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Clinical Comestibles?
1. This 45-year-old woman’s skin has multiple small, soft, compressible papules, one of which is apt to bleed when scratched. She is concerned that these “cherry red” lesions are precancerous.
Diagnosis: Cherry angiomas, also known as de Morgan spots, are extremely common lesions; though usually asymptomatic, they may bleed with trauma. They occur most commonly as multiple asymptomatic lesions on the trunk and arms. These capillary hemangiomas are dome-shaped, small (0.1 to 0.5 cm in diameter), and bright red to violaceous; they can be flat, raised, or nodular.
Cherry angiomas form as a result of the development of multiple capillaries with narrow lumens and prominent endothelial cells arranged in a lobular pattern in the papillary dermis. Effective treatment options include curettage, laser ablation, and electrodesiccation.
For more information, see Kim, J-H, Park H-Y, Ahn SK. Cherry Angiomas on the Scalp. Case Rep Dermatol. 2009;1(1):82–86.
2. A 60-year-old African-American woman presents with painful swelling of two years’ duration. She delayed care due to lack of insurance. The patient is hypertensive, and her left breast and nipple are retracted, with darkened skin and a peau d’orange texture.
Diagnosis: Palpation of the firm, matted nodes in the left axilla elucidated the diagnosis of breast cancer with lymphedema. Lymphedema causes the skin of the breast to resemble that of an orange.
The patient was referred to the local university’s breast center. Although the prognosis was poor, it was important to make every effort to have the disease staged to determine the most appropriate therapy.
Photos and text for Photo Rounds Friday courtesy of Richard P. Usatine, MD. This case was adapted from: Mayeaux, EJ. Breast cancer. In: Usatine R, Smith M, Mayeaux EJ, et al, eds. Color Atlas of Family Medicine. 2nd ed. New York, NY: McGraw-Hill; 2013:551-556.
For more information, see “Swollen breast and arm.” The Journal of Family Practice. February 6, 2015.
3. This patient presents with her cheeks quite red and covered with hyperkeratotic, rough, pinpoint papules. No other blemishes or lesions are seen on her face, but there are hundreds of hyperkeratotic papules on her bilateral triceps, giving a “chicken skin” appearance.
Diagnosis: Keratosis pilaris (KP) is an extremely common and harmless problem that affects up to 70% of newborns, though it may not fully express until age 1 or 2. Caused by an overproduction of perifollicular keratin, KP is inherited in an autosomal dominant pattern and is often seen in conjunction with atopic dermatitis and related conditions (eg, eczema, xerosis, asthma, icthyosis). KP can manifest anywhere on the body except glabrous skin (palms and soles).
Variants of KP are also common; the one affecting this patient is keratosis pilaris rubra facei. This condition is often confused with acne, but treating it as such worsens irritation—especially in the wintertime, when humidity levels are low.
For more information, see “Acne: Maybe She's Born With It?” Clinician Reviews. 2016 July;26(7):W1.
4. A 5-year-old girl has a fever of 102.4°F and “strawberry tongue.” The posterior pharynx is erythematous with slight exudate visible. The anterior cervical lymph nodes are mildly tender and somewhat enlarged. No rashes are noted.
Diagnosis: The child’s strawberry tongue and scarlet fever were caused by strep pharyngitis. Strawberry tongue, identified by prominent papillae along with erythema (resembling a strawberry), is most commonly seen in children with scarlet fever or Kawasaki disease and usually develops within the first two to three days of illness. A white or yellowish coating typically precedes the distinctive red tongue with white papillae.
In this case, oral penicillin VK was prescribed, along with ibuprofen for the fever and sore throat. Improvement was noted within 24 hours, but the full 10-day course of pencillin was completed to prevent rheumatic fever.
For more information, see “Papillae on tongue.” Journal of Family Practice. January 24, 2014.
1. This 45-year-old woman’s skin has multiple small, soft, compressible papules, one of which is apt to bleed when scratched. She is concerned that these “cherry red” lesions are precancerous.
Diagnosis: Cherry angiomas, also known as de Morgan spots, are extremely common lesions; though usually asymptomatic, they may bleed with trauma. They occur most commonly as multiple asymptomatic lesions on the trunk and arms. These capillary hemangiomas are dome-shaped, small (0.1 to 0.5 cm in diameter), and bright red to violaceous; they can be flat, raised, or nodular.
Cherry angiomas form as a result of the development of multiple capillaries with narrow lumens and prominent endothelial cells arranged in a lobular pattern in the papillary dermis. Effective treatment options include curettage, laser ablation, and electrodesiccation.
For more information, see Kim, J-H, Park H-Y, Ahn SK. Cherry Angiomas on the Scalp. Case Rep Dermatol. 2009;1(1):82–86.
2. A 60-year-old African-American woman presents with painful swelling of two years’ duration. She delayed care due to lack of insurance. The patient is hypertensive, and her left breast and nipple are retracted, with darkened skin and a peau d’orange texture.
Diagnosis: Palpation of the firm, matted nodes in the left axilla elucidated the diagnosis of breast cancer with lymphedema. Lymphedema causes the skin of the breast to resemble that of an orange.
The patient was referred to the local university’s breast center. Although the prognosis was poor, it was important to make every effort to have the disease staged to determine the most appropriate therapy.
Photos and text for Photo Rounds Friday courtesy of Richard P. Usatine, MD. This case was adapted from: Mayeaux, EJ. Breast cancer. In: Usatine R, Smith M, Mayeaux EJ, et al, eds. Color Atlas of Family Medicine. 2nd ed. New York, NY: McGraw-Hill; 2013:551-556.
For more information, see “Swollen breast and arm.” The Journal of Family Practice. February 6, 2015.
3. This patient presents with her cheeks quite red and covered with hyperkeratotic, rough, pinpoint papules. No other blemishes or lesions are seen on her face, but there are hundreds of hyperkeratotic papules on her bilateral triceps, giving a “chicken skin” appearance.
Diagnosis: Keratosis pilaris (KP) is an extremely common and harmless problem that affects up to 70% of newborns, though it may not fully express until age 1 or 2. Caused by an overproduction of perifollicular keratin, KP is inherited in an autosomal dominant pattern and is often seen in conjunction with atopic dermatitis and related conditions (eg, eczema, xerosis, asthma, icthyosis). KP can manifest anywhere on the body except glabrous skin (palms and soles).
Variants of KP are also common; the one affecting this patient is keratosis pilaris rubra facei. This condition is often confused with acne, but treating it as such worsens irritation—especially in the wintertime, when humidity levels are low.
For more information, see “Acne: Maybe She's Born With It?” Clinician Reviews. 2016 July;26(7):W1.
4. A 5-year-old girl has a fever of 102.4°F and “strawberry tongue.” The posterior pharynx is erythematous with slight exudate visible. The anterior cervical lymph nodes are mildly tender and somewhat enlarged. No rashes are noted.
Diagnosis: The child’s strawberry tongue and scarlet fever were caused by strep pharyngitis. Strawberry tongue, identified by prominent papillae along with erythema (resembling a strawberry), is most commonly seen in children with scarlet fever or Kawasaki disease and usually develops within the first two to three days of illness. A white or yellowish coating typically precedes the distinctive red tongue with white papillae.
In this case, oral penicillin VK was prescribed, along with ibuprofen for the fever and sore throat. Improvement was noted within 24 hours, but the full 10-day course of pencillin was completed to prevent rheumatic fever.
For more information, see “Papillae on tongue.” Journal of Family Practice. January 24, 2014.
1. This 45-year-old woman’s skin has multiple small, soft, compressible papules, one of which is apt to bleed when scratched. She is concerned that these “cherry red” lesions are precancerous.
Diagnosis: Cherry angiomas, also known as de Morgan spots, are extremely common lesions; though usually asymptomatic, they may bleed with trauma. They occur most commonly as multiple asymptomatic lesions on the trunk and arms. These capillary hemangiomas are dome-shaped, small (0.1 to 0.5 cm in diameter), and bright red to violaceous; they can be flat, raised, or nodular.
Cherry angiomas form as a result of the development of multiple capillaries with narrow lumens and prominent endothelial cells arranged in a lobular pattern in the papillary dermis. Effective treatment options include curettage, laser ablation, and electrodesiccation.
For more information, see Kim, J-H, Park H-Y, Ahn SK. Cherry Angiomas on the Scalp. Case Rep Dermatol. 2009;1(1):82–86.
2. A 60-year-old African-American woman presents with painful swelling of two years’ duration. She delayed care due to lack of insurance. The patient is hypertensive, and her left breast and nipple are retracted, with darkened skin and a peau d’orange texture.
Diagnosis: Palpation of the firm, matted nodes in the left axilla elucidated the diagnosis of breast cancer with lymphedema. Lymphedema causes the skin of the breast to resemble that of an orange.
The patient was referred to the local university’s breast center. Although the prognosis was poor, it was important to make every effort to have the disease staged to determine the most appropriate therapy.
Photos and text for Photo Rounds Friday courtesy of Richard P. Usatine, MD. This case was adapted from: Mayeaux, EJ. Breast cancer. In: Usatine R, Smith M, Mayeaux EJ, et al, eds. Color Atlas of Family Medicine. 2nd ed. New York, NY: McGraw-Hill; 2013:551-556.
For more information, see “Swollen breast and arm.” The Journal of Family Practice. February 6, 2015.
3. This patient presents with her cheeks quite red and covered with hyperkeratotic, rough, pinpoint papules. No other blemishes or lesions are seen on her face, but there are hundreds of hyperkeratotic papules on her bilateral triceps, giving a “chicken skin” appearance.
