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Boston magazine has named a number of Dana-Farber/Boston Children’s Cancer and Blood Disorders Center faculty members to its annual "Top Doctors" guide.
Drawing from a Castle Connolly Medical Ltd. physician database, the Boston magazine Top Doctors™ list includes hundreds of Boston-area physicians from many medical specialties and is also available on Boston magazine's website.
The following Dana-Farber/Boston Children’s faculty members are listed as "Top Doctors" in the January 2022 issue of Boston magazine:
Dana-Farber/Boston Children’s Cancer and Blood Disorders Center was ranked as one of 15 transplant centers in the country that performed above the expected survival rate, according to a new report. The report, compiled by the Center for International Blood and Marrow Transplant Research (CIBMTR), analyzes outcomes for recipients of allogeneic (unrelated, matched donor) hematopoietic stem cell transplant by measuring one-year overall survival.
"Because centers vary considerably in the risk level of cases treated, a statistical model was developed to adjust for several risk factors known or suspected to influence outcome," according to a statement from the CIBMTR.
The report generated from this analysis is available to the public and is intended to provide transplant recipients and their families with the latest survival rate information for centers performing transplants across the country.
With the ability to score only one of three ratings — a -1, 0 or the highly-sought-after 1 rating — the vast majority of transplant centers score a 0, indicating that their patient survival rate met expectations.
This year, fewer than 9% of programs achieved a 1.
"I'm very proud that the 2021 Transplant Center-Specific Survival Report published by CIBMTR has determined that our program has exceeded the expected survival rate for transplant patients,” says Leslie S. Kean, MD, PhD, director of the Stem Cell Transplant Center at Dana-Farber/Boston Children’s.
With a score of 1, Dana-Farber/Boston Children's is one of only four pediatric stem cell transplant centers in the U.S. to achieve this rating for 2021.
“This distinction reflects the exceptional care that the entire hematopoietic stem cell transplant team provides our patients, the outstanding facilities available to them at Dana-Farber/Boston Children’s, and the state-of-the art clinical trials that are led by members of our team, designed to make transplant safer and more effective for the patients we treat,” says Dr. Kean.
In December 2021, the FDA approved the first drug to prevent acute graft-versus-host disease (GVHD) based on results from a study led by Dr. Kean. According to Dr. Kean, this approval will help to make transplants safer for more patients.
This score – achieved by a small minority of programs – reflects the incredible multidisciplinary care provided to our patients and families,” says Leslie E. Lehmann, MD, clinical director of the Stem Cell Transplant Center. “Physicians, nurses, nursing assistants, pharmacists, child life specialists and many others give their utmost every day to create a medically excellent, safe and nurturing environment that optimizes the process of recovery.”
Dana-Farber/Boston Children’s Cancer and Blood Disorders Center was ranked as one of 15 transplant centers in the country that performed above the expected survival rate
Award honors transformative contributions to research impacting human health
The Gairdner Foundation has announced that Stuart H. Orkin, MD, is one of the 2022 Canada Gairdner Award laureates for seminal discoveries and contributions to biomedical science.
Orkin, the David G. Nathan Distinguished Professor of Pediatrics at Dana-Farber/Boston Children’s Cancer and Blood Disorders Center and Harvard Medical School, and a Howard Hughes Medical Institute Investigator, was recognized for the discovery of the molecular mechanism responsible for the switch from fetal to adult hemoglobin gene expression during human development and translating that knowledge into a novel treatment for the hemoglobin disorders -- sickle cell disease and beta-thalassemia.
The Gairdner Foundation was established in 1957 by Toronto stockbroker, James Gairdner to award annual prizes to scientists whose discoveries have had major impact on scientific progress and on human health. Since 1959 when the first awards were granted, 402 scientists have received a Canada Gairdner Award and 96 to date have gone on to receive the Nobel Prize. The Canada Gairdner Awards promote a stronger culture of research and innovation across the country through outreach programs including lectures and research symposia.
Orkin’s pioneering work in genetic disorders of hemoglobin spans four decades and has unraveled molecular mysteries behind how blood cells develop and how disorders of blood arise. His most recent studies led to the discovery of the molecular mechanism responsible for the switch from fetal (HbF) to adult (HbA) hemoglobin gene expression that occurs during human development. Capitalizing on genetic clues from human population studies, Orkin and colleagues established that the protein BCL11A acts as the critical silencer of HbF expression in adults. Recognizing that turning HbF expression back on could lessen disease severity of sickle cell disease and beta-thalassemia -- genetic disorders affecting HbA production – he proposed downregulation of BCL11A as a therapeutic approach. Dialing down the amount of BCL11A would reactivate HbF expression and effectively substitute for mutant or deficient HbA in these disorders. His group first demonstrated that downregulation of BCL11A expression corrects sickle cell disease in engineered mice, an important proof-of-principle for therapeutic translation. He and his colleagues identified a discrete site in a regulatory element within the BCL11A gene itself that, if deleted by CRISPR gene editing in blood stem cells, would impair BCL11A expression only within developing red blood cells, and safely reactivate HbF expression. This work laid the groundwork for highly promising, ongoing clinical trials in patients with sickle cell disease and beta-thalassemia, diseases that affect >5 million individuals worldwide. Reactivation of HbF in patients in these genetic therapy trials has yielded transformative results: freedom from sickle crises and anemia in sickle cell disease and transfusion-independence in beta-thalassemia.
