97.7% for patients who underwent fertility preservation and 94.1% for those who did not (P = .05), the investigators reported in a poster at the annual meeting of the American Society for Reproductive Medicine.

“Pursuing fertility preservation results in minimal delays to initiation of cancer treatment and is unlikely to be clinically significant,” the investigators wrote. “There is no evidence of increased recurrence or mortality in fertility preservation patients versus controls, suggesting fertility preservation is safe for eligible cancer patients.”

Progress in cancer detection and survival has sharpened the focus on quality of life issues, including fertility preservation, the researchers said. But oncologists and patients themselves have raised concerns about postponing cancer treatment for this reason, and some have recommended shortening the delay by triggering ovarian stimulation regardless of the phase of the menstrual cycle – known as the “random start” protocol.

To explore these issues, the researchers reviewed the charts of all 553 cancer patients who had used the online patient navigator for fertility preservation at Northwestern University from 2006 to 2015.

A total of 213 patients pursued fertility preservation, while 340 did not. Undergoing fertility preservation postponed treatment of breast, hematologic, gynecologic, and other cancers by an average of 10 days, but this delay did not translate to worse recurrence rates or mortality, either overall or for any cancer subtype.

Cycle outcomes were similar between the 117 patients who underwent random-start protocols and the 23 patients underwent cycle-specific protocols, the investigators reported. Both protocols were associated with similar numbers of oocytes retrieved, numbers of mature oocytes, peak serum estradiol levels, days of stimulation, and times to cancer treatment.

The Northwestern Memorial Foundation supported the work. Dr. Moravek reported having no relevant financial disclosures.

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– Delaying cancer treatment to allow women to undergo fertility preservation did not affect long-term cancer outcomes, suggest the findings from a retrospective study of more than 500 patients.

Over a median of 3.7 years of follow-up, 2.3% of patients who elected fertility preservation developed recurrent cancer, compared with 5.3% of patients who did not undergo fertility preservation (P = .09), according to Molly Moravek, MD, of the University of Michigan, Ann Arbor, and her associates.

Survival rates were 97.7% for patients who underwent fertility preservation and 94.1% for those who did not (P = .05), the investigators reported in a poster at the annual meeting of the American Society for Reproductive Medicine.

“Pursuing fertility preservation results in minimal delays to initiation of cancer treatment and is unlikely to be clinically significant,” the investigators wrote. “There is no evidence of increased recurrence or mortality in fertility preservation patients versus controls, suggesting fertility preservation is safe for eligible cancer patients.”

Progress in cancer detection and survival has sharpened the focus on quality of life issues, including fertility preservation, the researchers said. But oncologists and patients themselves have raised concerns about postponing cancer treatment for this reason, and some have recommended shortening the delay by triggering ovarian stimulation regardless of the phase of the menstrual cycle – known as the “random start” protocol.

To explore these issues, the researchers reviewed the charts of all 553 cancer patients who had used the online patient navigator for fertility preservation at Northwestern University from 2006 to 2015.

A total of 213 patients pursued fertility preservation, while 340 did not. Undergoing fertility preservation postponed treatment of breast, hematologic, gynecologic, and other cancers by an average of 10 days, but this delay did not translate to worse recurrence rates or mortality, either overall or for any cancer subtype.

Cycle outcomes were similar between the 117 patients who underwent random-start protocols and the 23 patients underwent cycle-specific protocols, the investigators reported. Both protocols were associated with similar numbers of oocytes retrieved, numbers of mature oocytes, peak serum estradiol levels, days of stimulation, and times to cancer treatment.

The Northwestern Memorial Foundation supported the work. Dr. Moravek reported having no relevant financial disclosures.

 

– Delaying cancer treatment to allow women to undergo fertility preservation did not affect long-term cancer outcomes, suggest the findings from a retrospective study of more than 500 patients.

Over a median of 3.7 years of follow-up, 2.3% of patients who elected fertility preservation developed recurrent cancer, compared with 5.3% of patients who did not undergo fertility preservation (P = .09), according to Molly Moravek, MD, of the University of Michigan, Ann Arbor, and her associates.

Survival rates were 97.7% for patients who underwent fertility preservation and 94.1% for those who did not (P = .05), the investigators reported in a poster at the annual meeting of the American Society for Reproductive Medicine.