Diagnosis: Keratosis pilaris (KP) is an extremely common and harmless problem that affects up to 70% of newborns, though it may not fully express until age 1 or 2. Caused by an overproduction of perifollicular keratin, KP is inherited in an autosomal dominant pattern and is often seen in conjunction with atopic dermatitis and related conditions (eg, eczema, xerosis, asthma, icthyosis). KP can manifest anywhere on the body except glabrous skin (palms and soles).
Variants of KP are also common; the one affecting this patient is keratosis pilaris rubra facei. This condition is often confused with acne, but treating it as such worsens irritation—especially in the wintertime, when humidity levels are low.
For more information, see “Acne: Maybe She's Born With It?” Clinician Reviews. 2016 July;26(7):W1.
4. A 5-year-old girl has a fever of 102.4°F and “strawberry tongue.” The posterior pharynx is erythematous with slight exudate visible. The anterior cervical lymph nodes are mildly tender and somewhat enlarged. No rashes are noted.
Diagnosis: The child’s strawberry tongue and scarlet fever were caused by strep pharyngitis. Strawberry tongue, identified by prominent papillae along with erythema (resembling a strawberry), is most commonly seen in children with scarlet fever or Kawasaki disease and usually develops within the first two to three days of illness. A white or yellowish coating typically precedes the distinctive red tongue with white papillae.
In this case, oral penicillin VK was prescribed, along with ibuprofen for the fever and sore throat. Improvement was noted within 24 hours, but the full 10-day course of pencillin was completed to prevent rheumatic fever.
For more information, see “Papillae on tongue.” Journal of Family Practice. January 24, 2014.
Best Practices: In the Management of Hemophilia
The treatment of patients with hemophilia has rapidly evolved from one-size-fits-all factor replacement strategies to highly individualized, patient-specific care.
Faculty:
Erik Berntorp, MD, PhD
Malmö Centre for Thrombosis and Haemostasis
Lund University
Malmö, Sweden
Faculty Disclosures:
This sponsored content was prepared by Dr. Berntorp and reviewed by Shire. Dr. Berntorp discloses that he is a consultant and on the advisory boards and speakers’ bureaus for Bayer, CSL Behring, Octapharma, Shire, and Sobi. The production of this section did not involve the news or editorial staff of Frontline Medical Communications.
S28001
2/17
Click here to read the supplement
The treatment of patients with hemophilia has rapidly evolved from one-size-fits-all factor replacement strategies to highly individualized, patient-specific care.
Faculty:
Erik Berntorp, MD, PhD
Malmö Centre for Thrombosis and Haemostasis
Lund University
Malmö, Sweden
Faculty Disclosures:
This sponsored content was prepared by Dr. Berntorp and reviewed by Shire. Dr. Berntorp discloses that he is a consultant and on the advisory boards and speakers’ bureaus for Bayer, CSL Behring, Octapharma, Shire, and Sobi. The production of this section did not involve the news or editorial staff of Frontline Medical Communications.
S28001
2/17
Click here to read the supplement
The treatment of patients with hemophilia has rapidly evolved from one-size-fits-all factor replacement strategies to highly individualized, patient-specific care.
Faculty:
Erik Berntorp, MD, PhD
Malmö Centre for Thrombosis and Haemostasis
Lund University
Malmö, Sweden
Faculty Disclosures:
This sponsored content was prepared by Dr. Berntorp and reviewed by Shire. Dr. Berntorp discloses that he is a consultant and on the advisory boards and speakers’ bureaus for Bayer, CSL Behring, Octapharma, Shire, and Sobi. The production of this section did not involve the news or editorial staff of Frontline Medical Communications.
S28001
2/17
Click here to read the supplement
Imbalance drives development of B-ALL, team says
Researchers say they have discovered an imbalance that drives the development of B-cell acute lymphoblastic leukemia (B-ALL).
The group’s study suggests that activation of STAT5 causes competition among other transcription factors that leads to B-ALL.
Therefore, the researchers believe that inhibiting the activation of STAT5 and restoring the natural balance of proteins could mean more effective treatment for B-ALL.
Seth Frietze, PhD, of the University of Vermont in Burlington, Vermont, and his colleagues conducted this research and reported the results in Nature Immunology.
The researchers first studied the role of STAT5 in B-ALL using mouse models.
The experiments revealed that STAT5 activation and defects in a signaling pathway worked together to promote B-ALL. The defects were in signaling components of the B-cell antigen receptor precursor—IKAROS, NF-κB, BLNK, BTK, and PKCβ.
With further investigation, the researchers found that STAT5 “antagonized NF-κB and IKAROS by opposing the regulation of shared target genes.”
The team also studied samples from patients with B-ALL and found that patients with a high ratio of active STAT5 to NF-κB or IKAROS had more aggressive disease.
Specifically, the ratio of active STAT5 to IKAROS was negatively correlated with patient survival and the duration of remission. The ratio of active STAT5 to the NF-κB subunit RELA correlated with remission duration but not survival.
“The major outcome of this story is that a signature emerged from looking at the level of activated proteins compared to other proteins that’s very predictive of how a patient will respond to therapy,” Dr Frietze said.
“That’s a novel finding. If we could find drugs to target that activation, that could be an incredibly effective way to treat leukemia.” 
Researchers say they have discovered an imbalance that drives the development of B-cell acute lymphoblastic leukemia (B-ALL).
The group’s study suggests that activation of STAT5 causes competition among other transcription factors that leads to B-ALL.
Therefore, the researchers believe that inhibiting the activation of STAT5 and restoring the natural balance of proteins could mean more effective treatment for B-ALL.
Seth Frietze, PhD, of the University of Vermont in Burlington, Vermont, and his colleagues conducted this research and reported the results in Nature Immunology.
The researchers first studied the role of STAT5 in B-ALL using mouse models.
The experiments revealed that STAT5 activation and defects in a signaling pathway worked together to promote B-ALL. The defects were in signaling components of the B-cell antigen receptor precursor—IKAROS, NF-κB, BLNK, BTK, and PKCβ.
With further investigation, the researchers found that STAT5 “antagonized NF-κB and IKAROS by opposing the regulation of shared target genes.”
The team also studied samples from patients with B-ALL and found that patients with a high ratio of active STAT5 to NF-κB or IKAROS had more aggressive disease.
Specifically, the ratio of active STAT5 to IKAROS was negatively correlated with patient survival and the duration of remission. The ratio of active STAT5 to the NF-κB subunit RELA correlated with remission duration but not survival.
“The major outcome of this story is that a signature emerged from looking at the level of activated proteins compared to other proteins that’s very predictive of how a patient will respond to therapy,” Dr Frietze said.
“That’s a novel finding. If we could find drugs to target that activation, that could be an incredibly effective way to treat leukemia.”
Researchers say they have discovered an imbalance that drives the development of B-cell acute lymphoblastic leukemia (B-ALL).
The group’s study suggests that activation of STAT5 causes competition among other transcription factors that leads to B-ALL.
Therefore, the researchers believe that inhibiting the activation of STAT5 and restoring the natural balance of proteins could mean more effective treatment for B-ALL.
Seth Frietze, PhD, of the University of Vermont in Burlington, Vermont, and his colleagues conducted this research and reported the results in Nature Immunology.
The researchers first studied the role of STAT5 in B-ALL using mouse models.
The experiments revealed that STAT5 activation and defects in a signaling pathway worked together to promote B-ALL. The defects were in signaling components of the B-cell antigen receptor precursor—IKAROS, NF-κB, BLNK, BTK, and PKCβ.
With further investigation, the researchers found that STAT5 “antagonized NF-κB and IKAROS by opposing the regulation of shared target genes.”
The team also studied samples from patients with B-ALL and found that patients with a high ratio of active STAT5 to NF-κB or IKAROS had more aggressive disease.
Specifically, the ratio of active STAT5 to IKAROS was negatively correlated with patient survival and the duration of remission. The ratio of active STAT5 to the NF-κB subunit RELA correlated with remission duration but not survival.
“The major outcome of this story is that a signature emerged from looking at the level of activated proteins compared to other proteins that’s very predictive of how a patient will respond to therapy,” Dr Frietze said.
“That’s a novel finding. If we could find drugs to target that activation, that could be an incredibly effective way to treat leukemia.”
TKI shows promise in preclinical study of AML
WASHINGTON, DC—Preclinical data suggest a novel tyrosine kinase inhibitor (TKI) may be an effective treatment for patients with NTRK-rearranged acute myeloid leukemia (AML).
Entrectinib is a TKI targeting tumors that harbor TRK, ROS1, or ALK fusions.
Researchers found that entrectinib inhibited cell proliferation in NTRK-rearranged AML cell lines.
In mouse models of NTRK-rearranged AML, entrectinib induced tumor regression and eliminated residual AML cells from the bone marrow.
These results were presented at the AACR Annual Meeting 2017 (abstract 5158). The research was conducted by employees of Ignyta, Inc., the company developing entrectinib.
The researchers first tested entrectinib in AML cell lines. They observed “potent” anti-proliferative activity in a pair of NTRK-fusion-positive AML cell lines, IMS-M2 and M0-91.
Entrectinib inhibited TRK signaling and induced cell-cycle arrest in these cell lines. The TKI also induced both caspase 3-dependent apoptosis and PARP cleavage in a dose- and time-dependent manner.
However, entrectinib showed minimal activity against an NTRK-fusion-negative AML cell line, Kasumi-1.
The researchers also tested entrectinib in mouse models of NTRK-fusion-driven AML.
The TKI induced tumor regression in both IMS-M2 and M0-91 models, and the drug eliminated leukemic cells in the bone marrow.
The researchers said these results provide rationale for the clinical development of entrectinib in molecularly defined hematologic malignancies.
