Biomarker combination may forecast remission in lupus nephritis

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MELBOURNE – A reduction in urinary protein creatinine ratio and normalization of inflammatory biomarkers early in treatment of lupus nephritis may predict response rates at 24 and 48 weeks, according to data from the AURION study presented at an international congress on systemic lupus erythematosus.

Robert Huizinga
Presenter Robert B. Huizinga, vice president of clinical affairs for Aurinia Pharmaceuticals, said the data showed there was unlikely to be one biomarker with both high sensitivity and high specificity.

“Certainly if you use C3, C4, and urinary protein creatinine ratio, you can say to the patient after week 8, ‘I don’t think this is going to work for you. You need to come off this therapy and move to something else,’ ” he said in an interview.

Mr. Huizinga said the company, which recently released 48-week data from the larger AURA-LV study of the same regimen in 265 patients, was now building this week-8 analysis into its studies, and hoped it would also provide an early predictive marker for other clinical trials.

Commenting on the findings, Brad Rovin, MD, professor of nephrology and pathology at Ohio State University, Columbus, and also an adviser to Aurinia, said this predictive ability would be extremely useful for clinicians.

“If a patient isn’t responding appropriately and you can really know that with some degree of certainty at 8 weeks, then instead of waiting 6 months to change therapy, maybe you should change earlier,” Dr. Rovin said in an interview.

This was particularly important in lupus nephritis, as the longer inflammation is allowed to continue, the greater the likelihood that it might tip over into fibrosis, he noted.

The study was funded by Aurinia.
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MELBOURNE – A reduction in urinary protein creatinine ratio and normalization of inflammatory biomarkers early in treatment of lupus nephritis may predict response rates at 24 and 48 weeks, according to data from the AURION study presented at an international congress on systemic lupus erythematosus.

Robert Huizinga
Presenter Robert B. Huizinga, vice president of clinical affairs for Aurinia Pharmaceuticals, said the data showed there was unlikely to be one biomarker with both high sensitivity and high specificity.

“Certainly if you use C3, C4, and urinary protein creatinine ratio, you can say to the patient after week 8, ‘I don’t think this is going to work for you. You need to come off this therapy and move to something else,’ ” he said in an interview.

Mr. Huizinga said the company, which recently released 48-week data from the larger AURA-LV study of the same regimen in 265 patients, was now building this week-8 analysis into its studies, and hoped it would also provide an early predictive marker for other clinical trials.

Commenting on the findings, Brad Rovin, MD, professor of nephrology and pathology at Ohio State University, Columbus, and also an adviser to Aurinia, said this predictive ability would be extremely useful for clinicians.

“If a patient isn’t responding appropriately and you can really know that with some degree of certainty at 8 weeks, then instead of waiting 6 months to change therapy, maybe you should change earlier,” Dr. Rovin said in an interview.

This was particularly important in lupus nephritis, as the longer inflammation is allowed to continue, the greater the likelihood that it might tip over into fibrosis, he noted.

The study was funded by Aurinia.

 

MELBOURNE – A reduction in urinary protein creatinine ratio and normalization of inflammatory biomarkers early in treatment of lupus nephritis may predict response rates at 24 and 48 weeks, according to data from the AURION study presented at an international congress on systemic lupus erythematosus.

Robert Huizinga
Presenter Robert B. Huizinga, vice president of clinical affairs for Aurinia Pharmaceuticals, said the data showed there was unlikely to be one biomarker with both high sensitivity and high specificity.

“Certainly if you use C3, C4, and urinary protein creatinine ratio, you can say to the patient after week 8, ‘I don’t think this is going to work for you. You need to come off this therapy and move to something else,’ ” he said in an interview.

Mr. Huizinga said the company, which recently released 48-week data from the larger AURA-LV study of the same regimen in 265 patients, was now building this week-8 analysis into its studies, and hoped it would also provide an early predictive marker for other clinical trials.

Commenting on the findings, Brad Rovin, MD, professor of nephrology and pathology at Ohio State University, Columbus, and also an adviser to Aurinia, said this predictive ability would be extremely useful for clinicians.

“If a patient isn’t responding appropriately and you can really know that with some degree of certainty at 8 weeks, then instead of waiting 6 months to change therapy, maybe you should change earlier,” Dr. Rovin said in an interview.

This was particularly important in lupus nephritis, as the longer inflammation is allowed to continue, the greater the likelihood that it might tip over into fibrosis, he noted.

The study was funded by Aurinia.
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Key clinical point: Early reduction in urinary protein creatinine ratio and normalization of inflammatory biomarkers may predict 24- and 48-week lupus nephritis treatment response rates.

Major finding: A 25% reduction in urine protein creatinine ratio, and normalization of C3 or C4 levels at 8 weeks may be predictive of complete remission at 48 weeks.

Data source: The single-center exploratory AURION study of 10 patients with active lupus nephritis.

Disclosures: Mr. Huizinga is vice president of clinical affairs for Aurinia Pharmaceuticals, which funded the study.

SPECT reveals perfusion problems in antiphospholipid syndrome

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MELBOURNE – SPECT imaging can identify abnormalities in brain perfusion in patients with multiple antiphospholipid antibodies and neuropsychiatric symptoms, but without a history of thrombosis, according to a study presented at an international congress on systemic lupus erythematosus.

Dr. Ting-Syuan Lin
Based on the study’s findings. Dr. Lin said that if patients did not show any abnormalities on CT scanning or MRI, SPECT imaging might be suitable to investigate for problems with perfusion.

“Some physicians may not give the patient early treatment because they do not fill the criteria,” Dr. Lin said in an interview. “But if we have SPECT image to document the abnormality, then the physician can have more confidence to give them early treatment.”

Dr. Lin and his colleagues looked at the brain SPECT images of 54 patients with a history of positive antiphospholipid antibodies and neuropsychiatric symptoms, but who had no history of thromboembolism or other lupus-related antibodies such as antibodies to double-stranded DNA. When the researchers looked simply at mean brain perfusion according to the number of antiphospholipid antibodies each patient had, they found no significant differences between the groups, including a control group of six patients without antiphospholipid antibodies.

But when they examined heterogeneity of brain perfusion, they saw significantly greater heterogeneity (P = .01) in patients with four antiphospholipid antibodies compared with patients who had no antibodies.

The patients enrolled in the study presented with a range of neuropsychiatric symptoms. The most common was headache (56.7%), followed by dizziness (41.7%), depression (28.3%), psychosis (15%), vertigo (8.3%), and seizures (6.7%). The mean age of the patients was 38 years, and 52 of the patients were women.

One of the patients – a 39-year-old woman with more than four antiphospholipid antibodies – had a normal CT scan but showed significant heterogeneity in brain perfusion on the SPECT imaging. She experienced a stroke 1 year after the study.

Commenting on the presentation, session cochair Timothy Godfrey, MBBS, of St. Vincent’s Hospital in Melbourne, said neuropsychiatric lupus was particularly problematic, especially when patients had normal imaging.

“You’re wondering is the patient just depressed or is there some other explanation, or do they truly have a manifestation of lupus which may require immunosuppression or anticoagulation?” he said in an interview.

This study “is highlighting the fact that maybe we do need to do these tests when the MRI is normal, particularly in people that have documented abnormalities in their blood test.”

Dr. Lin said the next phase of the study would look at whether treatment was associated with changes in brain perfusion on SPECT, and whether the abnormality of the SPECT imaging correlated with clinical outcomes.

No conflicts of interest were declared.
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MELBOURNE – SPECT imaging can identify abnormalities in brain perfusion in patients with multiple antiphospholipid antibodies and neuropsychiatric symptoms, but without a history of thrombosis, according to a study presented at an international congress on systemic lupus erythematosus.

Dr. Ting-Syuan Lin
Based on the study’s findings. Dr. Lin said that if patients did not show any abnormalities on CT scanning or MRI, SPECT imaging might be suitable to investigate for problems with perfusion.

“Some physicians may not give the patient early treatment because they do not fill the criteria,” Dr. Lin said in an interview. “But if we have SPECT image to document the abnormality, then the physician can have more confidence to give them early treatment.”

Dr. Lin and his colleagues looked at the brain SPECT images of 54 patients with a history of positive antiphospholipid antibodies and neuropsychiatric symptoms, but who had no history of thromboembolism or other lupus-related antibodies such as antibodies to double-stranded DNA. When the researchers looked simply at mean brain perfusion according to the number of antiphospholipid antibodies each patient had, they found no significant differences between the groups, including a control group of six patients without antiphospholipid antibodies.

But when they examined heterogeneity of brain perfusion, they saw significantly greater heterogeneity (P = .01) in patients with four antiphospholipid antibodies compared with patients who had no antibodies.

The patients enrolled in the study presented with a range of neuropsychiatric symptoms. The most common was headache (56.7%), followed by dizziness (41.7%), depression (28.3%), psychosis (15%), vertigo (8.3%), and seizures (6.7%). The mean age of the patients was 38 years, and 52 of the patients were women.

One of the patients – a 39-year-old woman with more than four antiphospholipid antibodies – had a normal CT scan but showed significant heterogeneity in brain perfusion on the SPECT imaging. She experienced a stroke 1 year after the study.

Commenting on the presentation, session cochair Timothy Godfrey, MBBS, of St. Vincent’s Hospital in Melbourne, said neuropsychiatric lupus was particularly problematic, especially when patients had normal imaging.

“You’re wondering is the patient just depressed or is there some other explanation, or do they truly have a manifestation of lupus which may require immunosuppression or anticoagulation?” he said in an interview.

This study “is highlighting the fact that maybe we do need to do these tests when the MRI is normal, particularly in people that have documented abnormalities in their blood test.”

Dr. Lin said the next phase of the study would look at whether treatment was associated with changes in brain perfusion on SPECT, and whether the abnormality of the SPECT imaging correlated with clinical outcomes.

No conflicts of interest were declared.

 

MELBOURNE – SPECT imaging can identify abnormalities in brain perfusion in patients with multiple antiphospholipid antibodies and neuropsychiatric symptoms, but without a history of thrombosis, according to a study presented at an international congress on systemic lupus erythematosus.