“Pursuing fertility preservation results in minimal delays to initiation of cancer treatment and is unlikely to be clinically significant,” the investigators wrote. “There is no evidence of increased recurrence or mortality in fertility preservation patients versus controls, suggesting fertility preservation is safe for eligible cancer patients.”

Progress in cancer detection and survival has sharpened the focus on quality of life issues, including fertility preservation, the researchers said. But oncologists and patients themselves have raised concerns about postponing cancer treatment for this reason, and some have recommended shortening the delay by triggering ovarian stimulation regardless of the phase of the menstrual cycle – known as the “random start” protocol.

To explore these issues, the researchers reviewed the charts of all 553 cancer patients who had used the online patient navigator for fertility preservation at Northwestern University from 2006 to 2015.

A total of 213 patients pursued fertility preservation, while 340 did not. Undergoing fertility preservation postponed treatment of breast, hematologic, gynecologic, and other cancers by an average of 10 days, but this delay did not translate to worse recurrence rates or mortality, either overall or for any cancer subtype.

Cycle outcomes were similar between the 117 patients who underwent random-start protocols and the 23 patients underwent cycle-specific protocols, the investigators reported. Both protocols were associated with similar numbers of oocytes retrieved, numbers of mature oocytes, peak serum estradiol levels, days of stimulation, and times to cancer treatment.

The Northwestern Memorial Foundation supported the work. Dr. Moravek reported having no relevant financial disclosures.

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Key clinical point: Delaying cancer treatment for fertility preservation did not affect long-term outcomes.

Major finding: Fertility preservation was associated with an average 10-day delay in cancer treatment, which did not affect rates of cancer recurrence or mortality.

Data source: Retrospective chart reviews of 553 patients with breast, hematologic, ovarian, and other cancers, with a median of 3.7 years of follow-up.

Disclosures: The Northwestern Memorial Foundation supported the work. Dr. Moravek reported having no relevant financial disclosures.

Women recovered half their ovarian reserve 13 months after chemotherapy

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– Women who resumed menstruating after undergoing chemotherapy for breast cancer usually recovered about half their ovarian reserve 13 months later, according to interim results from a prospective cohort study.

©BluePlanetEarth/thinkstockphotos.com
Receiving leuprolide (Lupron) during chemotherapy appeared to increase post-chemo antral follicle counts, with trends toward statistical significance at several time points, the investigators also reported.

Chemotherapy increases the risk of infertility and early menopause. But in past studies, some 70% to 80% of women regained at least some level of ovarian function after completing treatment, the researchers noted. Over the course of 6-12 months, quiescent follicles mature to the antral stage, in which exposure to follicle-stimulating hormone triggers their rapid growth and maturation. Thus, antral follicle count is an accepted marker of ovarian reserve that reliably predicts how many oocytes will be retrieved during in vitro fertilization and fertility preservation, according to the investigators.

Based on these observations, they tracked antral follicle counts over time among 199 patients who were seen for fertility preservation consultations before starting cyclophosphamide-based chemotherapy for breast cancer, and who resumed menstruating afterward. Before chemotherapy, these women had an average antral follicle count of 15, with a standard deviation of 12. They averaged 35 years of age, with a standard deviation of 5 years.

A total of 66 women returned after chemotherapy for follow-up, and underwent an average of four antral follicle counts, with a standard deviation of two, the researchers said. These measurements showed that for up to 12 months after finishing chemotherapy, patients typically had only about 14% to 25% of their baseline ovarian reserve. By month 13, however, antral follicle counts rose to an average of 52% of baseline, and remained at this level through month 18 and beyond.

Treatment with leuprolide during chemotherapy appeared to increase ovarian recovery from about month 7 onward, the researchers reported. Between 7 and 9 months after chemotherapy, antral follicle counts averaged 32% of baseline among patients who had received leuprolide, but were about 8% of baseline among patients who had not received leuprolide. Between months 13 and 18, leuprolide recipients recovered about 74% of their ovarian reserve, while other patients recovered about 35% (P = .09). Beyond month 18, leuprolide recipients recovered an average of 69% of their baseline antral follicle count and other patients recovered an average of 4 (P = .07).

“Women who did not take [leuprolide] during chemotherapy represent 75% of our study population,” the researchers said. “[These women] appeared to have lower antral follicle count recovery, despite resumption of menses, than those whose menses resumed after chemotherapy with concurrent [leuprolide].”

Dr. Letourneau reported having no relevant financial disclosures.
 