Entrectinib is currently being studied in a phase 2 trial of solid tumor malignancies.
WASHINGTON, DC—Preclinical data suggest a novel tyrosine kinase inhibitor (TKI) may be an effective treatment for patients with NTRK-rearranged acute myeloid leukemia (AML).
Entrectinib is a TKI targeting tumors that harbor TRK, ROS1, or ALK fusions.
Researchers found that entrectinib inhibited cell proliferation in NTRK-rearranged AML cell lines.
In mouse models of NTRK-rearranged AML, entrectinib induced tumor regression and eliminated residual AML cells from the bone marrow.
These results were presented at the AACR Annual Meeting 2017 (abstract 5158). The research was conducted by employees of Ignyta, Inc., the company developing entrectinib.
The researchers first tested entrectinib in AML cell lines. They observed “potent” anti-proliferative activity in a pair of NTRK-fusion-positive AML cell lines, IMS-M2 and M0-91.
Entrectinib inhibited TRK signaling and induced cell-cycle arrest in these cell lines. The TKI also induced both caspase 3-dependent apoptosis and PARP cleavage in a dose- and time-dependent manner.
However, entrectinib showed minimal activity against an NTRK-fusion-negative AML cell line, Kasumi-1.
The researchers also tested entrectinib in mouse models of NTRK-fusion-driven AML.
The TKI induced tumor regression in both IMS-M2 and M0-91 models, and the drug eliminated leukemic cells in the bone marrow.
The researchers said these results provide rationale for the clinical development of entrectinib in molecularly defined hematologic malignancies.
Entrectinib is currently being studied in a phase 2 trial of solid tumor malignancies.
WASHINGTON, DC—Preclinical data suggest a novel tyrosine kinase inhibitor (TKI) may be an effective treatment for patients with NTRK-rearranged acute myeloid leukemia (AML).
Entrectinib is a TKI targeting tumors that harbor TRK, ROS1, or ALK fusions.
Researchers found that entrectinib inhibited cell proliferation in NTRK-rearranged AML cell lines.
In mouse models of NTRK-rearranged AML, entrectinib induced tumor regression and eliminated residual AML cells from the bone marrow.
These results were presented at the AACR Annual Meeting 2017 (abstract 5158). The research was conducted by employees of Ignyta, Inc., the company developing entrectinib.
The researchers first tested entrectinib in AML cell lines. They observed “potent” anti-proliferative activity in a pair of NTRK-fusion-positive AML cell lines, IMS-M2 and M0-91.
Entrectinib inhibited TRK signaling and induced cell-cycle arrest in these cell lines. The TKI also induced both caspase 3-dependent apoptosis and PARP cleavage in a dose- and time-dependent manner.
However, entrectinib showed minimal activity against an NTRK-fusion-negative AML cell line, Kasumi-1.
The researchers also tested entrectinib in mouse models of NTRK-fusion-driven AML.
The TKI induced tumor regression in both IMS-M2 and M0-91 models, and the drug eliminated leukemic cells in the bone marrow.
The researchers said these results provide rationale for the clinical development of entrectinib in molecularly defined hematologic malignancies.
Entrectinib is currently being studied in a phase 2 trial of solid tumor malignancies.
Minimal residual disease eyed for myeloma management
NEW YORK – Testing for minimal residual disease is clearly the state-of-the-art way to gauge the status of treated patients with multiple myeloma, agreed experts. The issue now is whether reliance on MRD to guide management remains investigational or ready for routine practice.
Paul G. Richardson, MD, gave MRD full endorsement as the wave of the future, but cautioned against routine use right now. “MRD is an exciting new tool [but] is not yet the standard of care for routine practice,” he said at the conference held by Imedex.
He cited results from a recent meta-analysis that included data from 14 studies of multiple myeloma patients that examined the correlation of MRD status with progression-free survival and 12 studies that addressed how MRD related to overall survival. The meta-analysis results showed that, among patients in complete remission, the average duration of progression-free survival was 56 months in patients who began follow-up without detectable MRD and 34 months in patients with detectable MRD (JAMA Oncology. 2017 Jan;3[1]:28-35). The average duration of overall survival was 112 months in patients with undetectable MRD and 82 months in those with detectable MRD.
But MRD assessment still has several limitations. Evaluations of its strength for prognosis have largely been based on flow cytometry measures of MRD, an approach recently eclipsed by next-generation sequencing and polymerase chain reaction–based assays. Lack of standardization across the multiple tests now available is another hindrance. In addition, undetectable MRD in a myeloma patient in no way means that the malignancy is gone and that treatment can stop. Patients with undetectable MRD may have better medium-term outcomes, compared with patients with detectable MRD, but even patients with undetectable multiple myeloma still succumb to disease progression several years later.
“Patients negative for MRD do better than patients with detectable MRD, but they still relapse and die. You must maintain treatment in patients negative for MRD,” Dr. Richardson said. The clinical experience “supports the idea that, going forward, MRD has real potential, but MRD is not yet standard of care for routine practice,” he said.
In late 2015, Memorial Sloan-Kettering Cancer Center began routinely using MRD to assess multiple myeloma patients, said C. Ola Landgren, MD, PhD, professor of medicine at Weill Cornell Medical College and chief of the Myeloma Service at Memorial Sloan-Kettering.
He and his associates focus on newly diagnosed multiple myeloma patients who have undergone induction therapy. Patients who come out of the regimen with an undetectable level of MRD at a sensitivity of one cell in a million have the option of receiving high-dose melphalan (Alkeran) followed by autologous stem cell transplantation or maintenance therapy with lenalidomide (Revlimid). Patients who finish induction with detectable MRD can either receive immediate treatment with high-dose melphalan followed by autologous stem cell transplantation or further combination treatment with lenalidomide, carfilzomib (Kyprolis), and dexamethasone, then followed by melphalan and stem cell transplantation (Bone Marrow Transplantation. 2016 July;51[7]:913-4).
“The data show that patients who become MRD negative have longer progression-free survival and overall survival, and we find that more and more patients achieve a MRD-negative state,” said Dr. Landgren. “In our practice, it’s about half of newly diagnosed patients. I think that achieving MRD negativity is more important than the specific treatment a patient receives” for predicting prognosis, he said.
Dr. Richardson has been a consultant to Celgene, Genmab, Janssen, Novartis, Oncopeptides, and Takeda and has received research funding from Celgene and Takeda. Dr. Landgren has been a consultant to Amgen and Takeda and a speaker on behalf of Plexus.
[email protected]
On Twitter @mitchelzoler
NEW YORK – Testing for minimal residual disease is clearly the state-of-the-art way to gauge the status of treated patients with multiple myeloma, agreed experts. The issue now is whether reliance on MRD to guide management remains investigational or ready for routine practice.
Paul G. Richardson, MD, gave MRD full endorsement as the wave of the future, but cautioned against routine use right now. “MRD is an exciting new tool [but] is not yet the standard of care for routine practice,” he said at the conference held by Imedex.
He cited results from a recent meta-analysis that included data from 14 studies of multiple myeloma patients that examined the correlation of MRD status with progression-free survival and 12 studies that addressed how MRD related to overall survival. The meta-analysis results showed that, among patients in complete remission, the average duration of progression-free survival was 56 months in patients who began follow-up without detectable MRD and 34 months in patients with detectable MRD (JAMA Oncology. 2017 Jan;3[1]:28-35). The average duration of overall survival was 112 months in patients with undetectable MRD and 82 months in those with detectable MRD.
But MRD assessment still has several limitations. Evaluations of its strength for prognosis have largely been based on flow cytometry measures of MRD, an approach recently eclipsed by next-generation sequencing and polymerase chain reaction–based assays. Lack of standardization across the multiple tests now available is another hindrance. In addition, undetectable MRD in a myeloma patient in no way means that the malignancy is gone and that treatment can stop. Patients with undetectable MRD may have better medium-term outcomes, compared with patients with detectable MRD, but even patients with undetectable multiple myeloma still succumb to disease progression several years later.
“Patients negative for MRD do better than patients with detectable MRD, but they still relapse and die. You must maintain treatment in patients negative for MRD,” Dr. Richardson said. The clinical experience “supports the idea that, going forward, MRD has real potential, but MRD is not yet standard of care for routine practice,” he said.
In late 2015, Memorial Sloan-Kettering Cancer Center began routinely using MRD to assess multiple myeloma patients, said C. Ola Landgren, MD, PhD, professor of medicine at Weill Cornell Medical College and chief of the Myeloma Service at Memorial Sloan-Kettering.
He and his associates focus on newly diagnosed multiple myeloma patients who have undergone induction therapy. Patients who come out of the regimen with an undetectable level of MRD at a sensitivity of one cell in a million have the option of receiving high-dose melphalan (Alkeran) followed by autologous stem cell transplantation or maintenance therapy with lenalidomide (Revlimid). Patients who finish induction with detectable MRD can either receive immediate treatment with high-dose melphalan followed by autologous stem cell transplantation or further combination treatment with lenalidomide, carfilzomib (Kyprolis), and dexamethasone, then followed by melphalan and stem cell transplantation (Bone Marrow Transplantation. 2016 July;51[7]:913-4).
“The data show that patients who become MRD negative have longer progression-free survival and overall survival, and we find that more and more patients achieve a MRD-negative state,” said Dr. Landgren. “In our practice, it’s about half of newly diagnosed patients. I think that achieving MRD negativity is more important than the specific treatment a patient receives” for predicting prognosis, he said.
Dr. Richardson has been a consultant to Celgene, Genmab, Janssen, Novartis, Oncopeptides, and Takeda and has received research funding from Celgene and Takeda. Dr. Landgren has been a consultant to Amgen and Takeda and a speaker on behalf of Plexus.