Dr. Ting-Syuan Lin
Based on the study’s findings. Dr. Lin said that if patients did not show any abnormalities on CT scanning or MRI, SPECT imaging might be suitable to investigate for problems with perfusion.

“Some physicians may not give the patient early treatment because they do not fill the criteria,” Dr. Lin said in an interview. “But if we have SPECT image to document the abnormality, then the physician can have more confidence to give them early treatment.”

Dr. Lin and his colleagues looked at the brain SPECT images of 54 patients with a history of positive antiphospholipid antibodies and neuropsychiatric symptoms, but who had no history of thromboembolism or other lupus-related antibodies such as antibodies to double-stranded DNA. When the researchers looked simply at mean brain perfusion according to the number of antiphospholipid antibodies each patient had, they found no significant differences between the groups, including a control group of six patients without antiphospholipid antibodies.

But when they examined heterogeneity of brain perfusion, they saw significantly greater heterogeneity (P = .01) in patients with four antiphospholipid antibodies compared with patients who had no antibodies.

The patients enrolled in the study presented with a range of neuropsychiatric symptoms. The most common was headache (56.7%), followed by dizziness (41.7%), depression (28.3%), psychosis (15%), vertigo (8.3%), and seizures (6.7%). The mean age of the patients was 38 years, and 52 of the patients were women.

One of the patients – a 39-year-old woman with more than four antiphospholipid antibodies – had a normal CT scan but showed significant heterogeneity in brain perfusion on the SPECT imaging. She experienced a stroke 1 year after the study.

Commenting on the presentation, session cochair Timothy Godfrey, MBBS, of St. Vincent’s Hospital in Melbourne, said neuropsychiatric lupus was particularly problematic, especially when patients had normal imaging.

“You’re wondering is the patient just depressed or is there some other explanation, or do they truly have a manifestation of lupus which may require immunosuppression or anticoagulation?” he said in an interview.

This study “is highlighting the fact that maybe we do need to do these tests when the MRI is normal, particularly in people that have documented abnormalities in their blood test.”

Dr. Lin said the next phase of the study would look at whether treatment was associated with changes in brain perfusion on SPECT, and whether the abnormality of the SPECT imaging correlated with clinical outcomes.

No conflicts of interest were declared.
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Key clinical point: SPECT may be worthwhile in patients who don’t meet antiphospholipid syndrome criteria but have aPL antibodies, neuropsychiatric symptoms, and no thrombosis history.

Major finding: Patients with four antiphospholipid antibodies have significantly greater heterogeneity in brain perfusion on SPECT imaging than do patients with no antiphospholipid antibodies.

Data source: A retrospective cohort study in 54 patients with antiphospholipid syndrome.

Disclosures: No conflicts of interest were declared.

Onstep hernia repair decreased postop pain during sexual activity

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The Onstep technique for inguinal hernia repair is associated with a lower incidence of postoperative pain during sexual activity than the Lichtenstein surgical technique, according to a paper published online in Surgery.

ChesiireCat/Thinkstock
The Onli trial was a randomized clinical trial comparing the Onstep method with the Lichtenstein method in 259 patients undergoing inguinal hernia repair found that pain during sexual activity 6 months after the operation was reported by 17 patients (13.1%) operated on with the Onstep technique and 30 patients (23%) operated on with the Lichtenstein technique (P = .034). A questionnaire, specific to pain-related impairment of sexual function, was administered at 6 months’ follow-up (Surgery. 2017 Mar 2. doi: 10.1016/j.surg.2016.12.030).

“The Onstep technique could be considered when surgeons and patients are discussing the optimal technique for repair of an inguinal hernia,” wrote Kristoffer Andresen, MD, of the University of Copenhagen, and his coauthors. “If the patient has pain during sexual activity as part of the complaint from the hernia, the Onstep technique seems to have a greater chance of removing this pain and alleviating the complaints.”

Among the 17 patients in the Onstep group who experienced postoperative pain during sexual activity, 8 had experienced preoperative pain during sexual activity and 7 had not. In the Lichtenstein group, 14 of the 30 patients who experienced postoperative pain had experienced preoperative pain, 14 had not. The remaining patients in both groups reported not having sexual activity before surgery or declined to answer.

The Lichtenstein technique was associated with new pain in 14 of the 70 patients (20%) while 7 of the 74 patients (9%) in the Onstep group reported new pain after surgery.

“For patients who experienced pain during sexual activity preoperatively, the Onstep technique removed the pain during sexual activity in the majority of patients while still resulting in few new cases,” the authors wrote.

Among the Lichtenstein group, 20 patients experienced pain from the surgical scar, compared with 7 patients in the Onstep group.

When asked about the degree of impairment of sexual function because of the postoperative pain, four patients in the Lichtenstein group said they had moderate to severe impairment, but none in the Onstep group reported that level of impairment.

Commenting on the possible mechanisms that might result in pain during sexual activity after hernia repair, the authors noted that mesh has been found to shrink from a mass around the vas deferens after a Lichtenstein repair. Previous studies have also found sutures around the iliohypogastric and the ilioinguinal nerves, which could also result in some pain.

“Because the mesh in the Onstep technique is not fixed with sutures, the risk of capturing nerves during the fixation of mesh is nonexistent,” they wrote. “There could, however, still be a risk of scar tissue and mesh shrinkage.”

PolySoft mesh (Bard Davol) was used for the Lichtenstein group and SoftMesh (Bard Davol) was used for the Onstep group.

In the 6 months’ follow-up period, two patients in the Lichtenstein group and one patient in the Onstep group experienced a recurrence of their hernia.

Three authors reported personal fees and travel grants from private industry – including Bard Medical – outside the submitted work. No other conflicts of interest were declared.

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The Onstep technique for inguinal hernia repair is associated with a lower incidence of postoperative pain during sexual activity than the Lichtenstein surgical technique, according to a paper published online in Surgery.

ChesiireCat/Thinkstock
The Onli trial was a randomized clinical trial comparing the Onstep method with the Lichtenstein method in 259 patients undergoing inguinal hernia repair found that pain during sexual activity 6 months after the operation was reported by 17 patients (13.1%) operated on with the Onstep technique and 30 patients (23%) operated on with the Lichtenstein technique (P = .034). A questionnaire, specific to pain-related impairment of sexual function, was administered at 6 months’ follow-up (Surgery. 2017 Mar 2. doi: 10.1016/j.surg.2016.12.030).

“The Onstep technique could be considered when surgeons and patients are discussing the optimal technique for repair of an inguinal hernia,” wrote Kristoffer Andresen, MD, of the University of Copenhagen, and his coauthors. “If the patient has pain during sexual activity as part of the complaint from the hernia, the Onstep technique seems to have a greater chance of removing this pain and alleviating the complaints.”

Among the 17 patients in the Onstep group who experienced postoperative pain during sexual activity, 8 had experienced preoperative pain during sexual activity and 7 had not. In the Lichtenstein group, 14 of the 30 patients who experienced postoperative pain had experienced preoperative pain, 14 had not. The remaining patients in both groups reported not having sexual activity before surgery or declined to answer.

The Lichtenstein technique was associated with new pain in 14 of the 70 patients (20%) while 7 of the 74 patients (9%) in the Onstep group reported new pain after surgery.

“For patients who experienced pain during sexual activity preoperatively, the Onstep technique removed the pain during sexual activity in the majority of patients while still resulting in few new cases,” the authors wrote.

Among the Lichtenstein group, 20 patients experienced pain from the surgical scar, compared with 7 patients in the Onstep group.

When asked about the degree of impairment of sexual function because of the postoperative pain, four patients in the Lichtenstein group said they had moderate to severe impairment, but none in the Onstep group reported that level of impairment.

Commenting on the possible mechanisms that might result in pain during sexual activity after hernia repair, the authors noted that mesh has been found to shrink from a mass around the vas deferens after a Lichtenstein repair. Previous studies have also found sutures around the iliohypogastric and the ilioinguinal nerves, which could also result in some pain.

“Because the mesh in the Onstep technique is not fixed with sutures, the risk of capturing nerves during the fixation of mesh is nonexistent,” they wrote. “There could, however, still be a risk of scar tissue and mesh shrinkage.”

PolySoft mesh (Bard Davol) was used for the Lichtenstein group and SoftMesh (Bard Davol) was used for the Onstep group.

In the 6 months’ follow-up period, two patients in the Lichtenstein group and one patient in the Onstep group experienced a recurrence of their hernia.

Three authors reported personal fees and travel grants from private industry – including Bard Medical – outside the submitted work. No other conflicts of interest were declared.

 

The Onstep technique for inguinal hernia repair is associated with a lower incidence of postoperative pain during sexual activity than the Lichtenstein surgical technique, according to a paper published online in Surgery.

ChesiireCat/Thinkstock
The Onli trial was a randomized clinical trial comparing the Onstep method with the Lichtenstein method in 259 patients undergoing inguinal hernia repair found that pain during sexual activity 6 months after the operation was reported by 17 patients (13.1%) operated on with the Onstep technique and 30 patients (23%) operated on with the Lichtenstein technique (P = .034). A questionnaire, specific to pain-related impairment of sexual function, was administered at 6 months’ follow-up (Surgery. 2017 Mar 2. doi: 10.1016/j.surg.2016.12.030).

“The Onstep technique could be considered when surgeons and patients are discussing the optimal technique for repair of an inguinal hernia,” wrote Kristoffer Andresen, MD, of the University of Copenhagen, and his coauthors. “If the patient has pain during sexual activity as part of the complaint from the hernia, the Onstep technique seems to have a greater chance of removing this pain and alleviating the complaints.”

Among the 17 patients in the Onstep group who experienced postoperative pain during sexual activity, 8 had experienced preoperative pain during sexual activity and 7 had not. In the Lichtenstein group, 14 of the 30 patients who experienced postoperative pain had experienced preoperative pain, 14 had not. The remaining patients in both groups reported not having sexual activity before surgery or declined to answer.