His work has had a tremendous impact and much of what is known about the control of gene expression during blood cell development can be traced directly to Orkin’s pioneering studies. His discoveries have paved the way for clinical approaches that will revolutionize the treatment of hemoglobin disorders – sickle cell disease and beta-thalassemia – that affect more than five million people worldwide. Clinical trials that are currently underway establish the therapeutic potential of HbF reactivation. The outcomes of these trials will have significant impact for patients suffering from hemoglobin disorders around the globe, and will encourage the future development of cheaper and more readily accessible therapies for global application.
More information about the other award winners is available at www.gairdner.org
Children with high-risk neuroblastoma had worse outcomes if they were from certain racial/ethnic groups or were on public rather than private insurance, despite being treated in clinical trials with standardized protocols, according to a study led by investigators from Dana-Farber/Boston Children’s Cancer and Blood Disorders Center.
The study shows that young patients from historically marginalized populations or from lower-income backgrounds had poorer five-year survival rates even when they were assigned to receive uniform initial treatment after diagnosis with high-risk neuroblastoma.
“These findings recapitulate what we have known for decades at the population level—children from historically marginalized groups are less likely to survive their cancer,” said Puja J. Umaretiya, MD, a clinical fellow in pediatric hematology/oncology at Dana-Farber/Boston Children’s. “They add an essential next layer to our understanding of racial and ethnic disparities in childhood cancer, and that is that enrollment in clinical trials is not enough to achieve racial and ethnic equity in survival.” Umaretiya is presenting the study results at the American Society of Clinical Oncology (ASCO) Annual meeting, being held June 3-7, 2022, and the study is included in the ASCO press program.
“Clinical trials represent highly standardized care – yet even when receiving care on clinical trials, children with high-risk neuroblastoma do not experience the same outcomes based on their race, ethnicity, and whether they live in poverty,” said Umaretiya, lead author of the study. “This is key, because thus far attention has been paid to getting historically marginalized groups to trials with the assumption that this will reduce survival disparities, but our data suggest that in pediatrics, trial-enrollment is a first step, but clearly not a sufficient one.”
Senior author is Kira Bona, MD, MPH, a pediatric oncologist at Dana-Farber/Boston Children’s with research focused on identifying poverty-associated outcome disparities in childhood cancer and developing interventions to mitigate those disparities.
Bona notes, “That stark racial/ethnic disparities in survival persist despite clinical trial participation makes it crystal clear that pediatric oncology trials must incorporate health equity interventions. If a new gene mutation were found to increase risk for trial-enrolled patients, pediatric oncology would not hesitate to begin intervening. That same urgency must apply to these data. It is imperative that pediatric oncologists begin to test healthcare delivery and supportive care interventions in our trials just like we do new drugs.”
The study looked at outcomes in 696 children enrolled in three Children’s Oncology Group (COG) clinical trials of treatment for high-risk neuroblastoma. Neuroblastoma is a type of cancer that forms in nerve tissue. It frequently begins in one of the adrenal glands but can also originate in the neck, chest, abdomen, or spine.
High-risk disease is defined by age, how widely the disease has spread, and biologic characteristics of the cancer cells. The prognosis for long-term survival remains challenging. Treatment is usually an intensive combination of chemotherapy, surgery, stem cell transplantation, radiation, and immunotherapy.
Of the 696 patients in the COG trials, 11% were Hispanic, 16% were Black non-Hispanic, 4% were other non-Hispanic, and 69% were white non-Hispanic. One-third of the children were household poverty-exposed (covered by public insurance); 26% were exposed to neighborhood level poverty (living in a high-poverty ZIP code defined by 20% or more of the population living below the federal poverty line).
The five-year overall survival rate varied by race/ethnicity (47% for Hispanic children; 50% for other non-Hispanic children; 61% for white non-Hispanic children; and 63% for Black non-Hispanic children.) After adjusting for disease-associated factors, Hispanic children were 1.8 times more likely to die and other non-Hispanic patients were 1.5 times more likely to die than white non-Hispanic children.