[email protected]
On Twitter @mitchelzoler
NEW YORK – Testing for minimal residual disease is clearly the state-of-the-art way to gauge the status of treated patients with multiple myeloma, agreed experts. The issue now is whether reliance on MRD to guide management remains investigational or ready for routine practice.
Paul G. Richardson, MD, gave MRD full endorsement as the wave of the future, but cautioned against routine use right now. “MRD is an exciting new tool [but] is not yet the standard of care for routine practice,” he said at the conference held by Imedex.
He cited results from a recent meta-analysis that included data from 14 studies of multiple myeloma patients that examined the correlation of MRD status with progression-free survival and 12 studies that addressed how MRD related to overall survival. The meta-analysis results showed that, among patients in complete remission, the average duration of progression-free survival was 56 months in patients who began follow-up without detectable MRD and 34 months in patients with detectable MRD (JAMA Oncology. 2017 Jan;3[1]:28-35). The average duration of overall survival was 112 months in patients with undetectable MRD and 82 months in those with detectable MRD.
But MRD assessment still has several limitations. Evaluations of its strength for prognosis have largely been based on flow cytometry measures of MRD, an approach recently eclipsed by next-generation sequencing and polymerase chain reaction–based assays. Lack of standardization across the multiple tests now available is another hindrance. In addition, undetectable MRD in a myeloma patient in no way means that the malignancy is gone and that treatment can stop. Patients with undetectable MRD may have better medium-term outcomes, compared with patients with detectable MRD, but even patients with undetectable multiple myeloma still succumb to disease progression several years later.
“Patients negative for MRD do better than patients with detectable MRD, but they still relapse and die. You must maintain treatment in patients negative for MRD,” Dr. Richardson said. The clinical experience “supports the idea that, going forward, MRD has real potential, but MRD is not yet standard of care for routine practice,” he said.
In late 2015, Memorial Sloan-Kettering Cancer Center began routinely using MRD to assess multiple myeloma patients, said C. Ola Landgren, MD, PhD, professor of medicine at Weill Cornell Medical College and chief of the Myeloma Service at Memorial Sloan-Kettering.
He and his associates focus on newly diagnosed multiple myeloma patients who have undergone induction therapy. Patients who come out of the regimen with an undetectable level of MRD at a sensitivity of one cell in a million have the option of receiving high-dose melphalan (Alkeran) followed by autologous stem cell transplantation or maintenance therapy with lenalidomide (Revlimid). Patients who finish induction with detectable MRD can either receive immediate treatment with high-dose melphalan followed by autologous stem cell transplantation or further combination treatment with lenalidomide, carfilzomib (Kyprolis), and dexamethasone, then followed by melphalan and stem cell transplantation (Bone Marrow Transplantation. 2016 July;51[7]:913-4).
“The data show that patients who become MRD negative have longer progression-free survival and overall survival, and we find that more and more patients achieve a MRD-negative state,” said Dr. Landgren. “In our practice, it’s about half of newly diagnosed patients. I think that achieving MRD negativity is more important than the specific treatment a patient receives” for predicting prognosis, he said.
Dr. Richardson has been a consultant to Celgene, Genmab, Janssen, Novartis, Oncopeptides, and Takeda and has received research funding from Celgene and Takeda. Dr. Landgren has been a consultant to Amgen and Takeda and a speaker on behalf of Plexus.
[email protected]
On Twitter @mitchelzoler
EXPERT ANALYSIS FROM A MEETING ON HEMATOLOGIC MALIGNANCIES
Order entry tool halves prescription drug costs
SAN DIEGO – What if you could cut patient drug costs, improve adherence, and cut hospitalization by using an online tool?
“Sounds like science fiction, right? I’m here to tell you it’s reality,” Alan A. Kubey, MD, said at the annual meeting of the American College of Physicians.
During his medical internship 3 years ago, Dr. Kubey met an 83-year-old woman on Medicare Part A and B, but not D, coverage. She was on a cardiology service, came in with a non-ST segment elevation myocardial infarction, and was treated conservatively. “She confided in me that it was hard for her, on a fixed income, to afford her medications and to do the things she loved to do,” said Dr. Kubey, who is now a hospitalist at Mayo Clinic, Rochester, Minn., and at Thomas Jefferson University Hospital, Philadelphia. After researching pharmacy options online, Dr. Kubey told the patient that he could get her medication cost down from $465 per day to $65 per day. “She was blown away, and I was blown away,” he said. “It seemed too good to be true. I then did a long retrospective analysis of patients in similar situations.”
During the ACP’s “Dragon’s Lair” competition at the meeting, Dr. Kubey earned the top prize of $7,500 to study the idea further. To date, he and his fellow researchers have used the tool for 25 patients and have saved each one about $5,600 per year in medication costs. Specifically, average costs have dropped from $6,282.54 per year to $598.84 per year, a savings of 88%. “Our plan now is to prospectively validate this in a small, single-site trial to show that this works,” he said. “If it does, we want to study it and develop it further and provide it more broadly to empower clinicians to improve the lives of patients.”
Each year, patients in the United States spend $325 billion on outpatient medications that hold the power to improve health and to save lives. “Yet, we know that up to 45% of patients don’t fill those scripts because of cost,” Dr. Kubey said. “Medication nonadherence leads to an additional $100 billion to $300 billion per year in excess care and an incalculable human cost in lost health. Providers are responsible for guiding patients in their medication decision making, yet study after study shows that physicians are woefully ill informed on the cost of medications. Yet even the astute clinician faces a Byzantine landscape that makes no sense, where a medication may cost up to 69-fold from one pharmacy to the next. What are we to do?
“We believe that you have to do it at the point of entry. There are innovative tools for patients like GoodRx, but, in my opinion, that puts too much on the patients’ shoulders. We want to bring this information directly to the provider – to build upon the progress that the likes of GoodRx have accomplished and add our advanced algorithm to rapidly empower the physician to send the patient to the most cost-effective pharmacy for the most cost-effective regimen on day 1.”
Dr. Kubey reported having no financial disclosures.
SAN DIEGO – What if you could cut patient drug costs, improve adherence, and cut hospitalization by using an online tool?
“Sounds like science fiction, right? I’m here to tell you it’s reality,” Alan A. Kubey, MD, said at the annual meeting of the American College of Physicians.
During his medical internship 3 years ago, Dr. Kubey met an 83-year-old woman on Medicare Part A and B, but not D, coverage. She was on a cardiology service, came in with a non-ST segment elevation myocardial infarction, and was treated conservatively. “She confided in me that it was hard for her, on a fixed income, to afford her medications and to do the things she loved to do,” said Dr. Kubey, who is now a hospitalist at Mayo Clinic, Rochester, Minn., and at Thomas Jefferson University Hospital, Philadelphia. After researching pharmacy options online, Dr. Kubey told the patient that he could get her medication cost down from $465 per day to $65 per day. “She was blown away, and I was blown away,” he said. “It seemed too good to be true. I then did a long retrospective analysis of patients in similar situations.”
During the ACP’s “Dragon’s Lair” competition at the meeting, Dr. Kubey earned the top prize of $7,500 to study the idea further. To date, he and his fellow researchers have used the tool for 25 patients and have saved each one about $5,600 per year in medication costs. Specifically, average costs have dropped from $6,282.54 per year to $598.84 per year, a savings of 88%. “Our plan now is to prospectively validate this in a small, single-site trial to show that this works,” he said. “If it does, we want to study it and develop it further and provide it more broadly to empower clinicians to improve the lives of patients.”
Each year, patients in the United States spend $325 billion on outpatient medications that hold the power to improve health and to save lives. “Yet, we know that up to 45% of patients don’t fill those scripts because of cost,” Dr. Kubey said. “Medication nonadherence leads to an additional $100 billion to $300 billion per year in excess care and an incalculable human cost in lost health. Providers are responsible for guiding patients in their medication decision making, yet study after study shows that physicians are woefully ill informed on the cost of medications. Yet even the astute clinician faces a Byzantine landscape that makes no sense, where a medication may cost up to 69-fold from one pharmacy to the next. What are we to do?
“We believe that you have to do it at the point of entry. There are innovative tools for patients like GoodRx, but, in my opinion, that puts too much on the patients’ shoulders. We want to bring this information directly to the provider – to build upon the progress that the likes of GoodRx have accomplished and add our advanced algorithm to rapidly empower the physician to send the patient to the most cost-effective pharmacy for the most cost-effective regimen on day 1.”
Dr. Kubey reported having no financial disclosures.
SAN DIEGO – What if you could cut patient drug costs, improve adherence, and cut hospitalization by using an online tool?
“Sounds like science fiction, right? I’m here to tell you it’s reality,” Alan A. Kubey, MD, said at the annual meeting of the American College of Physicians.
During his medical internship 3 years ago, Dr. Kubey met an 83-year-old woman on Medicare Part A and B, but not D, coverage. She was on a cardiology service, came in with a non-ST segment elevation myocardial infarction, and was treated conservatively. “She confided in me that it was hard for her, on a fixed income, to afford her medications and to do the things she loved to do,” said Dr. Kubey, who is now a hospitalist at Mayo Clinic, Rochester, Minn., and at Thomas Jefferson University Hospital, Philadelphia. After researching pharmacy options online, Dr. Kubey told the patient that he could get her medication cost down from $465 per day to $65 per day. “She was blown away, and I was blown away,” he said. “It seemed too good to be true. I then did a long retrospective analysis of patients in similar situations.”