The Lichtenstein technique was associated with new pain in 14 of the 70 patients (20%) while 7 of the 74 patients (9%) in the Onstep group reported new pain after surgery.

“For patients who experienced pain during sexual activity preoperatively, the Onstep technique removed the pain during sexual activity in the majority of patients while still resulting in few new cases,” the authors wrote.

Among the Lichtenstein group, 20 patients experienced pain from the surgical scar, compared with 7 patients in the Onstep group.

When asked about the degree of impairment of sexual function because of the postoperative pain, four patients in the Lichtenstein group said they had moderate to severe impairment, but none in the Onstep group reported that level of impairment.

Commenting on the possible mechanisms that might result in pain during sexual activity after hernia repair, the authors noted that mesh has been found to shrink from a mass around the vas deferens after a Lichtenstein repair. Previous studies have also found sutures around the iliohypogastric and the ilioinguinal nerves, which could also result in some pain.

“Because the mesh in the Onstep technique is not fixed with sutures, the risk of capturing nerves during the fixation of mesh is nonexistent,” they wrote. “There could, however, still be a risk of scar tissue and mesh shrinkage.”

PolySoft mesh (Bard Davol) was used for the Lichtenstein group and SoftMesh (Bard Davol) was used for the Onstep group.

In the 6 months’ follow-up period, two patients in the Lichtenstein group and one patient in the Onstep group experienced a recurrence of their hernia.

Three authors reported personal fees and travel grants from private industry – including Bard Medical – outside the submitted work. No other conflicts of interest were declared.

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Key clinical point: The Onstep technique for inguinal hernia repair is associated with a lower incidence of pain during sexual activity than the Lichtenstein technique.

Major finding: The Lichtenstein technique was associated with new pain in 14 of the 70 patients (20%) while 7 of the 74 patients (9%) in the Onstep group reported new pain after surgery.

Data source: A randomized trial in 259 patients undergoing inguinal hernia repair.

Disclosures: Three authors reported personal fees and travel grants from private industry – including Bard Medical – outside the submitted work. No other conflicts of interest were declared.

Anti-TNF agents show clinical benefit in refractory sarcoidosis

Comment by Dr. Eric Gartman, MD, FCCP,
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FROM SEMINARS IN ARTHRITIS & RHEUMATISM

Around two-thirds of patients with severe or refractory sarcoidosis show a significant clinical response to tumor necrosis factor (TNF) antagonists, according to findings from a retrospective, multicenter cohort study.

Biologic agents targeting TNF, such as etanercept, infliximab, and adalimumab, have been introduced as a third-line option for patients with disease that is refractory to other treatments. However, Yvan Jamilloux, MD, of the Hospices Civils de Lyon (France) and his coauthors reported that there are still insufficient data available on efficacy and safety of these drugs in the context of sarcoidosis.

Dr. Jamilloux and his colleagues analyzed data from 132 sarcoidosis patients who received TNF antagonists, 122 (92%) of whom had severe sarcoidosis (Semin Arthritis Rheum. 2017 Mar 8. doi: 10.1016/j.semarthrit.2017.03.005).

Overall, 64% of patients showed clinical improvements in response to TNF antagonists; 18% had a complete response, and 46% had a partial response. However, 33 (25%) patients showed no change, and 14 (11%) had continued disease progression despite treatment with TNF antagonists. In another 16 patients who received a second TNF antagonist, 10 (63%) had a complete or partial clinical response. The investigators could find no differences in response between anti-TNF agents or between monotherapy and a combination with an immunosuppressant.

Pulmonary involvement was associated with a significantly lower clinical response, but none of the other factors examined in a multivariate analysis (sex, age, ethnicity, organ involvement, disease duration, steroid dosage, or prior immunosuppressant use) distinguished responders and nonresponders.

The authors noted that these response rates were lower than those seen in the literature and suggested this may be attributable to the multicenter design, more patients with longer-lasting and more refractory disease, and longer times under biologic therapy (median 12 months).

The researchers reported significant improvements in central nervous system, peripheral nervous system, heart, skin, and upper respiratory tract involvements based on declines in Extrapulmonary Physician Organ Severity Tool (ePOST) scores. There were also improvements in the eye, muscle, and lung, but these were not statistically significant.

TNF-antagonist therapy was associated with a high rate of adverse events. Around half of all patients (52%) experienced adverse events, such as pneumonia, urinary tract infections, bacterial sepsis, and herpes zoster. In 31 patients (23%), these led to treatment cessation.

Nine patients also had severe allergic reactions, four had paradoxical granulomatous reactions, three developed neutralizing antibodies against anti-TNF agents, two patients had demyelinating lesions, and one had a serum sickness-like reaction. All of these events led to discontinuation.

Overall, 128 (97%) of the patients in the study had received corticosteroids as first-line therapy, and 125 (95%) had received at least one second-line immunosuppressive drug over a median duration of 16 months. Most were treated with infliximab (91%) as the first-line TNF antagonist, followed by adalimumab (6%), etanercept (2%), and certolizumab pegol (1%).

Treatment with TNF antagonists was associated with significant reductions in corticosteroid use; the mean daily prednisone dose decreased from 23 mg/day to 11 mg/day over the median 20.5-month follow-up. This was seen even in the 33 patients who showed no change in their disease course after TNF-antagonist therapy.

No conflicts of interest were declared.

Body

This uncontrolled, unblinded retrospective observational study reports the outcomes of anti-TNF therapy in a heterogenous group of refractory sarcoid patients, with only 12% of the severe sarcoidosis population studied having the indication for treatment based on lung involvement. Further, it is notable that the patients with primarily pulmonary involvement had a poorer response to anti-TNF therapy. Over half of the patients had an adverse event related to the treatment, with nearly a quarter having to discontinue therapy. Given the limitations of this type of study, the low numbers of pulmonary sarcoid patients included, the lack of an efficacy signal in pulmonary sarcoid, and the high rate of serious adverse events – the role of anti-TNF agents for pulmonary sarcoid remains unclear and limited. However

Dr. Eric J. Gartman
, in a larger way it should be questioned if the timing of administration of these agents is important – i.e., if they are given only after significant pulmonary damage has been seen and the disease is “refractory,” this significantly may limit their potential beneficial clinical effect.
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This uncontrolled, unblinded retrospective observational study reports the outcomes of anti-TNF therapy in a heterogenous group of refractory sarcoid patients, with only 12% of the severe sarcoidosis population studied having the indication for treatment based on lung involvement. Further, it is notable that the patients with primarily pulmonary involvement had a poorer response to anti-TNF therapy. Over half of the patients had an adverse event related to the treatment, with nearly a quarter having to discontinue therapy. Given the limitations of this type of study, the low numbers of pulmonary sarcoid patients included, the lack of an efficacy signal in pulmonary sarcoid, and the high rate of serious adverse events – the role of anti-TNF agents for pulmonary sarcoid remains unclear and limited. However

Dr. Eric J. Gartman
, in a larger way it should be questioned if the timing of administration of these agents is important – i.e., if they are given only after significant pulmonary damage has been seen and the disease is “refractory,” this significantly may limit their potential beneficial clinical effect.
Body

This uncontrolled, unblinded retrospective observational study reports the outcomes of anti-TNF therapy in a heterogenous group of refractory sarcoid patients, with only 12% of the severe sarcoidosis population studied having the indication for treatment based on lung involvement. Further, it is notable that the patients with primarily pulmonary involvement had a poorer response to anti-TNF therapy. Over half of the patients had an adverse event related to the treatment, with nearly a quarter having to discontinue therapy. Given the limitations of this type of study, the low numbers of pulmonary sarcoid patients included, the lack of an efficacy signal in pulmonary sarcoid, and the high rate of serious adverse events – the role of anti-TNF agents for pulmonary sarcoid remains unclear and limited. However

Dr. Eric J. Gartman
, in a larger way it should be questioned if the timing of administration of these agents is important – i.e., if they are given only after significant pulmonary damage has been seen and the disease is “refractory,” this significantly may limit their potential beneficial clinical effect.
Title
Comment by Dr. Eric Gartman, MD, FCCP,
Comment by Dr. Eric Gartman, MD, FCCP,

 

FROM SEMINARS IN ARTHRITIS & RHEUMATISM

Around two-thirds of patients with severe or refractory sarcoidosis show a significant clinical response to tumor necrosis factor (TNF) antagonists, according to findings from a retrospective, multicenter cohort study.

Biologic agents targeting TNF, such as etanercept, infliximab, and adalimumab, have been introduced as a third-line option for patients with disease that is refractory to other treatments. However, Yvan Jamilloux, MD, of the Hospices Civils de Lyon (France) and his coauthors reported that there are still insufficient data available on efficacy and safety of these drugs in the context of sarcoidosis.

Dr. Jamilloux and his colleagues analyzed data from 132 sarcoidosis patients who received TNF antagonists, 122 (92%) of whom had severe sarcoidosis (Semin Arthritis Rheum. 2017 Mar 8. doi: 10.1016/j.semarthrit.2017.03.005).

Overall, 64% of patients showed clinical improvements in response to TNF antagonists; 18% had a complete response, and 46% had a partial response. However, 33 (25%) patients showed no change, and 14 (11%) had continued disease progression despite treatment with TNF antagonists. In another 16 patients who received a second TNF antagonist, 10 (63%) had a complete or partial clinical response. The investigators could find no differences in response between anti-TNF agents or between monotherapy and a combination with an immunosuppressant.

Pulmonary involvement was associated with a significantly lower clinical response, but none of the other factors examined in a multivariate analysis (sex, age, ethnicity, organ involvement, disease duration, steroid dosage, or prior immunosuppressant use) distinguished responders and nonresponders.

The authors noted that these response rates were lower than those seen in the literature and suggested this may be attributable to the multicenter design, more patients with longer-lasting and more refractory disease, and longer times under biologic therapy (median 12 months).

The researchers reported significant improvements in central nervous system, peripheral nervous system, heart, skin, and upper respiratory tract involvements based on declines in Extrapulmonary Physician Organ Severity Tool (ePOST) scores. There were also improvements in the eye, muscle, and lung, but these were not statistically significant.