Patients who had only public insurance (a proxy for household poverty) had a 53% five-year survival rate compared to 63% for others. The survival rate was also lower – 54% – in children living in neighborhood level poverty compared with 62% for others.
“A huge strength of the way that this dataset was created is that we have the ability to look at potential mechanisms that may explain these survival disparities,” said Umaretiya. “For the first time, we will be able to ask whether certain groups experienced delays in therapy or were more likely to stop participating in trials perhaps because of competing family needs secondary to poverty. Most importantly, we will be able to start to look at what happens after relapse – a time when we know treatment becomes less standardized, which may increase the chance that racial, ethnic, or socioeconomic privilege helps some families access life-extending therapy for their children while others are less able to. Understanding what happens after relapse will be essential to guiding interventions to improve survival disparities and we are excited to take this on next.”
Children with high-risk neuroblastoma had worse outcomes if they were from certain racial/ethnic groups or were on public rather than private insurance, despite being treated in clinical trials with standardized protocols, according to a study led by investigators from Dana-Farber/Boston Children’s Cancer and Blood Disorders Center.
U.S. News & World Report has named Dana-Farber/Boston Children's Cancer and Blood Disorders Center #1 in the nation in its 2022-23 Best Children's Hospitals report. Dana-Farber/Boston Children's has been recognized as one of the top three pediatric cancer centers in the country each year since the ranking's inception, earning more #1 rankings than any other program.
Boston Children’s Hospital remains the #1 pediatric hospital in the nation for the ninth year in a row in the 2022-23 Best Children’s Hospitals Honor Roll.
“Being recognized as the top pediatric cancer program in the nation by U.S. News & World Report is an honor,” said Scott A. Armstrong, MD, PhD, President of Dana-Farber/Boston Children’s. “This recognition is testament to our outstanding doctors, nurses and staff who work tirelessly providing exceptional clinical care while also conducting world-class research, on behalf of patients and their families.”
“We are proud to continuously be named one of the top pediatric cancer centers in the nation,” said David A. Williams, MD, Chief of the Division of Hematology/Oncology at Boston Children’s Hospital. “Not only do we provide world-class care to children from all over the world, we are also conducting life-changing research to find new treatments for our patients. Our team works tirelessly for our patients and families and this ranking celebrates that.”
Dana-Farber/Boston Children’s brings together two internationally known research and teaching institutions that have provided comprehensive care for pediatric oncology and hematology patients since 1947. The Harvard Medical School affiliates share a clinical staff that delivers inpatient care and surgery at Boston Children’s and most outpatient care at Dana-Farber Cancer Institute.
“Choosing the right hospital for a sick child is a critical decision for many parents,” said Ben Harder, chief of health analysis and managing editor at U.S. News. “The Best Children’s Hospitals rankings spotlight hospitals that excel in specialized care.”
The Best Children's Hospitals methodology factors objective measures such as patient outcomes, including mortality and infection rates, as well as health equity and available clinical resources and compliance with best practices. To calculate the Best Children’s Hospitals rankings, U.S. News gathered relevant data from children’s hospitals in early 2022 and from pediatric physicians and other healthcare organizations as well as available clinical resources, compliance with best practices, and efforts to promote diversity, equity and inclusion among patients and staff in 2022.
U.S. News & World Report has named Dana-Farber/Boston Children's Cancer and Blood Disorders Center #1 in the nation in its 2022-23 Best Children's Hospitals report.
Study results show that genomic profiling of solid tumors in pediatric patients can have significant clinical benefits, including opportunities for targeted therapy.
86% of 345 patients with solid tumors, excluding brain tumors, were found to have genetic alterations in their cancers with potential for impact on care.
Some patients had significant clinical responses when treated with precision drugs matched to genetic changes in their tumors and some patients had their diagnosis clarified as a result of tumor profiling.
Results of a study of molecular tumor profiling in young patients revealed a high rate of genetic alterations with potential for impacting clinical care, including clarifying diagnoses and treatment with matched, precision cancer drugs.
Reporting in Nature Medicine, researchers from Dana-Farber/Boston Children’s Cancer and Blood Disorders Center said molecular profiling of solid tumors found clinically significant genetic variations in 298, or 86% of 345 pediatric patients. In 240 patients, the genetic “fingerprint” or pattern of cancer-related changes in the tumor’s DNA could be used to choose a targeted, precision therapy matched to those alterations. Of these patients, 200 were eligible for matched drug therapy.
Targeted therapies were used to treat 29 patients, and 24% of the patients responded to the targeted drugs or experienced a durable clinical benefit. In addition, the molecular profiling – done by a process called next-generation sequencing – clarified the diagnostic classification in 17 patients.