During the ACP’s “Dragon’s Lair” competition at the meeting, Dr. Kubey earned the top prize of $7,500 to study the idea further. To date, he and his fellow researchers have used the tool for 25 patients and have saved each one about $5,600 per year in medication costs. Specifically, average costs have dropped from $6,282.54 per year to $598.84 per year, a savings of 88%. “Our plan now is to prospectively validate this in a small, single-site trial to show that this works,” he said. “If it does, we want to study it and develop it further and provide it more broadly to empower clinicians to improve the lives of patients.”
Each year, patients in the United States spend $325 billion on outpatient medications that hold the power to improve health and to save lives. “Yet, we know that up to 45% of patients don’t fill those scripts because of cost,” Dr. Kubey said. “Medication nonadherence leads to an additional $100 billion to $300 billion per year in excess care and an incalculable human cost in lost health. Providers are responsible for guiding patients in their medication decision making, yet study after study shows that physicians are woefully ill informed on the cost of medications. Yet even the astute clinician faces a Byzantine landscape that makes no sense, where a medication may cost up to 69-fold from one pharmacy to the next. What are we to do?
“We believe that you have to do it at the point of entry. There are innovative tools for patients like GoodRx, but, in my opinion, that puts too much on the patients’ shoulders. We want to bring this information directly to the provider – to build upon the progress that the likes of GoodRx have accomplished and add our advanced algorithm to rapidly empower the physician to send the patient to the most cost-effective pharmacy for the most cost-effective regimen on day 1.”
Dr. Kubey reported having no financial disclosures.
EXPERT ANALYSIS AT ACP INTERNAL MEDICINE
VIDEO: GI innovators dive into the Shark Tank
BOSTON – The innovators who presented their novel technologies to a “shark tank” panel during the 2017 AGA Tech Summit sponsored by the AGA Center for GI Innovation and Technology proved that innovation is alive and well in the field of gastroenterology.
“Shark Tank this year was fabulous,” Dr. Michael L. Kochman, MD, AGAF, executive committee chair of the AGA Center for GI Innovation and Technology, said in a video interview. “It was great to see some really novel ideas and some great, innovative applications.”
Presenters received feedback on their proposals from representatives of the physician, medtech, and regulatory communities and were uniformly positive about the experience, citing the value of such information to decide whether or how to move their projects forward.
“Diving into meet with the “sharks” was “a great opportunity,” says Susan Hutfless, PhD., an epidemiologist at Johns Hopkins University School of Medicine, Baltimore. “I’m still swimming, still alive ... I would do it again.”
BOSTON – The innovators who presented their novel technologies to a “shark tank” panel during the 2017 AGA Tech Summit sponsored by the AGA Center for GI Innovation and Technology proved that innovation is alive and well in the field of gastroenterology.
“Shark Tank this year was fabulous,” Dr. Michael L. Kochman, MD, AGAF, executive committee chair of the AGA Center for GI Innovation and Technology, said in a video interview. “It was great to see some really novel ideas and some great, innovative applications.”
Presenters received feedback on their proposals from representatives of the physician, medtech, and regulatory communities and were uniformly positive about the experience, citing the value of such information to decide whether or how to move their projects forward.
“Diving into meet with the “sharks” was “a great opportunity,” says Susan Hutfless, PhD., an epidemiologist at Johns Hopkins University School of Medicine, Baltimore. “I’m still swimming, still alive ... I would do it again.”
BOSTON – The innovators who presented their novel technologies to a “shark tank” panel during the 2017 AGA Tech Summit sponsored by the AGA Center for GI Innovation and Technology proved that innovation is alive and well in the field of gastroenterology.
“Shark Tank this year was fabulous,” Dr. Michael L. Kochman, MD, AGAF, executive committee chair of the AGA Center for GI Innovation and Technology, said in a video interview. “It was great to see some really novel ideas and some great, innovative applications.”
Presenters received feedback on their proposals from representatives of the physician, medtech, and regulatory communities and were uniformly positive about the experience, citing the value of such information to decide whether or how to move their projects forward.
“Diving into meet with the “sharks” was “a great opportunity,” says Susan Hutfless, PhD., an epidemiologist at Johns Hopkins University School of Medicine, Baltimore. “I’m still swimming, still alive ... I would do it again.”
FROM THE AGA 2017 TECH SUMMIT
Three strategies target high quality care with new technologies
BOSTON – Unforeseen complications and steep learning curves represent the downside of new devices and new procedures, but there are some strategies to minimize these risks, according to experts outlining three such approaches during a session on quality outcomes at the 2017 AGA Tech Summit.
One approach involves a reorientation in skill acquisition in health care delivery. Another involves novel strategies to identify problems with new devices more rapidly. The third involves an ongoing evolution in mentoring clinicians through telecommunication.
Mastering new skills
The strategy described for equipping clinicians with the skills to produce uniform, reproducible outcomes is a technique referred to as simulation-based mastery learning, explained E. Matthew Ritter, MD, vice chairman for education and program director of the general surgery residency program at Walter Reed National Military Medical Center’s Uniformed Services University, Bethesda, Md. The technique is based on the goal of eliciting a “specific level of performance without regard to a time-based approach that is the typical standard in learning processes.”
“More and more evidence is available that performing something a lot of times does not necessarily correlate with performing something well,” Dr. Ritter said. He proposed that direct measurements of skill acquisition address this problem.
“My work has been primarily in laparoscopic surgery, but this approach is not specialty-specific,” Dr. Ritter said in an interview, explaining that the approach focuses on breaking down clinical procedures into tasks for which mastery can be objectively defined. The mastery learning for any one task is complete when performance criteria are met rather than after any specific number of repetitions.
“There is substantial evidence that this approach improves skill acquisition and improves outcome,” Dr. Ritter reported.
Identifying problems more quickly
To reduce the risks and complications of new devices, various strategies for postmarketing surveillance are being pursued simultaneously, according to Dana Telem, MD, MPH, director of the Michigan Comprehensive Hernia Program and an associate professor of surgery at the University of Michigan, Ann Arbor. As has been demonstrated repeatedly, the premarket testing and regulatory approval process does not rule out risk of unforeseen complications, including serious risks that may ultimately lead to the technology being discarded.
One approach has been the increasing use of registries so that constant capture of patient data allows isolated but recurring events to become rapidly identifiable, but Dr. Telem said that the value of these registries can be increased “by encouraging and empowering patients to report issues when they occur” to improve capture of adverse events.
As an example of a strategy to more rapidly drill down to the cause of complications, Dr. Telem reported on an effort to encourage creation of unique device identifiers for a given device. As devices are commonly updated and modified, the serial number may be useful when trying to determine whether the issue can be linked to a specific design change. More importantly, these numbers would be identifiable in billing data and potentially linked to registries to improve real time identification of adverse event patterns.
“The problem with adding new layers of premarketing regulation is the risk of stifling innovation, but we have not done a great job of postmarketing surveillance, and I think the focus now being placed on better strategies will greatly improve patient safety,” Dr. Telem reported.
Adding more layers of review and testing prior to approval of a new technology does not necessarily identify all risks, because not all complications are foreseeable, Dr. Telem explained.
“The long-term monitoring of new devices and technologies is mission critical if we really hope to safely innovate in the future.” Dr. Telem said.
Improving learning through telementoring
Employing avenues of telecommunication to better mentor clinicians acquiring new skills was the third example of an innovative area of improving quality assurance. A large focus of the presentation by Christopher Schlachta, MD, professor of surgery, Schulich School of Medicine and Dentistry, Western University, London, Ont., was on telementoring. He explained that this concept is not new, but it is becoming increasingly sophisticated, and there is a growing body of objective evidence that it is effective.
The Internet expands the possibilities. Remote screens can accommodate multiple images, including, in the case of surgical procedures, the surgical field and situational cameras that provide one or more views of the surgical team as they proceed. For laparoscopy, the information being fed to the mentor is often even more comprehensive.
“So much of what we do now is computer assisted, so the chip on the end of scope, for example, is capturing images digitally. These data can be transmitted and translated into images essentially simultaneously for the operator and a mentor who could be hundreds of miles away,” Dr. Schlachta reported.
There are now “plenty of studies that telementoring is effective in a variety of different surgical procedures,” Dr. Schlachta added. While the data confirming the value of telementoring for teaching skills in endoscopic procedures are fewer, Dr. Schlachta predicted this will soon change. He noted that the Society of American Gastrointestinal and Endoscopic Surgeons (SAGES) recently created a telementoring task force.
Reviewing several do-it-yourself and proprietary systems that have been developed for telemonitoring, Dr. Schlachta acknowledged that is it not always clear who should pay for this form of learning. He noted that there is potential value for mentees, hospitals wishing to expand services, and health systems attempting to improve quality outcomes. So far, industry has been the primary source of funds.
“For a company introducing a new technology, there is a risk that the technology will fail rapidly if there are bad results in the early going, making it valuable for industry to invest in these telementoring programs,” Dr. Schlacta explained.
“There are related technologies, such as those involving the use of tablets, that are also likely to contribute to the ongoing evolution in how procedural skills are taught and validated. I think these will accelerate and improve learning with the very important potential of better patient outcomes,” Dr. Schlachta reported.
BOSTON – Unforeseen complications and steep learning curves represent the downside of new devices and new procedures, but there are some strategies to minimize these risks, according to experts outlining three such approaches during a session on quality outcomes at the 2017 AGA Tech Summit.
One approach involves a reorientation in skill acquisition in health care delivery. Another involves novel strategies to identify problems with new devices more rapidly. The third involves an ongoing evolution in mentoring clinicians through telecommunication.
Mastering new skills
The strategy described for equipping clinicians with the skills to produce uniform, reproducible outcomes is a technique referred to as simulation-based mastery learning, explained E. Matthew Ritter, MD, vice chairman for education and program director of the general surgery residency program at Walter Reed National Military Medical Center’s Uniformed Services University, Bethesda, Md. The technique is based on the goal of eliciting a “specific level of performance without regard to a time-based approach that is the typical standard in learning processes.”