TNF-antagonist therapy was associated with a high rate of adverse events. Around half of all patients (52%) experienced adverse events, such as pneumonia, urinary tract infections, bacterial sepsis, and herpes zoster. In 31 patients (23%), these led to treatment cessation.

Nine patients also had severe allergic reactions, four had paradoxical granulomatous reactions, three developed neutralizing antibodies against anti-TNF agents, two patients had demyelinating lesions, and one had a serum sickness-like reaction. All of these events led to discontinuation.

Overall, 128 (97%) of the patients in the study had received corticosteroids as first-line therapy, and 125 (95%) had received at least one second-line immunosuppressive drug over a median duration of 16 months. Most were treated with infliximab (91%) as the first-line TNF antagonist, followed by adalimumab (6%), etanercept (2%), and certolizumab pegol (1%).

Treatment with TNF antagonists was associated with significant reductions in corticosteroid use; the mean daily prednisone dose decreased from 23 mg/day to 11 mg/day over the median 20.5-month follow-up. This was seen even in the 33 patients who showed no change in their disease course after TNF-antagonist therapy.

No conflicts of interest were declared.

 

FROM SEMINARS IN ARTHRITIS & RHEUMATISM

Around two-thirds of patients with severe or refractory sarcoidosis show a significant clinical response to tumor necrosis factor (TNF) antagonists, according to findings from a retrospective, multicenter cohort study.

Biologic agents targeting TNF, such as etanercept, infliximab, and adalimumab, have been introduced as a third-line option for patients with disease that is refractory to other treatments. However, Yvan Jamilloux, MD, of the Hospices Civils de Lyon (France) and his coauthors reported that there are still insufficient data available on efficacy and safety of these drugs in the context of sarcoidosis.

Dr. Jamilloux and his colleagues analyzed data from 132 sarcoidosis patients who received TNF antagonists, 122 (92%) of whom had severe sarcoidosis (Semin Arthritis Rheum. 2017 Mar 8. doi: 10.1016/j.semarthrit.2017.03.005).

Overall, 64% of patients showed clinical improvements in response to TNF antagonists; 18% had a complete response, and 46% had a partial response. However, 33 (25%) patients showed no change, and 14 (11%) had continued disease progression despite treatment with TNF antagonists. In another 16 patients who received a second TNF antagonist, 10 (63%) had a complete or partial clinical response. The investigators could find no differences in response between anti-TNF agents or between monotherapy and a combination with an immunosuppressant.

Pulmonary involvement was associated with a significantly lower clinical response, but none of the other factors examined in a multivariate analysis (sex, age, ethnicity, organ involvement, disease duration, steroid dosage, or prior immunosuppressant use) distinguished responders and nonresponders.

The authors noted that these response rates were lower than those seen in the literature and suggested this may be attributable to the multicenter design, more patients with longer-lasting and more refractory disease, and longer times under biologic therapy (median 12 months).

The researchers reported significant improvements in central nervous system, peripheral nervous system, heart, skin, and upper respiratory tract involvements based on declines in Extrapulmonary Physician Organ Severity Tool (ePOST) scores. There were also improvements in the eye, muscle, and lung, but these were not statistically significant.

TNF-antagonist therapy was associated with a high rate of adverse events. Around half of all patients (52%) experienced adverse events, such as pneumonia, urinary tract infections, bacterial sepsis, and herpes zoster. In 31 patients (23%), these led to treatment cessation.

Nine patients also had severe allergic reactions, four had paradoxical granulomatous reactions, three developed neutralizing antibodies against anti-TNF agents, two patients had demyelinating lesions, and one had a serum sickness-like reaction. All of these events led to discontinuation.

Overall, 128 (97%) of the patients in the study had received corticosteroids as first-line therapy, and 125 (95%) had received at least one second-line immunosuppressive drug over a median duration of 16 months. Most were treated with infliximab (91%) as the first-line TNF antagonist, followed by adalimumab (6%), etanercept (2%), and certolizumab pegol (1%).

Treatment with TNF antagonists was associated with significant reductions in corticosteroid use; the mean daily prednisone dose decreased from 23 mg/day to 11 mg/day over the median 20.5-month follow-up. This was seen even in the 33 patients who showed no change in their disease course after TNF-antagonist therapy.

No conflicts of interest were declared.

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Key clinical point: About two-thirds of patients with sarcoidosis refractory to corticosteroids or a second-line immunosuppressive drug show a significant response to anti-TNF drugs.

Major finding: A total of 18% had a complete response, and 46% had a partial response, to TNF antagonists.

Data source: A retrospective, multicenter study in 132 sarcoidosis patients who received TNF antagonists.

Disclosures: No conflicts of interest were declared.

Racial differences in skin cancer risk after organ transplantation

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Nonwhite organ transplant recipients (OTRs) are more likely to present with inflammatory or infectious conditions after transplantation, while white organ recipients more commonly present with malignant disease, new research suggests.

While the high incidence of skin cancers has been well described in patients who undergo solid organ transplants, little is known about the risk factors, incidence, locations, and types of skin disease that occur in nonwhite OTRs, wrote Christina Lee Chung, MD, from Drexel University, Philadelphia, and her coauthors in JAMA Dermatology.

In a retrospective review, the investigators examined the medical records of 412 organ transplant recipients treated at an academic referral center during 2011-2016, of whom 154 were white, 35 were Asian, 33 were Hispanic, and 190 were black (JAMA Dermatology. 2017 Mar 8. doi: 10.1001/jamadermatol.2017.0045).

Among the white patients, malignant or premalignant disease was the most common diagnostic category (67.8%), followed by inflammatory (20.7%) and infectious processes (11.6%). However, among nonwhite organ transplant recipients, inflammatory processes were present in 48.8% of patients, infectious processes in 37.5% and the remaining 13.7% presented with malignant or premalignant lesions.

Black and Hispanic patients were more likely to present with inflammatory or infectious disease; only 8.6% presented with malignant conditions and 16% presented with premalignant disease.

Among the Asian patient population, one-third presented with malignant or premalignant, one-third presented with infectious, and one-third presented with inflammatory conditions.

“Although early detection and treatment of cancer is vital, nonwhite OTRs would also benefit from addressing nonmalignant processes that are exacerbated by immunosuppression,” the authors wrote.

Overall, 389 skin cancers were diagnosed, with squamous cell carcinoma in situ (SCC) the most common type of skin cancer diagnosed in each racial or ethnic group. The mean time between transplant and first skin cancer lesion was 12.67 years in black patients, 6.5 years in Hispanic patients, 6.13 years among white patients, and 3.75 years in Asian patients.

The vast majority of skin cancers (95.1%) were found in white patients. While the majority of lesions in white and Asian patients were found in sun-exposed areas, the few skin cancers seen in black patients were more likely to be found in sun-protected areas, particularly the genitals.

Four of the six genital SCCs tested positive for high-risk human papillomavirus strains – in one Asian patient and three black patients – while the two SCCs found on lower extremities in Hispanic patients tested negative for HPV.

Researchers also looked at skin cancer awareness among the organ transplant recipients using data from initial visit questionnaires. They found that more than 17 of the 22 (77.3%) white organ transplant recipients surveyed were aware their skin cancer risk was increased, compared with 30 of the 44 (68.2%) nonwhite patients.

Similarly, 72.7% of white patients surveyed were aware that sunscreen decreased the risk of cancer, compared with 59.1% of nonwhite patients; 27.3% of white patients reported using a daily sunscreen, compared with 13.6% of nonwhite patients.

“Based on our findings, we suggest that optimal posttransplant dermatologic care be determined based on the race or ethnicity of the patients; however, regardless of skin type or race or ethnicity, a baseline full-skin assessment should be performed in all patients,” the authors wrote.

They proposed that skin cancer follow-up screenings should be given to Asian and Hispanic patients immediately after transplant, but that black organ transplant recipients could delay yearly screenings.

However, they said routine skin checks should begin earlier after transplantation for all nonwhite transplant recipients with a history of, or clinically evident HPV infection.

No conflicts of interest were declared.

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Nonwhite organ transplant recipients (OTRs) are more likely to present with inflammatory or infectious conditions after transplantation, while white organ recipients more commonly present with malignant disease, new research suggests.

While the high incidence of skin cancers has been well described in patients who undergo solid organ transplants, little is known about the risk factors, incidence, locations, and types of skin disease that occur in nonwhite OTRs, wrote Christina Lee Chung, MD, from Drexel University, Philadelphia, and her coauthors in JAMA Dermatology.

In a retrospective review, the investigators examined the medical records of 412 organ transplant recipients treated at an academic referral center during 2011-2016, of whom 154 were white, 35 were Asian, 33 were Hispanic, and 190 were black (JAMA Dermatology. 2017 Mar 8. doi: 10.1001/jamadermatol.2017.0045).

Among the white patients, malignant or premalignant disease was the most common diagnostic category (67.8%), followed by inflammatory (20.7%) and infectious processes (11.6%). However, among nonwhite organ transplant recipients, inflammatory processes were present in 48.8% of patients, infectious processes in 37.5% and the remaining 13.7% presented with malignant or premalignant lesions.

Black and Hispanic patients were more likely to present with inflammatory or infectious disease; only 8.6% presented with malignant conditions and 16% presented with premalignant disease.

Among the Asian patient population, one-third presented with malignant or premalignant, one-third presented with infectious, and one-third presented with inflammatory conditions.

“Although early detection and treatment of cancer is vital, nonwhite OTRs would also benefit from addressing nonmalignant processes that are exacerbated by immunosuppression,” the authors wrote.

Overall, 389 skin cancers were diagnosed, with squamous cell carcinoma in situ (SCC) the most common type of skin cancer diagnosed in each racial or ethnic group. The mean time between transplant and first skin cancer lesion was 12.67 years in black patients, 6.5 years in Hispanic patients, 6.13 years among white patients, and 3.75 years in Asian patients.