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– Women who resumed menstruating after undergoing chemotherapy for breast cancer usually recovered about half their ovarian reserve 13 months later, according to interim results from a prospective cohort study.

©BluePlanetEarth/thinkstockphotos.com
Receiving leuprolide (Lupron) during chemotherapy appeared to increase post-chemo antral follicle counts, with trends toward statistical significance at several time points, the investigators also reported.

Chemotherapy increases the risk of infertility and early menopause. But in past studies, some 70% to 80% of women regained at least some level of ovarian function after completing treatment, the researchers noted. Over the course of 6-12 months, quiescent follicles mature to the antral stage, in which exposure to follicle-stimulating hormone triggers their rapid growth and maturation. Thus, antral follicle count is an accepted marker of ovarian reserve that reliably predicts how many oocytes will be retrieved during in vitro fertilization and fertility preservation, according to the investigators.

Based on these observations, they tracked antral follicle counts over time among 199 patients who were seen for fertility preservation consultations before starting cyclophosphamide-based chemotherapy for breast cancer, and who resumed menstruating afterward. Before chemotherapy, these women had an average antral follicle count of 15, with a standard deviation of 12. They averaged 35 years of age, with a standard deviation of 5 years.

A total of 66 women returned after chemotherapy for follow-up, and underwent an average of four antral follicle counts, with a standard deviation of two, the researchers said. These measurements showed that for up to 12 months after finishing chemotherapy, patients typically had only about 14% to 25% of their baseline ovarian reserve. By month 13, however, antral follicle counts rose to an average of 52% of baseline, and remained at this level through month 18 and beyond.

Treatment with leuprolide during chemotherapy appeared to increase ovarian recovery from about month 7 onward, the researchers reported. Between 7 and 9 months after chemotherapy, antral follicle counts averaged 32% of baseline among patients who had received leuprolide, but were about 8% of baseline among patients who had not received leuprolide. Between months 13 and 18, leuprolide recipients recovered about 74% of their ovarian reserve, while other patients recovered about 35% (P = .09). Beyond month 18, leuprolide recipients recovered an average of 69% of their baseline antral follicle count and other patients recovered an average of 4 (P = .07).

“Women who did not take [leuprolide] during chemotherapy represent 75% of our study population,” the researchers said. “[These women] appeared to have lower antral follicle count recovery, despite resumption of menses, than those whose menses resumed after chemotherapy with concurrent [leuprolide].”

Dr. Letourneau reported having no relevant financial disclosures.
 

 

– Women who resumed menstruating after undergoing chemotherapy for breast cancer usually recovered about half their ovarian reserve 13 months later, according to interim results from a prospective cohort study.

©BluePlanetEarth/thinkstockphotos.com
Receiving leuprolide (Lupron) during chemotherapy appeared to increase post-chemo antral follicle counts, with trends toward statistical significance at several time points, the investigators also reported.

Chemotherapy increases the risk of infertility and early menopause. But in past studies, some 70% to 80% of women regained at least some level of ovarian function after completing treatment, the researchers noted. Over the course of 6-12 months, quiescent follicles mature to the antral stage, in which exposure to follicle-stimulating hormone triggers their rapid growth and maturation. Thus, antral follicle count is an accepted marker of ovarian reserve that reliably predicts how many oocytes will be retrieved during in vitro fertilization and fertility preservation, according to the investigators.

Based on these observations, they tracked antral follicle counts over time among 199 patients who were seen for fertility preservation consultations before starting cyclophosphamide-based chemotherapy for breast cancer, and who resumed menstruating afterward. Before chemotherapy, these women had an average antral follicle count of 15, with a standard deviation of 12. They averaged 35 years of age, with a standard deviation of 5 years.

A total of 66 women returned after chemotherapy for follow-up, and underwent an average of four antral follicle counts, with a standard deviation of two, the researchers said. These measurements showed that for up to 12 months after finishing chemotherapy, patients typically had only about 14% to 25% of their baseline ovarian reserve. By month 13, however, antral follicle counts rose to an average of 52% of baseline, and remained at this level through month 18 and beyond.

Treatment with leuprolide during chemotherapy appeared to increase ovarian recovery from about month 7 onward, the researchers reported. Between 7 and 9 months after chemotherapy, antral follicle counts averaged 32% of baseline among patients who had received leuprolide, but were about 8% of baseline among patients who had not received leuprolide. Between months 13 and 18, leuprolide recipients recovered about 74% of their ovarian reserve, while other patients recovered about 35% (P = .09). Beyond month 18, leuprolide recipients recovered an average of 69% of their baseline antral follicle count and other patients recovered an average of 4 (P = .07).