“More and more evidence is available that performing something a lot of times does not necessarily correlate with performing something well,” Dr. Ritter said. He proposed that direct measurements of skill acquisition address this problem.
“My work has been primarily in laparoscopic surgery, but this approach is not specialty-specific,” Dr. Ritter said in an interview, explaining that the approach focuses on breaking down clinical procedures into tasks for which mastery can be objectively defined. The mastery learning for any one task is complete when performance criteria are met rather than after any specific number of repetitions.
“There is substantial evidence that this approach improves skill acquisition and improves outcome,” Dr. Ritter reported.
Identifying problems more quickly
To reduce the risks and complications of new devices, various strategies for postmarketing surveillance are being pursued simultaneously, according to Dana Telem, MD, MPH, director of the Michigan Comprehensive Hernia Program and an associate professor of surgery at the University of Michigan, Ann Arbor. As has been demonstrated repeatedly, the premarket testing and regulatory approval process does not rule out risk of unforeseen complications, including serious risks that may ultimately lead to the technology being discarded.
One approach has been the increasing use of registries so that constant capture of patient data allows isolated but recurring events to become rapidly identifiable, but Dr. Telem said that the value of these registries can be increased “by encouraging and empowering patients to report issues when they occur” to improve capture of adverse events.
As an example of a strategy to more rapidly drill down to the cause of complications, Dr. Telem reported on an effort to encourage creation of unique device identifiers for a given device. As devices are commonly updated and modified, the serial number may be useful when trying to determine whether the issue can be linked to a specific design change. More importantly, these numbers would be identifiable in billing data and potentially linked to registries to improve real time identification of adverse event patterns.
“The problem with adding new layers of premarketing regulation is the risk of stifling innovation, but we have not done a great job of postmarketing surveillance, and I think the focus now being placed on better strategies will greatly improve patient safety,” Dr. Telem reported.
Adding more layers of review and testing prior to approval of a new technology does not necessarily identify all risks, because not all complications are foreseeable, Dr. Telem explained.
“The long-term monitoring of new devices and technologies is mission critical if we really hope to safely innovate in the future.” Dr. Telem said.
Improving learning through telementoring
Employing avenues of telecommunication to better mentor clinicians acquiring new skills was the third example of an innovative area of improving quality assurance. A large focus of the presentation by Christopher Schlachta, MD, professor of surgery, Schulich School of Medicine and Dentistry, Western University, London, Ont., was on telementoring. He explained that this concept is not new, but it is becoming increasingly sophisticated, and there is a growing body of objective evidence that it is effective.
The Internet expands the possibilities. Remote screens can accommodate multiple images, including, in the case of surgical procedures, the surgical field and situational cameras that provide one or more views of the surgical team as they proceed. For laparoscopy, the information being fed to the mentor is often even more comprehensive.
“So much of what we do now is computer assisted, so the chip on the end of scope, for example, is capturing images digitally. These data can be transmitted and translated into images essentially simultaneously for the operator and a mentor who could be hundreds of miles away,” Dr. Schlachta reported.
There are now “plenty of studies that telementoring is effective in a variety of different surgical procedures,” Dr. Schlachta added. While the data confirming the value of telementoring for teaching skills in endoscopic procedures are fewer, Dr. Schlachta predicted this will soon change. He noted that the Society of American Gastrointestinal and Endoscopic Surgeons (SAGES) recently created a telementoring task force.
Reviewing several do-it-yourself and proprietary systems that have been developed for telemonitoring, Dr. Schlachta acknowledged that is it not always clear who should pay for this form of learning. He noted that there is potential value for mentees, hospitals wishing to expand services, and health systems attempting to improve quality outcomes. So far, industry has been the primary source of funds.
“For a company introducing a new technology, there is a risk that the technology will fail rapidly if there are bad results in the early going, making it valuable for industry to invest in these telementoring programs,” Dr. Schlacta explained.
“There are related technologies, such as those involving the use of tablets, that are also likely to contribute to the ongoing evolution in how procedural skills are taught and validated. I think these will accelerate and improve learning with the very important potential of better patient outcomes,” Dr. Schlachta reported.
BOSTON – Unforeseen complications and steep learning curves represent the downside of new devices and new procedures, but there are some strategies to minimize these risks, according to experts outlining three such approaches during a session on quality outcomes at the 2017 AGA Tech Summit.
One approach involves a reorientation in skill acquisition in health care delivery. Another involves novel strategies to identify problems with new devices more rapidly. The third involves an ongoing evolution in mentoring clinicians through telecommunication.
Mastering new skills
The strategy described for equipping clinicians with the skills to produce uniform, reproducible outcomes is a technique referred to as simulation-based mastery learning, explained E. Matthew Ritter, MD, vice chairman for education and program director of the general surgery residency program at Walter Reed National Military Medical Center’s Uniformed Services University, Bethesda, Md. The technique is based on the goal of eliciting a “specific level of performance without regard to a time-based approach that is the typical standard in learning processes.”
“More and more evidence is available that performing something a lot of times does not necessarily correlate with performing something well,” Dr. Ritter said. He proposed that direct measurements of skill acquisition address this problem.
“My work has been primarily in laparoscopic surgery, but this approach is not specialty-specific,” Dr. Ritter said in an interview, explaining that the approach focuses on breaking down clinical procedures into tasks for which mastery can be objectively defined. The mastery learning for any one task is complete when performance criteria are met rather than after any specific number of repetitions.
“There is substantial evidence that this approach improves skill acquisition and improves outcome,” Dr. Ritter reported.
Identifying problems more quickly
To reduce the risks and complications of new devices, various strategies for postmarketing surveillance are being pursued simultaneously, according to Dana Telem, MD, MPH, director of the Michigan Comprehensive Hernia Program and an associate professor of surgery at the University of Michigan, Ann Arbor. As has been demonstrated repeatedly, the premarket testing and regulatory approval process does not rule out risk of unforeseen complications, including serious risks that may ultimately lead to the technology being discarded.
One approach has been the increasing use of registries so that constant capture of patient data allows isolated but recurring events to become rapidly identifiable, but Dr. Telem said that the value of these registries can be increased “by encouraging and empowering patients to report issues when they occur” to improve capture of adverse events.
As an example of a strategy to more rapidly drill down to the cause of complications, Dr. Telem reported on an effort to encourage creation of unique device identifiers for a given device. As devices are commonly updated and modified, the serial number may be useful when trying to determine whether the issue can be linked to a specific design change. More importantly, these numbers would be identifiable in billing data and potentially linked to registries to improve real time identification of adverse event patterns.
“The problem with adding new layers of premarketing regulation is the risk of stifling innovation, but we have not done a great job of postmarketing surveillance, and I think the focus now being placed on better strategies will greatly improve patient safety,” Dr. Telem reported.
Adding more layers of review and testing prior to approval of a new technology does not necessarily identify all risks, because not all complications are foreseeable, Dr. Telem explained.
“The long-term monitoring of new devices and technologies is mission critical if we really hope to safely innovate in the future.” Dr. Telem said.
Improving learning through telementoring
Employing avenues of telecommunication to better mentor clinicians acquiring new skills was the third example of an innovative area of improving quality assurance. A large focus of the presentation by Christopher Schlachta, MD, professor of surgery, Schulich School of Medicine and Dentistry, Western University, London, Ont., was on telementoring. He explained that this concept is not new, but it is becoming increasingly sophisticated, and there is a growing body of objective evidence that it is effective.
The Internet expands the possibilities. Remote screens can accommodate multiple images, including, in the case of surgical procedures, the surgical field and situational cameras that provide one or more views of the surgical team as they proceed. For laparoscopy, the information being fed to the mentor is often even more comprehensive.
“So much of what we do now is computer assisted, so the chip on the end of scope, for example, is capturing images digitally. These data can be transmitted and translated into images essentially simultaneously for the operator and a mentor who could be hundreds of miles away,” Dr. Schlachta reported.
There are now “plenty of studies that telementoring is effective in a variety of different surgical procedures,” Dr. Schlachta added. While the data confirming the value of telementoring for teaching skills in endoscopic procedures are fewer, Dr. Schlachta predicted this will soon change. He noted that the Society of American Gastrointestinal and Endoscopic Surgeons (SAGES) recently created a telementoring task force.
Reviewing several do-it-yourself and proprietary systems that have been developed for telemonitoring, Dr. Schlachta acknowledged that is it not always clear who should pay for this form of learning. He noted that there is potential value for mentees, hospitals wishing to expand services, and health systems attempting to improve quality outcomes. So far, industry has been the primary source of funds.
“For a company introducing a new technology, there is a risk that the technology will fail rapidly if there are bad results in the early going, making it valuable for industry to invest in these telementoring programs,” Dr. Schlacta explained.
“There are related technologies, such as those involving the use of tablets, that are also likely to contribute to the ongoing evolution in how procedural skills are taught and validated. I think these will accelerate and improve learning with the very important potential of better patient outcomes,” Dr. Schlachta reported.
FROM THE 2017 AGA TECH SUMMIT
VIDEO: Awardee's work targets mucosal stem cells in Crohn’s, ulcerative colitis
BOSTON – A series of important technological advances in cloning stem cells of the gastrointestinal tract is rewriting the basic concepts surrounding these cells and has the potential to advance understanding of GI conditions such as Barrett’s esophagus and Crohn’s disease, according to the winner of the 2016 AGA-Medtronic Research and Development Pilot Award in Technology.