The vast majority of skin cancers (95.1%) were found in white patients. While the majority of lesions in white and Asian patients were found in sun-exposed areas, the few skin cancers seen in black patients were more likely to be found in sun-protected areas, particularly the genitals.

Four of the six genital SCCs tested positive for high-risk human papillomavirus strains – in one Asian patient and three black patients – while the two SCCs found on lower extremities in Hispanic patients tested negative for HPV.

Researchers also looked at skin cancer awareness among the organ transplant recipients using data from initial visit questionnaires. They found that more than 17 of the 22 (77.3%) white organ transplant recipients surveyed were aware their skin cancer risk was increased, compared with 30 of the 44 (68.2%) nonwhite patients.

Similarly, 72.7% of white patients surveyed were aware that sunscreen decreased the risk of cancer, compared with 59.1% of nonwhite patients; 27.3% of white patients reported using a daily sunscreen, compared with 13.6% of nonwhite patients.

“Based on our findings, we suggest that optimal posttransplant dermatologic care be determined based on the race or ethnicity of the patients; however, regardless of skin type or race or ethnicity, a baseline full-skin assessment should be performed in all patients,” the authors wrote.

They proposed that skin cancer follow-up screenings should be given to Asian and Hispanic patients immediately after transplant, but that black organ transplant recipients could delay yearly screenings.

However, they said routine skin checks should begin earlier after transplantation for all nonwhite transplant recipients with a history of, or clinically evident HPV infection.

No conflicts of interest were declared.

 

Nonwhite organ transplant recipients (OTRs) are more likely to present with inflammatory or infectious conditions after transplantation, while white organ recipients more commonly present with malignant disease, new research suggests.

While the high incidence of skin cancers has been well described in patients who undergo solid organ transplants, little is known about the risk factors, incidence, locations, and types of skin disease that occur in nonwhite OTRs, wrote Christina Lee Chung, MD, from Drexel University, Philadelphia, and her coauthors in JAMA Dermatology.

In a retrospective review, the investigators examined the medical records of 412 organ transplant recipients treated at an academic referral center during 2011-2016, of whom 154 were white, 35 were Asian, 33 were Hispanic, and 190 were black (JAMA Dermatology. 2017 Mar 8. doi: 10.1001/jamadermatol.2017.0045).

Among the white patients, malignant or premalignant disease was the most common diagnostic category (67.8%), followed by inflammatory (20.7%) and infectious processes (11.6%). However, among nonwhite organ transplant recipients, inflammatory processes were present in 48.8% of patients, infectious processes in 37.5% and the remaining 13.7% presented with malignant or premalignant lesions.

Black and Hispanic patients were more likely to present with inflammatory or infectious disease; only 8.6% presented with malignant conditions and 16% presented with premalignant disease.

Among the Asian patient population, one-third presented with malignant or premalignant, one-third presented with infectious, and one-third presented with inflammatory conditions.

“Although early detection and treatment of cancer is vital, nonwhite OTRs would also benefit from addressing nonmalignant processes that are exacerbated by immunosuppression,” the authors wrote.

Overall, 389 skin cancers were diagnosed, with squamous cell carcinoma in situ (SCC) the most common type of skin cancer diagnosed in each racial or ethnic group. The mean time between transplant and first skin cancer lesion was 12.67 years in black patients, 6.5 years in Hispanic patients, 6.13 years among white patients, and 3.75 years in Asian patients.

The vast majority of skin cancers (95.1%) were found in white patients. While the majority of lesions in white and Asian patients were found in sun-exposed areas, the few skin cancers seen in black patients were more likely to be found in sun-protected areas, particularly the genitals.

Four of the six genital SCCs tested positive for high-risk human papillomavirus strains – in one Asian patient and three black patients – while the two SCCs found on lower extremities in Hispanic patients tested negative for HPV.

Researchers also looked at skin cancer awareness among the organ transplant recipients using data from initial visit questionnaires. They found that more than 17 of the 22 (77.3%) white organ transplant recipients surveyed were aware their skin cancer risk was increased, compared with 30 of the 44 (68.2%) nonwhite patients.

Similarly, 72.7% of white patients surveyed were aware that sunscreen decreased the risk of cancer, compared with 59.1% of nonwhite patients; 27.3% of white patients reported using a daily sunscreen, compared with 13.6% of nonwhite patients.

“Based on our findings, we suggest that optimal posttransplant dermatologic care be determined based on the race or ethnicity of the patients; however, regardless of skin type or race or ethnicity, a baseline full-skin assessment should be performed in all patients,” the authors wrote.

They proposed that skin cancer follow-up screenings should be given to Asian and Hispanic patients immediately after transplant, but that black organ transplant recipients could delay yearly screenings.

However, they said routine skin checks should begin earlier after transplantation for all nonwhite transplant recipients with a history of, or clinically evident HPV infection.

No conflicts of interest were declared.

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Key clinical point: Nonwhite organ transplant recipients are more likely than are white recipients to present with inflammatory or infectious conditions than with skin cancer after transplantation.

Major finding: Malignant or premalignant disease was seen in 67.8% of white organ transplant recipients but just 13.7% of nonwhite recipients.

Data source: A retrospective review of medical records from 412 organ transplant recipients.

Disclosures: No conflicts of interest were declared.

Inclisiran shows sustained and significant declines in LDL-C

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The small interfering RNA molecule inclisiran has achieved significant reductions in LDL-cholesterol levels in patients at high cardiovascular risk, according to data presented at the annual meeting of the American College of Cardiology and published simultaneously in the New England Journal of Medicine.

Inclisiran is an investigational, chemically synthesized, small interfering RNA molecule that targets the liver-derived serine protease proprotein convertase subtilisin-kexin type 9 (PCSK9), which promotes degradation of the LDL-receptor.

An earlier phase I trial of inclisiran in healthy volunteers showed sustained reductions in LDL-C levels. In this phase II randomized, placebo-controlled trial, researchers tested a single dose (200, 300, or 500 mg) or double dose (100, 200, or 300 mg on day 1 and day 90) subcutaneous injection, compared with placebo in 501 patients with elevated LDL-C who were at high risk for cardiovascular disease.

By day 30 after the first injection, mean reductions in LDL-C ranged from 44.5% to 50.5% below baseline levels across all dosages of inclisiran (N Engl J Med. 2017 Mar 17. doi: 10.1056/NEJMoa1615758).

By day 180, the mean reductions in LDL-C were 27.9%-41.9% in patients who received a single dose and 35.5%-52.6% in those who received a double dose. By comparison, patients who received placebo showed a 2.1% mean increase in LDL-C.

All patients who received two 300-mg doses of inclisiran showed reductions in LDL-C by day 180, with a mean absolute change of –64.2 ± 20.7 mg/dL. More than half of this group (54%) achieved LDL-C reductions of 50% or more below baseline.

In this same dosage group, 5% of patients achieved LDL-C levels below 25 mg/dL, 48% achieved levels below 50 mg/dL, and 66% achieved levels below 70 mg/dL by day 180 after the first dose.

Researchers observed a nadir in patients’ response at around day 60 for the single-dose group and day 140 for the double-dose group.

“Maintaining consistent and effective reductions in LDL-cholesterol levels in the long term through the use of statins is, in part, hindered by adherence,” wrote Kausik K. Ray, MD, of Imperial College London, and his coauthors. “Hence, approaches that not only lower LDL-cholesterol levels safely but also can maintain reduction consistently over time, when applied either in lieu of or simultaneously with statin therapy, are being sought.”

However, they also clarified that it was still unknown as to whether these LDL-C reductions would translate into reductions in cardiovascular events.

Researchers also saw significant declines in PCSK9 levels from baseline in all patients who received inclisiran. Among those who received a single dose, mean reductions in PCSK9 at 180 days ranged from 47.9% to 59.3%, while those who received a double dose showed mean reductions ranging from 53.2% to 69.1%.

The overall rate of adverse events was similar between the inclisiran and placebo groups (76% vs. 79%, respectively), while the rate of serious adverse events was 11% in the inclisiran group and 8% in the placebo group.

One patient in the inclisiran group and one in the placebo group showed increased levels of hepatic aspartate aminotransferase, and three patients in the inclisiran group had elevated alanine aminotransferase. No increases in bilirubin levels were observed.

“Symptoms of immune activation, which is often a concern with therapies targeting RNA, were rare in association with inclisiran: There were few instances of flu-like symptoms and no observed elevations in C-reactive protein,” the authors wrote.

Injection-site reactions were also relatively uncommon, occurring in 4% of patients treated with one dose of inclisiran, 7% of those treated with two doses, and none of the placebo group.

The authors, however, said that, given the small size and relatively short duration of the trial, they could not rule out the possibility of other infrequent serious side effects. A long-term open-label study also is being conducted.

The study was funded by the Medicines Company. Several authors declared receiving personal fees from pharmaceutical companies outside the submitted work and 10 also declared receiving personal fees from the Medicines Company, 6 involving the submitted work. One author was an employee of the Medicines Company with stock options.

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The small interfering RNA molecule inclisiran has achieved significant reductions in LDL-cholesterol levels in patients at high cardiovascular risk, according to data presented at the annual meeting of the American College of Cardiology and published simultaneously in the New England Journal of Medicine.

Inclisiran is an investigational, chemically synthesized, small interfering RNA molecule that targets the liver-derived serine protease proprotein convertase subtilisin-kexin type 9 (PCSK9), which promotes degradation of the LDL-receptor.

An earlier phase I trial of inclisiran in healthy volunteers showed sustained reductions in LDL-C levels. In this phase II randomized, placebo-controlled trial, researchers tested a single dose (200, 300, or 500 mg) or double dose (100, 200, or 300 mg on day 1 and day 90) subcutaneous injection, compared with placebo in 501 patients with elevated LDL-C who were at high risk for cardiovascular disease.

By day 30 after the first injection, mean reductions in LDL-C ranged from 44.5% to 50.5% below baseline levels across all dosages of inclisiran (N Engl J Med. 2017 Mar 17. doi: 10.1056/NEJMoa1615758).