“Women who did not take [leuprolide] during chemotherapy represent 75% of our study population,” the researchers said. “[These women] appeared to have lower antral follicle count recovery, despite resumption of menses, than those whose menses resumed after chemotherapy with concurrent [leuprolide].”

Dr. Letourneau reported having no relevant financial disclosures.
 

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Key clinical point: Women with breast cancer who resume menstruating after completing chemotherapy can expect to recover about half their ovarian reserve after a year.

Major finding: Average antral follicle counts rose to 52% of baseline, on average, by 13 months after patients completed chemotherapy.

Data source: A prospective cohort study of 66 patients who resumed menstruating and returned for at least one follow-up visit after chemotherapy.

Disclosures: Dr. Letourneau reported having no relevant financial disclosures.

Polychlorinated Biphenyls Are Associated With Risk of Parkinson’s Disease

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PORTLAND, OR—Higher serum levels of polychlorinated biphenyls (PCBs) were associated with an increased risk of Parkinson’s disease in two independent study populations, according to research data presented at the Fourth World Parkinson Congress.

Samuel Goldman, MD, MPH, Principal Investigator of Neurology at San Francisco Veterans Affairs Medical Center and Associate Professor of Neurology at the University of California, San Francisco, and colleagues examined whether recently observed associations of serum PCBs and Parkinson’s disease could be replicated in an independent study population.

PCBs—persistent environmental pollutants that are detectable in most people despite a worldwide ban on their production that has been in place for more than 20 years—cause selective dopaminergic toxicity in animal models, but have been minimally studied in Parkinson’s disease, the researchers said.

The investigators recently reported a significantly increased risk of Parkinson’s disease associated with higher levels of serum PCBs in a case–control study of Alaska Native people. In the present study, they investigated this association in a demographically dissimilar study population.

They identified people with Parkinson’s disease within the Agricultural Health Study, a cohort of pesticide applicators and their spouses in Iowa and North Carolina. They also randomly selected controls matched for age, sex, and state. They confirmed Parkinson’s disease diagnoses by in-person neurologist evaluation and consensus review. PCB congeners 118, 138, 153, and 180 were measured as ng/g lipid in serum using gas chromatography–mass spectro­metry. Dose responsewas assessed using quartiles for each congener and for the sum of congeners. They used logistic regression, adjusting for age, gender, and state, to calculate odds ratios.

Ninety-seven people with Parkinson’s disease and 113 controls were included in the study. About 25% of the participants were women. Mean age was 69. Parkinson’s disease was associated with higher levels of PCBs. A significant dose response was seen across quartiles. Participants in the second, third, and fourth quartiles of total PCB levels had a 1.7-, 2.4-, and 2.7-fold greater risk of Parkinson’s disease, respectively, compared with participants in the lowest quartile of total PCB levels. Odds ratios were similar in the Agricultural Health Study and the Alaska study. In both studies, PCB levels correlated positively with age but not with disease duration, which argues against reverse causation, the researchers said.

Jake Remaly

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PORTLAND, OR—Higher serum levels of polychlorinated biphenyls (PCBs) were associated with an increased risk of Parkinson’s disease in two independent study populations, according to research data presented at the Fourth World Parkinson Congress.

Samuel Goldman, MD, MPH, Principal Investigator of Neurology at San Francisco Veterans Affairs Medical Center and Associate Professor of Neurology at the University of California, San Francisco, and colleagues examined whether recently observed associations of serum PCBs and Parkinson’s disease could be replicated in an independent study population.

PCBs—persistent environmental pollutants that are detectable in most people despite a worldwide ban on their production that has been in place for more than 20 years—cause selective dopaminergic toxicity in animal models, but have been minimally studied in Parkinson’s disease, the researchers said.

The investigators recently reported a significantly increased risk of Parkinson’s disease associated with higher levels of serum PCBs in a case–control study of Alaska Native people. In the present study, they investigated this association in a demographically dissimilar study population.