The award is sponsored by Medtronic and administered through the AGA Research Foundation. “Medtronic is pleased to partner with the AGA Research Foundation in supporting research to improve patient outcomes,” said Vafa Jamali, president of Early Technologies’ business with Medtronic. “We are dedicated to support patient-friendly innovations enabling the early detection and treatment of chronic GI diseases and cancers.”
“The enabling technology we developed is the means of cloning and propagating single mucosal stem cells from discrete regions of the gastrointestinal tract in their most immature form. What we have shown is that these stem cells from normal individuals are essentially immortal, are genetically stable, and, critically, are rigorously regiospecific,” Dr. Xian of the Institute of Molecular Medicine for the Prevention of Human Diseases, University of Texas Health Science Center, Houston, said in an interview.
“This regiospecificity – duodenum stem cells always yield duodenum epithelia, ascending colon stem cells produce ascending colon epithelia – has important implications for both regenerative medicine and drug discovery,” she added.
With regard to the dominant stem cell technologies in use today, including induced pluripotent stem cells and organoid cultures of the gastrointestinal tract, Dr. Xian’s technology represents a paradigm shift.
Dr. Xian’s system generates pure “ground state” stem cells that differentiate in 3-D culture to an epithelium indistinguishable from textbook images of in situ epithelia, at rates 250 times faster than intestinal organoid systems.
This system is being applied in major investigations into Barrett’s esophagus and inflammatory bowel disease in collaboration with investigators at multiple centers in the United States and Singapore.
For Barrett’s, Dr. Xian has developed patient-matched stem cells from endoscopic biopsies of esophageal, Barrett’s, and gastric cardia to show that each arises from distinct lineages, and that the Barrett’s stem cells accumulate genetic alterations not found in those of the matched normal epithelia. These stem cells have been adapted to a drug-screening platform to identify leads that specifically eliminate Barrett’s with an eye on preemptive therapies.
Her work on mucosal stem cells in patients with Crohn’s and ulcerative colitis is revealing a coexistence of normal and pathogenic stem cells in these patients that may alter concepts of both the etiology and treatment of these conditions.
Dr. Xian suggests that treatment of Crohn’s may become analogous to treatment of cancer – emphasizing preservation of healthy cells while eliminating diseased cells – and move therapy from the current effort to induce a quiescent inflammatory state to one in which the goal is cure.
“These findings appear to indicate that some of the chronic inflammatory conditions so prevalent in gastroenterology, as well as the cancers arising from them, may have as their basis profound alterations in mucosal stem cell biology,” she added.
Though it’s still early days with this technology, its single-cell resolution naturally melds with other emerging genomic and genome editing technologies into a powerful means of dissecting the molecular basis of disease and realizing the potential of autologous regenerative medicine, according to Dr. Xian.
Following Dr. Xian’s presentation, Michael L. Kochman, MD, who is executive committee chair of the AGA Center for GI Innovation and Technology, announced the winner of the 2017 AGA-Medtronic Research and Development Pilot Award in Technology. The award was presented to Bani Chander Roland, MD, of Lenox Hill Hospital & Northwell Health System, New York. Her research will involve assessment of the ileocecal valve.
“She has several aims in this proposal to really understand better the causes of ileosphincter dysfunction using a novel method of measuring ileocecal junction pressures,” reported Dr. Kochman. He explained that other parts of the proposal included ileocecal evaluation with wireless capsule endoscopy as well as an evaluation of flora across the GI tract.
A new award to foster innovation in GI medtech is also planned. In partnership with Boston Scientific, the AGA has established the AGA-Boston Scientific Technology and Innovation Pilot Award. This $30,000 research grant will support investigators at any career stage who are developing and testing new medical devices and technologies with applications in gastroenterology and hepatology. The details of the award will be available on the AGA’s website in May. Applications for the award will be accepted beginning in the fall.
BOSTON – A series of important technological advances in cloning stem cells of the gastrointestinal tract is rewriting the basic concepts surrounding these cells and has the potential to advance understanding of GI conditions such as Barrett’s esophagus and Crohn’s disease, according to the winner of the 2016 AGA-Medtronic Research and Development Pilot Award in Technology.
The award is sponsored by Medtronic and administered through the AGA Research Foundation. “Medtronic is pleased to partner with the AGA Research Foundation in supporting research to improve patient outcomes,” said Vafa Jamali, president of Early Technologies’ business with Medtronic. “We are dedicated to support patient-friendly innovations enabling the early detection and treatment of chronic GI diseases and cancers.”
“The enabling technology we developed is the means of cloning and propagating single mucosal stem cells from discrete regions of the gastrointestinal tract in their most immature form. What we have shown is that these stem cells from normal individuals are essentially immortal, are genetically stable, and, critically, are rigorously regiospecific,” Dr. Xian of the Institute of Molecular Medicine for the Prevention of Human Diseases, University of Texas Health Science Center, Houston, said in an interview.
“This regiospecificity – duodenum stem cells always yield duodenum epithelia, ascending colon stem cells produce ascending colon epithelia – has important implications for both regenerative medicine and drug discovery,” she added.
With regard to the dominant stem cell technologies in use today, including induced pluripotent stem cells and organoid cultures of the gastrointestinal tract, Dr. Xian’s technology represents a paradigm shift.
Dr. Xian’s system generates pure “ground state” stem cells that differentiate in 3-D culture to an epithelium indistinguishable from textbook images of in situ epithelia, at rates 250 times faster than intestinal organoid systems.
This system is being applied in major investigations into Barrett’s esophagus and inflammatory bowel disease in collaboration with investigators at multiple centers in the United States and Singapore.
For Barrett’s, Dr. Xian has developed patient-matched stem cells from endoscopic biopsies of esophageal, Barrett’s, and gastric cardia to show that each arises from distinct lineages, and that the Barrett’s stem cells accumulate genetic alterations not found in those of the matched normal epithelia. These stem cells have been adapted to a drug-screening platform to identify leads that specifically eliminate Barrett’s with an eye on preemptive therapies.
Her work on mucosal stem cells in patients with Crohn’s and ulcerative colitis is revealing a coexistence of normal and pathogenic stem cells in these patients that may alter concepts of both the etiology and treatment of these conditions.
Dr. Xian suggests that treatment of Crohn’s may become analogous to treatment of cancer – emphasizing preservation of healthy cells while eliminating diseased cells – and move therapy from the current effort to induce a quiescent inflammatory state to one in which the goal is cure.
“These findings appear to indicate that some of the chronic inflammatory conditions so prevalent in gastroenterology, as well as the cancers arising from them, may have as their basis profound alterations in mucosal stem cell biology,” she added.
Though it’s still early days with this technology, its single-cell resolution naturally melds with other emerging genomic and genome editing technologies into a powerful means of dissecting the molecular basis of disease and realizing the potential of autologous regenerative medicine, according to Dr. Xian.
Following Dr. Xian’s presentation, Michael L. Kochman, MD, who is executive committee chair of the AGA Center for GI Innovation and Technology, announced the winner of the 2017 AGA-Medtronic Research and Development Pilot Award in Technology. The award was presented to Bani Chander Roland, MD, of Lenox Hill Hospital & Northwell Health System, New York. Her research will involve assessment of the ileocecal valve.
“She has several aims in this proposal to really understand better the causes of ileosphincter dysfunction using a novel method of measuring ileocecal junction pressures,” reported Dr. Kochman. He explained that other parts of the proposal included ileocecal evaluation with wireless capsule endoscopy as well as an evaluation of flora across the GI tract.
A new award to foster innovation in GI medtech is also planned. In partnership with Boston Scientific, the AGA has established the AGA-Boston Scientific Technology and Innovation Pilot Award. This $30,000 research grant will support investigators at any career stage who are developing and testing new medical devices and technologies with applications in gastroenterology and hepatology. The details of the award will be available on the AGA’s website in May. Applications for the award will be accepted beginning in the fall.
BOSTON – A series of important technological advances in cloning stem cells of the gastrointestinal tract is rewriting the basic concepts surrounding these cells and has the potential to advance understanding of GI conditions such as Barrett’s esophagus and Crohn’s disease, according to the winner of the 2016 AGA-Medtronic Research and Development Pilot Award in Technology.
The award is sponsored by Medtronic and administered through the AGA Research Foundation. “Medtronic is pleased to partner with the AGA Research Foundation in supporting research to improve patient outcomes,” said Vafa Jamali, president of Early Technologies’ business with Medtronic. “We are dedicated to support patient-friendly innovations enabling the early detection and treatment of chronic GI diseases and cancers.”
“The enabling technology we developed is the means of cloning and propagating single mucosal stem cells from discrete regions of the gastrointestinal tract in their most immature form. What we have shown is that these stem cells from normal individuals are essentially immortal, are genetically stable, and, critically, are rigorously regiospecific,” Dr. Xian of the Institute of Molecular Medicine for the Prevention of Human Diseases, University of Texas Health Science Center, Houston, said in an interview.
“This regiospecificity – duodenum stem cells always yield duodenum epithelia, ascending colon stem cells produce ascending colon epithelia – has important implications for both regenerative medicine and drug discovery,” she added.
With regard to the dominant stem cell technologies in use today, including induced pluripotent stem cells and organoid cultures of the gastrointestinal tract, Dr. Xian’s technology represents a paradigm shift.
Dr. Xian’s system generates pure “ground state” stem cells that differentiate in 3-D culture to an epithelium indistinguishable from textbook images of in situ epithelia, at rates 250 times faster than intestinal organoid systems.
This system is being applied in major investigations into Barrett’s esophagus and inflammatory bowel disease in collaboration with investigators at multiple centers in the United States and Singapore.