By day 180, the mean reductions in LDL-C were 27.9%-41.9% in patients who received a single dose and 35.5%-52.6% in those who received a double dose. By comparison, patients who received placebo showed a 2.1% mean increase in LDL-C.

All patients who received two 300-mg doses of inclisiran showed reductions in LDL-C by day 180, with a mean absolute change of –64.2 ± 20.7 mg/dL. More than half of this group (54%) achieved LDL-C reductions of 50% or more below baseline.

In this same dosage group, 5% of patients achieved LDL-C levels below 25 mg/dL, 48% achieved levels below 50 mg/dL, and 66% achieved levels below 70 mg/dL by day 180 after the first dose.

Researchers observed a nadir in patients’ response at around day 60 for the single-dose group and day 140 for the double-dose group.

“Maintaining consistent and effective reductions in LDL-cholesterol levels in the long term through the use of statins is, in part, hindered by adherence,” wrote Kausik K. Ray, MD, of Imperial College London, and his coauthors. “Hence, approaches that not only lower LDL-cholesterol levels safely but also can maintain reduction consistently over time, when applied either in lieu of or simultaneously with statin therapy, are being sought.”

However, they also clarified that it was still unknown as to whether these LDL-C reductions would translate into reductions in cardiovascular events.

Researchers also saw significant declines in PCSK9 levels from baseline in all patients who received inclisiran. Among those who received a single dose, mean reductions in PCSK9 at 180 days ranged from 47.9% to 59.3%, while those who received a double dose showed mean reductions ranging from 53.2% to 69.1%.

The overall rate of adverse events was similar between the inclisiran and placebo groups (76% vs. 79%, respectively), while the rate of serious adverse events was 11% in the inclisiran group and 8% in the placebo group.

One patient in the inclisiran group and one in the placebo group showed increased levels of hepatic aspartate aminotransferase, and three patients in the inclisiran group had elevated alanine aminotransferase. No increases in bilirubin levels were observed.

“Symptoms of immune activation, which is often a concern with therapies targeting RNA, were rare in association with inclisiran: There were few instances of flu-like symptoms and no observed elevations in C-reactive protein,” the authors wrote.

Injection-site reactions were also relatively uncommon, occurring in 4% of patients treated with one dose of inclisiran, 7% of those treated with two doses, and none of the placebo group.

The authors, however, said that, given the small size and relatively short duration of the trial, they could not rule out the possibility of other infrequent serious side effects. A long-term open-label study also is being conducted.

The study was funded by the Medicines Company. Several authors declared receiving personal fees from pharmaceutical companies outside the submitted work and 10 also declared receiving personal fees from the Medicines Company, 6 involving the submitted work. One author was an employee of the Medicines Company with stock options.

 

The small interfering RNA molecule inclisiran has achieved significant reductions in LDL-cholesterol levels in patients at high cardiovascular risk, according to data presented at the annual meeting of the American College of Cardiology and published simultaneously in the New England Journal of Medicine.

Inclisiran is an investigational, chemically synthesized, small interfering RNA molecule that targets the liver-derived serine protease proprotein convertase subtilisin-kexin type 9 (PCSK9), which promotes degradation of the LDL-receptor.

An earlier phase I trial of inclisiran in healthy volunteers showed sustained reductions in LDL-C levels. In this phase II randomized, placebo-controlled trial, researchers tested a single dose (200, 300, or 500 mg) or double dose (100, 200, or 300 mg on day 1 and day 90) subcutaneous injection, compared with placebo in 501 patients with elevated LDL-C who were at high risk for cardiovascular disease.

By day 30 after the first injection, mean reductions in LDL-C ranged from 44.5% to 50.5% below baseline levels across all dosages of inclisiran (N Engl J Med. 2017 Mar 17. doi: 10.1056/NEJMoa1615758).

By day 180, the mean reductions in LDL-C were 27.9%-41.9% in patients who received a single dose and 35.5%-52.6% in those who received a double dose. By comparison, patients who received placebo showed a 2.1% mean increase in LDL-C.

All patients who received two 300-mg doses of inclisiran showed reductions in LDL-C by day 180, with a mean absolute change of –64.2 ± 20.7 mg/dL. More than half of this group (54%) achieved LDL-C reductions of 50% or more below baseline.

In this same dosage group, 5% of patients achieved LDL-C levels below 25 mg/dL, 48% achieved levels below 50 mg/dL, and 66% achieved levels below 70 mg/dL by day 180 after the first dose.

Researchers observed a nadir in patients’ response at around day 60 for the single-dose group and day 140 for the double-dose group.

“Maintaining consistent and effective reductions in LDL-cholesterol levels in the long term through the use of statins is, in part, hindered by adherence,” wrote Kausik K. Ray, MD, of Imperial College London, and his coauthors. “Hence, approaches that not only lower LDL-cholesterol levels safely but also can maintain reduction consistently over time, when applied either in lieu of or simultaneously with statin therapy, are being sought.”

However, they also clarified that it was still unknown as to whether these LDL-C reductions would translate into reductions in cardiovascular events.

Researchers also saw significant declines in PCSK9 levels from baseline in all patients who received inclisiran. Among those who received a single dose, mean reductions in PCSK9 at 180 days ranged from 47.9% to 59.3%, while those who received a double dose showed mean reductions ranging from 53.2% to 69.1%.

The overall rate of adverse events was similar between the inclisiran and placebo groups (76% vs. 79%, respectively), while the rate of serious adverse events was 11% in the inclisiran group and 8% in the placebo group.

One patient in the inclisiran group and one in the placebo group showed increased levels of hepatic aspartate aminotransferase, and three patients in the inclisiran group had elevated alanine aminotransferase. No increases in bilirubin levels were observed.

“Symptoms of immune activation, which is often a concern with therapies targeting RNA, were rare in association with inclisiran: There were few instances of flu-like symptoms and no observed elevations in C-reactive protein,” the authors wrote.

Injection-site reactions were also relatively uncommon, occurring in 4% of patients treated with one dose of inclisiran, 7% of those treated with two doses, and none of the placebo group.

The authors, however, said that, given the small size and relatively short duration of the trial, they could not rule out the possibility of other infrequent serious side effects. A long-term open-label study also is being conducted.

The study was funded by the Medicines Company. Several authors declared receiving personal fees from pharmaceutical companies outside the submitted work and 10 also declared receiving personal fees from the Medicines Company, 6 involving the submitted work. One author was an employee of the Medicines Company with stock options.

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Key clinical point: Investigational small interfering RNA molecule inclisiran has shown significant reductions in LDL-cholesterol levels in patients at high cardiovascular risk.

Major finding: A double dose of inclisiran was associated with a 35.5%-52.6% decline in LDL-cholesterol levels from baseline by day 180 after the first injection.

Data source: The phase II ORION-1 trial in 501 patients with elevated LDL-C and high risk for cardiovascular disease.

Disclosures: The study was funded by the Medicines Company. Several authors declared receiving personal fees from pharmaceutical companies outside the submitted work and 10 also declared personal fees from the Medicines Company, 6 involving the submitted work. One author was an employee of the Medicines Company with stock options.

Genetic variant linked to more antibiotics, corticosteroid use in COPD

Polymorphisms in COPD show disease’s complexity
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A genetic variant associated with a poorer therapeutic response in patients with asthma may also be linked to more severe chronic obstructive pulmonary disease, researchers have found.

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Dr. Vera De Palo
Vera De Palo, MD, FCCP, comments: This study demonstrates that the pulmonologist’s “bread and butter” disease, COPD, continues to be very complex and gives us an understanding of why patients may not respond as we expect them to.
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Dr. Vera De Palo
Vera De Palo, MD, FCCP, comments: This study demonstrates that the pulmonologist’s “bread and butter” disease, COPD, continues to be very complex and gives us an understanding of why patients may not respond as we expect them to.
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Dr. Vera De Palo
Vera De Palo, MD, FCCP, comments: This study demonstrates that the pulmonologist’s “bread and butter” disease, COPD, continues to be very complex and gives us an understanding of why patients may not respond as we expect them to.
Title
Polymorphisms in COPD show disease’s complexity
Polymorphisms in COPD show disease’s complexity

 

A genetic variant associated with a poorer therapeutic response in patients with asthma may also be linked to more severe chronic obstructive pulmonary disease, researchers have found.

 

A genetic variant associated with a poorer therapeutic response in patients with asthma may also be linked to more severe chronic obstructive pulmonary disease, researchers have found.

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Key clinical point: A genetic variation linked to poorer disease control in asthma is associated with a similar effect in patients with chronic obstructive pulmonary disease.

Major finding: Patients with COPD who are Arg/Arg homozygotes at codon 16 of the beta-2-adrenoreceptor gene were significantly more likely to require two or more courses of antibiotic therapy and more systemic corticosteroid therapy than were patients with other polymorphisms.

Data source: A retrospective cohort study of 92 patients with stable grade COPD.

Disclosures: The Ministry of Science and Education supported the study. No conflicts of interest were declared.

Hospitals rarely offer cessation therapy to smokers with MI

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Less than one-third of smokers hospitalized for myocardial infarction receive any kind of smoking cessation therapy during their stay in hospital, according to a poster presented at the annual meeting of the American College of Cardiology.

“Inpatient smoking cessation therapy coupled with outpatient follow-up can significantly improve long-term smoking cessation rates, but little is known about how often smoking cessation therapies are used among hospitalized patients,” wrote Dr Quinn R. Pack and coauthors from the Baystate Medical Center in Springfield, and Massachusetts General Hospital.

Brett Mulcahy/ThinkStock
Researchers analyzed billing data and ICD-9 codes for 36,675 current smokers hospitalized for MI at 282 hospitals in 2014, and found that overall only 29.9% of these individuals were given at least one kind of smoking cessation therapy, such as varenicline, bupropion, and nicotine replacement gums, patches, lozenges, and inhalers.

The nicotine patch was the most common therapy; 20.4% of patients received it with an average daily dose of 19.8 mg, while 2.2% of patients received bupropion, 0.4% received varenicline, 0.3% received nicotine gum, 0.2% received nicotine inhaler therapy, and just 0.04% received nicotine lozenge therapy. Nearly one in ten patients received professional counseling (9.6%).