“By providing a more accurate diagnosis or identifying a targeted therapy, molecular tumor profiling significantly impacts the care we provide. The result is cancer treatment that is more effective and, in some cases, has fewer side effects,” said Katherine A. Janeway, MD, MMSc, Senior Physician, Dana-Farber/Boston Children's Cancer and Blood Disorders Center; Director, Clinical Genomics, Dana-Farber Cancer Institute; and Associate Professor of Pediatrics, Harvard Medical School.
The ongoing study, known as the GAIN/iCat2, is being carried out at 12 centers in the United States to evaluate the clinical impact of genomic sequencing of pediatric solid tumors, which is much less commonly performed than in adult patients with solid tumors. In adults, tumor profiling is recommended in national practice guidelines as an aid to diagnosis and treatment. But there are no such guidelines or insurance coverage decisions for using tumor profiling in pediatric solid malignancies and few clinical trials of tumor profiling include young people with cancer.
Pediatric solid tumors are much rarer than they are in adults, and large studies of genetic alterations are difficult to carry out. In fact, there were 59 different tumor types affecting the patients in this study and some of the cancers are so rare that they affected just one patient. Pediatric tumors also tend to have fewer gene mutations than those in adults, so there are fewer targets for drugs to attack – and fewer drugs available to target them. The net result is that most solid tumors in children are treated with standard chemotherapy or radiation rather than precision drug agents.
The current study is a prospective observational cohort study led by Janeway and Alanna J. Church, MD, associate director for Molecular Pediatric Pathology at Boston Children’s Hospital. Janeway has been working to bring personalized, molecularly targeted treatments to children with cancer. The results of Janeway’s first study, called Individualized Cancer Therapy (iCat), were published in 2016 and showed that bringing clinical genomic sequencing to pediatric oncology practice is feasible. Janeway and colleagues are now conducting a follow-up study, the Genomic Assessment Improves Novel Therapy (GAIN) Consortium, iCat2 study.
The study participants were patients with relapsed/refractory or high-risk non-brain tumors at age 30 or younger; the mean age at diagnosis was 12 years. Researchers performed targeted DNA next-generation sequencing with OncoPanel testing on one or more tumor samples from each patient, with some samples subjected to RNA sequencing as well. Based on the analysis, a comprehensive report is created and sent to the referring physician. This allows the physician to develop a plan for the patient that takes into account the genetic changes detected in their tumors that are associated with matched drugs that have yielded success either in the laboratory or in the clinic in treating patients with such tumors. The study tracks each patient to determine the impact of the treatment plan on the patient’s outcome. The primary goal of the study is to observe if the tumor profiling and matched targeted drug treatment affects overall survival.
Recommendations for molecularly targeted therapy (MTT) based on data from the previous iCat study were returned to 240 of the 345 patients whose tumors had at least one gene variant. Of these, 200 patients were eligible for assessment of receipt and response to MTT, being alive and having complete follow-up data. Ninety-six of the patients would not have been expected to consider targeted therapy because they were receiving frontline therapy, had no cancer-directed systemic therapy during the follow-up period, or no matched targeted drugs were available.
Seven patients who received matched targeted therapy had measurable significant responses to the therapy. In six of those tumors the therapies were matched to a gene fusion. Fusion genes are formed within a cell when a piece of one chromosome breaks off and attaches to another chromosome, causing broken DNA segments to fuse together to form an entirely new gene. Some of these genes produce fusion proteins that can cause cells to grow uncontrollably and form tumors.
“Gene fusions are very important in pediatric tumors,” says Church. “It’s an exciting time because there are so many new drugs that can target these fusions and we have new tests that can reliably detect them.”
Church and Janeway hope their work will help make genomic profiling standard of care for new or relapsed pediatric solid tumors, reimbursable by insurance — just as it is for adults.
“We know there are patients who aren’t getting access to these tests because they are not being reimbursed consistently,” says Church. “We want to broaden access to molecular profiling for every child with a solid tumor.”
This study was funded by: Precision for Kids Pan Mass Challenge Team; 4C’s Fund Lamb Family Fund; C&S Wholesale Grocers and C&S Charities; and Alexandra Simpson Pediatric Research Fund.
Results of a study of molecular tumor profiling in young patients revealed a high rate of genetic alterations with potential for impacting clinical care, including clarifying diagnoses and treatment with matched, precision cancer drugs.
Dana-Farber/Boston Children’s provides advanced treatment for children with solid tumors and cancer. Learn about solid tumor symptoms, diagnosis and treatment.
Dana-Farber/Boston Children’s provides advanced treatment for children with solid tumors and cancer. Learn about solid tumor symptoms, diagnosis and treatment.
Dana-Farber/Boston Children's treats over 100 forms of cancer and blood disorders in children and young adults. Learn more about the conditions we treat.
Dana-Farber/Boston Children's treats over 100 forms of cancer and blood disorders in children and young adults. Learn more about the conditions we treat.