For Barrett’s, Dr. Xian has developed patient-matched stem cells from endoscopic biopsies of esophageal, Barrett’s, and gastric cardia to show that each arises from distinct lineages, and that the Barrett’s stem cells accumulate genetic alterations not found in those of the matched normal epithelia. These stem cells have been adapted to a drug-screening platform to identify leads that specifically eliminate Barrett’s with an eye on preemptive therapies.
Her work on mucosal stem cells in patients with Crohn’s and ulcerative colitis is revealing a coexistence of normal and pathogenic stem cells in these patients that may alter concepts of both the etiology and treatment of these conditions.
Dr. Xian suggests that treatment of Crohn’s may become analogous to treatment of cancer – emphasizing preservation of healthy cells while eliminating diseased cells – and move therapy from the current effort to induce a quiescent inflammatory state to one in which the goal is cure.
“These findings appear to indicate that some of the chronic inflammatory conditions so prevalent in gastroenterology, as well as the cancers arising from them, may have as their basis profound alterations in mucosal stem cell biology,” she added.
Though it’s still early days with this technology, its single-cell resolution naturally melds with other emerging genomic and genome editing technologies into a powerful means of dissecting the molecular basis of disease and realizing the potential of autologous regenerative medicine, according to Dr. Xian.
Following Dr. Xian’s presentation, Michael L. Kochman, MD, who is executive committee chair of the AGA Center for GI Innovation and Technology, announced the winner of the 2017 AGA-Medtronic Research and Development Pilot Award in Technology. The award was presented to Bani Chander Roland, MD, of Lenox Hill Hospital & Northwell Health System, New York. Her research will involve assessment of the ileocecal valve.
“She has several aims in this proposal to really understand better the causes of ileosphincter dysfunction using a novel method of measuring ileocecal junction pressures,” reported Dr. Kochman. He explained that other parts of the proposal included ileocecal evaluation with wireless capsule endoscopy as well as an evaluation of flora across the GI tract.
A new award to foster innovation in GI medtech is also planned. In partnership with Boston Scientific, the AGA has established the AGA-Boston Scientific Technology and Innovation Pilot Award. This $30,000 research grant will support investigators at any career stage who are developing and testing new medical devices and technologies with applications in gastroenterology and hepatology. The details of the award will be available on the AGA’s website in May. Applications for the award will be accepted beginning in the fall.
FROM THE AGA 2017 TECH SUMMIT
Debulking called reasonable for unresectable liver cancer
MIAMI BEACH – Cytoreductive debulking surgery for neuroendocrine liver metastases provides a lower but “reasonable” long-term survival, compared with curative intent surgery, according to results of a study presented at the annual meeting of the Americas Hepatico-Pancreato-Biliary Association.
Liver debulking for patients with grossly unresectable disease is therefore an option that may extend survival, the study researchers said.
“Surgical resection offers the best chance for long-term survival but may not be technically feasible for all our patients,” said Fabio Bagante, MD, a resident at the University of Verona (Italy). “Debulking neuroendocrine liver metastases has been proposed as an alternative.”
Dr. Bagante and coinvestigators compared outcomes between 180 patients who had debulking surgery (defined as an R2 outcome) and 449 who underwent curative intent resection (R0 or R1). Patients had surgery between 1990 and 2015 at eight institutions, and the primary outcome was overall survival. The mean follow-up was 51 months; during that time, 174 patients died.
The 5-year overall survival was 72% in the debulking group and 89% in the curative intent group. The 10-year overall survival was 41% in the debulking group and 77% among the curative intent surgery patients.
“Debulking operations for neuroendocrine liver metastases provide lower but reasonable long-term survival, compared with patients who underwent curative intent,” Dr. Bagante said.
“Nearly 3 in 10 patients underwent debulking surgery,” Dr. Bagante said. “It was more common among the elderly, males with symptomatic disease, and patients with greater liver involvement.”
Patients in the liver-debulking groups were also significantly more likely to have high–tumor grade disease, 35%, versus 13% of the curative intent resection group (P less than .001). They were also more likely to have metastatic disease, with an N1 rate of 73% versus 52% (P less than .001).
“Great work. Thanks for presenting this topic. It’s quite a debated topic,” said study discussant Aaron Lee, DO, of the department of general surgery at Cleveland Clinic Florida in Weston. “I looked at your data on asymptomatic patients who had a 5-year survival of 60%; U.K. data show [the] same survival without any intervention.” He asked Dr. Bagante to comment on operating on patients without any symptoms.
“Regarding the role of symptoms as a predictor of survival, there are many papers in the literature debating what are the true predictors of survival,” Dr. Bagante noted. “We don’t think [symptoms have an] impact; our data do not demonstrate that. We think the biologic behavior of the disease, the impact of the progression of the disease, and the grade of the tumor, [indicated by] Ki-67, could have more of a role.”
MIAMI BEACH – Cytoreductive debulking surgery for neuroendocrine liver metastases provides a lower but “reasonable” long-term survival, compared with curative intent surgery, according to results of a study presented at the annual meeting of the Americas Hepatico-Pancreato-Biliary Association.
Liver debulking for patients with grossly unresectable disease is therefore an option that may extend survival, the study researchers said.
“Surgical resection offers the best chance for long-term survival but may not be technically feasible for all our patients,” said Fabio Bagante, MD, a resident at the University of Verona (Italy). “Debulking neuroendocrine liver metastases has been proposed as an alternative.”
Dr. Bagante and coinvestigators compared outcomes between 180 patients who had debulking surgery (defined as an R2 outcome) and 449 who underwent curative intent resection (R0 or R1). Patients had surgery between 1990 and 2015 at eight institutions, and the primary outcome was overall survival. The mean follow-up was 51 months; during that time, 174 patients died.
The 5-year overall survival was 72% in the debulking group and 89% in the curative intent group. The 10-year overall survival was 41% in the debulking group and 77% among the curative intent surgery patients.
“Debulking operations for neuroendocrine liver metastases provide lower but reasonable long-term survival, compared with patients who underwent curative intent,” Dr. Bagante said.
“Nearly 3 in 10 patients underwent debulking surgery,” Dr. Bagante said. “It was more common among the elderly, males with symptomatic disease, and patients with greater liver involvement.”
Patients in the liver-debulking groups were also significantly more likely to have high–tumor grade disease, 35%, versus 13% of the curative intent resection group (P less than .001). They were also more likely to have metastatic disease, with an N1 rate of 73% versus 52% (P less than .001).
“Great work. Thanks for presenting this topic. It’s quite a debated topic,” said study discussant Aaron Lee, DO, of the department of general surgery at Cleveland Clinic Florida in Weston. “I looked at your data on asymptomatic patients who had a 5-year survival of 60%; U.K. data show [the] same survival without any intervention.” He asked Dr. Bagante to comment on operating on patients without any symptoms.
“Regarding the role of symptoms as a predictor of survival, there are many papers in the literature debating what are the true predictors of survival,” Dr. Bagante noted. “We don’t think [symptoms have an] impact; our data do not demonstrate that. We think the biologic behavior of the disease, the impact of the progression of the disease, and the grade of the tumor, [indicated by] Ki-67, could have more of a role.”
MIAMI BEACH – Cytoreductive debulking surgery for neuroendocrine liver metastases provides a lower but “reasonable” long-term survival, compared with curative intent surgery, according to results of a study presented at the annual meeting of the Americas Hepatico-Pancreato-Biliary Association.
Liver debulking for patients with grossly unresectable disease is therefore an option that may extend survival, the study researchers said.
“Surgical resection offers the best chance for long-term survival but may not be technically feasible for all our patients,” said Fabio Bagante, MD, a resident at the University of Verona (Italy). “Debulking neuroendocrine liver metastases has been proposed as an alternative.”
Dr. Bagante and coinvestigators compared outcomes between 180 patients who had debulking surgery (defined as an R2 outcome) and 449 who underwent curative intent resection (R0 or R1). Patients had surgery between 1990 and 2015 at eight institutions, and the primary outcome was overall survival. The mean follow-up was 51 months; during that time, 174 patients died.
The 5-year overall survival was 72% in the debulking group and 89% in the curative intent group. The 10-year overall survival was 41% in the debulking group and 77% among the curative intent surgery patients.
“Debulking operations for neuroendocrine liver metastases provide lower but reasonable long-term survival, compared with patients who underwent curative intent,” Dr. Bagante said.
“Nearly 3 in 10 patients underwent debulking surgery,” Dr. Bagante said. “It was more common among the elderly, males with symptomatic disease, and patients with greater liver involvement.”
Patients in the liver-debulking groups were also significantly more likely to have high–tumor grade disease, 35%, versus 13% of the curative intent resection group (P less than .001). They were also more likely to have metastatic disease, with an N1 rate of 73% versus 52% (P less than .001).
“Great work. Thanks for presenting this topic. It’s quite a debated topic,” said study discussant Aaron Lee, DO, of the department of general surgery at Cleveland Clinic Florida in Weston. “I looked at your data on asymptomatic patients who had a 5-year survival of 60%; U.K. data show [the] same survival without any intervention.” He asked Dr. Bagante to comment on operating on patients without any symptoms.
“Regarding the role of symptoms as a predictor of survival, there are many papers in the literature debating what are the true predictors of survival,” Dr. Bagante noted. “We don’t think [symptoms have an] impact; our data do not demonstrate that. We think the biologic behavior of the disease, the impact of the progression of the disease, and the grade of the tumor, [indicated by] Ki-67, could have more of a role.”
AT AHPBA 2017
Key clinical point:
Major finding: The 10-year overall survival was 77% in the curative intent surgery group, compared with 41% among the debulking patients.
Data source: A retrospective database study of 629 people with neuroendocrine liver metastases.
Disclosures: Dr. Bagante and Dr. Lee had no relevant financial disclosures.