They identified people with Parkinson’s disease within the Agricultural Health Study, a cohort of pesticide applicators and their spouses in Iowa and North Carolina. They also randomly selected controls matched for age, sex, and state. They confirmed Parkinson’s disease diagnoses by in-person neurologist evaluation and consensus review. PCB congeners 118, 138, 153, and 180 were measured as ng/g lipid in serum using gas chromatography–mass spectro­metry. Dose responsewas assessed using quartiles for each congener and for the sum of congeners. They used logistic regression, adjusting for age, gender, and state, to calculate odds ratios.

Ninety-seven people with Parkinson’s disease and 113 controls were included in the study. About 25% of the participants were women. Mean age was 69. Parkinson’s disease was associated with higher levels of PCBs. A significant dose response was seen across quartiles. Participants in the second, third, and fourth quartiles of total PCB levels had a 1.7-, 2.4-, and 2.7-fold greater risk of Parkinson’s disease, respectively, compared with participants in the lowest quartile of total PCB levels. Odds ratios were similar in the Agricultural Health Study and the Alaska study. In both studies, PCB levels correlated positively with age but not with disease duration, which argues against reverse causation, the researchers said.

Jake Remaly

PORTLAND, OR—Higher serum levels of polychlorinated biphenyls (PCBs) were associated with an increased risk of Parkinson’s disease in two independent study populations, according to research data presented at the Fourth World Parkinson Congress.

Samuel Goldman, MD, MPH, Principal Investigator of Neurology at San Francisco Veterans Affairs Medical Center and Associate Professor of Neurology at the University of California, San Francisco, and colleagues examined whether recently observed associations of serum PCBs and Parkinson’s disease could be replicated in an independent study population.

PCBs—persistent environmental pollutants that are detectable in most people despite a worldwide ban on their production that has been in place for more than 20 years—cause selective dopaminergic toxicity in animal models, but have been minimally studied in Parkinson’s disease, the researchers said.

The investigators recently reported a significantly increased risk of Parkinson’s disease associated with higher levels of serum PCBs in a case–control study of Alaska Native people. In the present study, they investigated this association in a demographically dissimilar study population.

They identified people with Parkinson’s disease within the Agricultural Health Study, a cohort of pesticide applicators and their spouses in Iowa and North Carolina. They also randomly selected controls matched for age, sex, and state. They confirmed Parkinson’s disease diagnoses by in-person neurologist evaluation and consensus review. PCB congeners 118, 138, 153, and 180 were measured as ng/g lipid in serum using gas chromatography–mass spectro­metry. Dose responsewas assessed using quartiles for each congener and for the sum of congeners. They used logistic regression, adjusting for age, gender, and state, to calculate odds ratios.

Ninety-seven people with Parkinson’s disease and 113 controls were included in the study. About 25% of the participants were women. Mean age was 69. Parkinson’s disease was associated with higher levels of PCBs. A significant dose response was seen across quartiles. Participants in the second, third, and fourth quartiles of total PCB levels had a 1.7-, 2.4-, and 2.7-fold greater risk of Parkinson’s disease, respectively, compared with participants in the lowest quartile of total PCB levels. Odds ratios were similar in the Agricultural Health Study and the Alaska study. In both studies, PCB levels correlated positively with age but not with disease duration, which argues against reverse causation, the researchers said.

Jake Remaly

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“Honoring Our Mentors” Fellowship Open for Submission

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The AATS Graham Foundation is calling for submission for its Denton A. Cooley “Honoring Our Mentors” fellowship. 


Denton A. Cooley Fellowship

New! Provides a deserving CT surgeon resident or young postgraduate surgeon the opportunity to enrich his/her education during four weeks of study at the Texas Heart Institute and Baylor St. Luke’s Medical Center.

Deadline: December 30, 2016

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The AATS Graham Foundation is calling for submission for its Denton A. Cooley “Honoring Our Mentors” fellowship. 


Denton A. Cooley Fellowship

New! Provides a deserving CT surgeon resident or young postgraduate surgeon the opportunity to enrich his/her education during four weeks of study at the Texas Heart Institute and Baylor St. Luke’s Medical Center.

Deadline: December 30, 2016

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The AATS Graham Foundation is calling for submission for its Denton A. Cooley “Honoring Our Mentors” fellowship. 


Denton A. Cooley Fellowship

New! Provides a deserving CT surgeon resident or young postgraduate surgeon the opportunity to enrich his/her education during four weeks of study at the Texas Heart Institute and Baylor St. Luke’s Medical Center.

Deadline: December 30, 2016

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