Smoking cessation was more commonly given to patients with lung disease, depression, alcohol use or who were younger but the researchers noted significant variations in the use of smoking cessation therapies across hospitals. While the median treatment rate was 26.2%, it ranged from as low as 11.4% to a high of 51.1%.

Given the variation across hospitals, the authors said they plan to identify the strategies and practices that the high-performing hospitals use to provide smoking cessation therapies.

“Smoking cessation is the single most effective behavior change that patients can make after a hospitalization for coronary heart disease to prevent recurrent events,” the authors wrote. “Given that hospitalization is usually a teachable moment with high patient motivation to quit smoking, there appears to be a large opportunity for improvement in the care of smokers hospitalized with CHD.”

The authors noted that their data were limited to smoking cessation options provided during hospitalization; the researchers said they did not assess whether those therapies helped MI patients quit smoking, or whether there were patients who declined the therapies offered.

No conflict of interest disclosures were provided with the data.

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Less than one-third of smokers hospitalized for myocardial infarction receive any kind of smoking cessation therapy during their stay in hospital, according to a poster presented at the annual meeting of the American College of Cardiology.

“Inpatient smoking cessation therapy coupled with outpatient follow-up can significantly improve long-term smoking cessation rates, but little is known about how often smoking cessation therapies are used among hospitalized patients,” wrote Dr Quinn R. Pack and coauthors from the Baystate Medical Center in Springfield, and Massachusetts General Hospital.

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Researchers analyzed billing data and ICD-9 codes for 36,675 current smokers hospitalized for MI at 282 hospitals in 2014, and found that overall only 29.9% of these individuals were given at least one kind of smoking cessation therapy, such as varenicline, bupropion, and nicotine replacement gums, patches, lozenges, and inhalers.

The nicotine patch was the most common therapy; 20.4% of patients received it with an average daily dose of 19.8 mg, while 2.2% of patients received bupropion, 0.4% received varenicline, 0.3% received nicotine gum, 0.2% received nicotine inhaler therapy, and just 0.04% received nicotine lozenge therapy. Nearly one in ten patients received professional counseling (9.6%).

Smoking cessation was more commonly given to patients with lung disease, depression, alcohol use or who were younger but the researchers noted significant variations in the use of smoking cessation therapies across hospitals. While the median treatment rate was 26.2%, it ranged from as low as 11.4% to a high of 51.1%.

Given the variation across hospitals, the authors said they plan to identify the strategies and practices that the high-performing hospitals use to provide smoking cessation therapies.

“Smoking cessation is the single most effective behavior change that patients can make after a hospitalization for coronary heart disease to prevent recurrent events,” the authors wrote. “Given that hospitalization is usually a teachable moment with high patient motivation to quit smoking, there appears to be a large opportunity for improvement in the care of smokers hospitalized with CHD.”

The authors noted that their data were limited to smoking cessation options provided during hospitalization; the researchers said they did not assess whether those therapies helped MI patients quit smoking, or whether there were patients who declined the therapies offered.

No conflict of interest disclosures were provided with the data.

Less than one-third of smokers hospitalized for myocardial infarction receive any kind of smoking cessation therapy during their stay in hospital, according to a poster presented at the annual meeting of the American College of Cardiology.

“Inpatient smoking cessation therapy coupled with outpatient follow-up can significantly improve long-term smoking cessation rates, but little is known about how often smoking cessation therapies are used among hospitalized patients,” wrote Dr Quinn R. Pack and coauthors from the Baystate Medical Center in Springfield, and Massachusetts General Hospital.

Brett Mulcahy/ThinkStock
Researchers analyzed billing data and ICD-9 codes for 36,675 current smokers hospitalized for MI at 282 hospitals in 2014, and found that overall only 29.9% of these individuals were given at least one kind of smoking cessation therapy, such as varenicline, bupropion, and nicotine replacement gums, patches, lozenges, and inhalers.

The nicotine patch was the most common therapy; 20.4% of patients received it with an average daily dose of 19.8 mg, while 2.2% of patients received bupropion, 0.4% received varenicline, 0.3% received nicotine gum, 0.2% received nicotine inhaler therapy, and just 0.04% received nicotine lozenge therapy. Nearly one in ten patients received professional counseling (9.6%).

Smoking cessation was more commonly given to patients with lung disease, depression, alcohol use or who were younger but the researchers noted significant variations in the use of smoking cessation therapies across hospitals. While the median treatment rate was 26.2%, it ranged from as low as 11.4% to a high of 51.1%.

Given the variation across hospitals, the authors said they plan to identify the strategies and practices that the high-performing hospitals use to provide smoking cessation therapies.

“Smoking cessation is the single most effective behavior change that patients can make after a hospitalization for coronary heart disease to prevent recurrent events,” the authors wrote. “Given that hospitalization is usually a teachable moment with high patient motivation to quit smoking, there appears to be a large opportunity for improvement in the care of smokers hospitalized with CHD.”

The authors noted that their data were limited to smoking cessation options provided during hospitalization; the researchers said they did not assess whether those therapies helped MI patients quit smoking, or whether there were patients who declined the therapies offered.

No conflict of interest disclosures were provided with the data.

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Key clinical point: Less than one-third of smokers hospitalized for myocardial infarction receive any kind of smoking cessation therapy during their stay in hospital.

Major finding: Only 29.9% of current smokers hospitalized for MI were given at least one kind of smoking cessation therapy while in hospital.

Data source: Cohort study of billing and ICD-9 data from 36,675 current smokers hospitalized for myocardial infarction.

Disclosures: No disclosures were available.

HT use associated with lower postmenopausal coronary artery calcium scores

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Hormone replacement therapy is associated with lower coronary artery calcium scores in postmenopausal women, and may also be linked to lower all-cause mortality, according to data presented in a poster at the annual meeting of the American College of Cardiology.

In a retrospective cohort study involving 4,286 consecutive asymptomatic postmenopausal women undergoing coronary artery calcium scanning between 1998 and 2012, researchers sought to clarify the controversial cardioprotective effects of hormone replacement therapy (HT).

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The average coronary artery calcium score was significantly lower in the 41% of women who were taking HT compared to those who weren’t (72.1 ± 223 vs. 3,119.2 plus or minus 322.2; P less than .001). This difference remained significant for each 5-year age group, reported Yoav Arnson, MD, of Cedars-Sinai Medical Center in Los Angeles, and coauthors.

After adjustment for age and cardiac risk factors, HT was associated with a higher prevalence of women with calcium artery score of 0, and a lower prevalence of women with calcium artery scores above 399.

Women taking HT also had significantly lower mortality compared with those not on HT (5.8% vs. 6.8%). After adjustment for age, cardiac risk factors, and coronary artery calcium scores, women on HT had a 30% lower long-term mortality (95% confidence interval, 0.49-0.98; P = .043).

“Hormone replacement therapy resulted in lower atherosclerosis and improved survival for all age groups and for all levels of coronary calcium,” Dr. Arnson said in a statement. “From this we do think it is beneficial, but we would need prospective or randomized studies to determine which groups might not benefit or even be harmed by this therapy.”

The women in the HT group were significantly younger than those not taking HT, and had a lower prevalence of hypertension and diabetes as well as higher average HDL cholesterol than women not taking HT.

Researchers saw a clear decrease in the number of women taking HT over the course of the study, declining from a peak of 60% in 1998 to 23% of women in 2012.

No disclosures were available.

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Hormone replacement therapy is associated with lower coronary artery calcium scores in postmenopausal women, and may also be linked to lower all-cause mortality, according to data presented in a poster at the annual meeting of the American College of Cardiology.

In a retrospective cohort study involving 4,286 consecutive asymptomatic postmenopausal women undergoing coronary artery calcium scanning between 1998 and 2012, researchers sought to clarify the controversial cardioprotective effects of hormone replacement therapy (HT).

Judith Flacke/Thinkstock
The average coronary artery calcium score was significantly lower in the 41% of women who were taking HT compared to those who weren’t (72.1 ± 223 vs. 3,119.2 plus or minus 322.2; P less than .001). This difference remained significant for each 5-year age group, reported Yoav Arnson, MD, of Cedars-Sinai Medical Center in Los Angeles, and coauthors.

After adjustment for age and cardiac risk factors, HT was associated with a higher prevalence of women with calcium artery score of 0, and a lower prevalence of women with calcium artery scores above 399.

Women taking HT also had significantly lower mortality compared with those not on HT (5.8% vs. 6.8%). After adjustment for age, cardiac risk factors, and coronary artery calcium scores, women on HT had a 30% lower long-term mortality (95% confidence interval, 0.49-0.98; P = .043).

“Hormone replacement therapy resulted in lower atherosclerosis and improved survival for all age groups and for all levels of coronary calcium,” Dr. Arnson said in a statement. “From this we do think it is beneficial, but we would need prospective or randomized studies to determine which groups might not benefit or even be harmed by this therapy.”

The women in the HT group were significantly younger than those not taking HT, and had a lower prevalence of hypertension and diabetes as well as higher average HDL cholesterol than women not taking HT.

Researchers saw a clear decrease in the number of women taking HT over the course of the study, declining from a peak of 60% in 1998 to 23% of women in 2012.

No disclosures were available.

Hormone replacement therapy is associated with lower coronary artery calcium scores in postmenopausal women, and may also be linked to lower all-cause mortality, according to data presented in a poster at the annual meeting of the American College of Cardiology.

In a retrospective cohort study involving 4,286 consecutive asymptomatic postmenopausal women undergoing coronary artery calcium scanning between 1998 and 2012, researchers sought to clarify the controversial cardioprotective effects of hormone replacement therapy (HT).

Judith Flacke/Thinkstock
The average coronary artery calcium score was significantly lower in the 41% of women who were taking HT compared to those who weren’t (72.1 ± 223 vs. 3,119.2 plus or minus 322.2; P less than .001). This difference remained significant for each 5-year age group, reported Yoav Arnson, MD, of Cedars-Sinai Medical Center in Los Angeles, and coauthors.

After adjustment for age and cardiac risk factors, HT was associated with a higher prevalence of women with calcium artery score of 0, and a lower prevalence of women with calcium artery scores above 399.

Women taking HT also had significantly lower mortality compared with those not on HT (5.8% vs. 6.8%). After adjustment for age, cardiac risk factors, and coronary artery calcium scores, women on HT had a 30% lower long-term mortality (95% confidence interval, 0.49-0.98; P = .043).

“Hormone replacement therapy resulted in lower atherosclerosis and improved survival for all age groups and for all levels of coronary calcium,” Dr. Arnson said in a statement. “From this we do think it is beneficial, but we would need prospective or randomized studies to determine which groups might not benefit or even be harmed by this therapy.”

The women in the HT group were significantly younger than those not taking HT, and had a lower prevalence of hypertension and diabetes as well as higher average HDL cholesterol than women not taking HT.

Researchers saw a clear decrease in the number of women taking HT over the course of the study, declining from a peak of 60% in 1998 to 23% of women in 2012.

No disclosures were available.

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Key clinical point: Hormone replacement therapy is associated with lower coronary artery calcium scores in postmenopausal women and may also be linked to lower all-cause mortality.

Major finding: The average coronary artery calcium score was significantly lower in the 41% of women who were taking HT compared to those who weren’t (72.1 vs. 3,119.2 ).

Data source: Retrospective cohort study of 4,286 postmenopausal women undergoing coronary artery calcium scanning.

Disclosures: No disclosures were available.

No benefit from adjuvant sunitinib or sorafenib for clear cell renal cancer

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Adjuvant sunitinib or sorafenib show no significant advantages in disease-free or overall survival over placebo in patients with high-risk clear cell renal cancer, according to secondary analysis of data from the ASSURE trial.

The primary analysis of data from the ASSURE trial, which included patients with all types of renal cell carcinoma, had failed to show a benefit in disease-free survival.

“Given recently published results of a 750-patient randomized trial, S-TRAC, (sunitinib 50 mg daily [4/2 schedule] vs placebo in clear cell predominant pT3-4 or node-positive disease) that show improved [disease-free survival], the appropriate adjuvant strategy for high-risk patients is unclear,” Naomi B. Haas, MD, and coauthors wrote (JAMA Oncol. 2017 Mar 9. doi: 10.1001/jamaoncol.2017.0076).

Therefore, the investigators focused on a subset of patients from the ASSURE trial with high-risk clear cell renal cancer to determine if there might be a benefit in this group.

The secondary analysis involved 1,069 participants with pT3 and higher or node-positive renal cancer with clear cell histology who were randomized to receive 54 weeks of sunitinib (50mg, oral daily for 28 of 42 days per cycle), sorafenib (400 mg, oral twice daily continuously), or placebo.

The 5-year disease-free survival rate was 47.7% for patients in the sunitinib arm, 49.9% for those taking sorafenib, and 50% for placebo, with no statistically significant difference between the three groups. The 5-year overall survival rate was 75.2% for the sunitinib arm, 80.2% in the sorafenib arm, and 76.5% for those on placebo, with no statistically significant differences between the groups, reported Dr. Haas of the Abramson Cancer Center of the University of Pennsylvania, Philadelphia, and colleagues.

“This high-risk population had a better 5-year recurrence-free rate (around 50%) than expected (41.9% for high-risk disease and 36.0% for node-positive disease), possibly a result of better surgical technique, more accurate staging, or unknown biologic factors,” the authors wrote.

When the researchers analyzed disease-free survival according to quartiles of total dose per 6-week cycle, they also found no differences between each quartile of average dose per cycle.

There was, however, a significantly higher rate of grade 3 or higher adverse events in the sunitinib arm (66%) and sorafenib group (72%), compared with placebo (22%).

“Based on this analysis, a rationale for adjuvant therapy in this high-risk population is not elucidated,” Dr. Haas and colleagues said.

The study was coordinated by the ECOG-ACRIN Cancer Research Group and supported by Public Health Service grants, the National Cancer Institute, National Institutes of Health, and the Department of Health & Human Services. The drugs and placebos were provided by Bayer and Pfizer through the National Cancer Institute.

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Adjuvant sunitinib or sorafenib show no significant advantages in disease-free or overall survival over placebo in patients with high-risk clear cell renal cancer, according to secondary analysis of data from the ASSURE trial.

The primary analysis of data from the ASSURE trial, which included patients with all types of renal cell carcinoma, had failed to show a benefit in disease-free survival.

“Given recently published results of a 750-patient randomized trial, S-TRAC, (sunitinib 50 mg daily [4/2 schedule] vs placebo in clear cell predominant pT3-4 or node-positive disease) that show improved [disease-free survival], the appropriate adjuvant strategy for high-risk patients is unclear,” Naomi B. Haas, MD, and coauthors wrote (JAMA Oncol. 2017 Mar 9. doi: 10.1001/jamaoncol.2017.0076).

Therefore, the investigators focused on a subset of patients from the ASSURE trial with high-risk clear cell renal cancer to determine if there might be a benefit in this group.

The secondary analysis involved 1,069 participants with pT3 and higher or node-positive renal cancer with clear cell histology who were randomized to receive 54 weeks of sunitinib (50mg, oral daily for 28 of 42 days per cycle), sorafenib (400 mg, oral twice daily continuously), or placebo.

The 5-year disease-free survival rate was 47.7% for patients in the sunitinib arm, 49.9% for those taking sorafenib, and 50% for placebo, with no statistically significant difference between the three groups. The 5-year overall survival rate was 75.2% for the sunitinib arm, 80.2% in the sorafenib arm, and 76.5% for those on placebo, with no statistically significant differences between the groups, reported Dr. Haas of the Abramson Cancer Center of the University of Pennsylvania, Philadelphia, and colleagues.

“This high-risk population had a better 5-year recurrence-free rate (around 50%) than expected (41.9% for high-risk disease and 36.0% for node-positive disease), possibly a result of better surgical technique, more accurate staging, or unknown biologic factors,” the authors wrote.

When the researchers analyzed disease-free survival according to quartiles of total dose per 6-week cycle, they also found no differences between each quartile of average dose per cycle.

There was, however, a significantly higher rate of grade 3 or higher adverse events in the sunitinib arm (66%) and sorafenib group (72%), compared with placebo (22%).

“Based on this analysis, a rationale for adjuvant therapy in this high-risk population is not elucidated,” Dr. Haas and colleagues said.

The study was coordinated by the ECOG-ACRIN Cancer Research Group and supported by Public Health Service grants, the National Cancer Institute, National Institutes of Health, and the Department of Health & Human Services. The drugs and placebos were provided by Bayer and Pfizer through the National Cancer Institute.

 

Adjuvant sunitinib or sorafenib show no significant advantages in disease-free or overall survival over placebo in patients with high-risk clear cell renal cancer, according to secondary analysis of data from the ASSURE trial.

The primary analysis of data from the ASSURE trial, which included patients with all types of renal cell carcinoma, had failed to show a benefit in disease-free survival.

“Given recently published results of a 750-patient randomized trial, S-TRAC, (sunitinib 50 mg daily [4/2 schedule] vs placebo in clear cell predominant pT3-4 or node-positive disease) that show improved [disease-free survival], the appropriate adjuvant strategy for high-risk patients is unclear,” Naomi B. Haas, MD, and coauthors wrote (JAMA Oncol. 2017 Mar 9. doi: 10.1001/jamaoncol.2017.0076).

Therefore, the investigators focused on a subset of patients from the ASSURE trial with high-risk clear cell renal cancer to determine if there might be a benefit in this group.

The secondary analysis involved 1,069 participants with pT3 and higher or node-positive renal cancer with clear cell histology who were randomized to receive 54 weeks of sunitinib (50mg, oral daily for 28 of 42 days per cycle), sorafenib (400 mg, oral twice daily continuously), or placebo.

The 5-year disease-free survival rate was 47.7% for patients in the sunitinib arm, 49.9% for those taking sorafenib, and 50% for placebo, with no statistically significant difference between the three groups. The 5-year overall survival rate was 75.2% for the sunitinib arm, 80.2% in the sorafenib arm, and 76.5% for those on placebo, with no statistically significant differences between the groups, reported Dr. Haas of the Abramson Cancer Center of the University of Pennsylvania, Philadelphia, and colleagues.

“This high-risk population had a better 5-year recurrence-free rate (around 50%) than expected (41.9% for high-risk disease and 36.0% for node-positive disease), possibly a result of better surgical technique, more accurate staging, or unknown biologic factors,” the authors wrote.

When the researchers analyzed disease-free survival according to quartiles of total dose per 6-week cycle, they also found no differences between each quartile of average dose per cycle.

There was, however, a significantly higher rate of grade 3 or higher adverse events in the sunitinib arm (66%) and sorafenib group (72%), compared with placebo (22%).

“Based on this analysis, a rationale for adjuvant therapy in this high-risk population is not elucidated,” Dr. Haas and colleagues said.

The study was coordinated by the ECOG-ACRIN Cancer Research Group and supported by Public Health Service grants, the National Cancer Institute, National Institutes of Health, and the Department of Health & Human Services. The drugs and placebos were provided by Bayer and Pfizer through the National Cancer Institute.

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Key clinical point: Adjuvant sunitinib or sorafenib show no significant benefit in disease-free or overall survival in patients with high-risk clear cell renal cancer.

Major finding: The 5-year disease-free survival rate was 47.7% for patients treated with sunitinib, 49.9% for those treated with sorafenib, and 50% for those given placebo.

Data source: Secondary analysis of data from the ASSURE trial in 1,943 patients with pT3 and higher or node-positive renal cancer with clear cell histology.

Disclosures: The study was coordinated by the ECOG-ACRIN Cancer Research Group and supported by Public Health Service grants, the National Cancer Institute, National Institutes of Health, and the Department of Health & Human Services. The drugs and placebos were provided by Bayer and Pfizer through the National Cancer Institute.