ASH brings together leading experts in classical and malignant blood diseases to share the latest breakthroughs, clinical studies and research impacting the field and patient care. Mass General Cancer Center researchers will cover a wide range of topics, including early phase and investigational therapies, CAR-T cell therapy, bone marrow transplants, advances in treatment of lymphomas, and more.
Below are selected highlights from this year’s top presentations. View a full list of Mass General Cancer Center presentations here.
Psychiatric and Substance Use Disorders are Independent Predictors of Treatment Response and Outcomes in United States Veterans with Newly Diagnosed Acute Myeloid Leukemia Treated with Venetoclax Combinations
When: December 9, 7:45 p.m. ET/4:45 p.m. PT
Who: Michelle Hyunju Lee, MD
What: Venetoclax (VEN) combinations have become the standard frontline therapy for patients with newly diagnosed acute myeloid leukemia (AML) who are deemed unable to tolerate intensive induction chemotherapy, either due to age or comorbidities. However, outcomes in the real-world setting have been inferior to those observed in clinical trials. Preexisting psychiatric diagnoses and substance use disorder (SUD) have previously been associated with increased mortality in patients with cancer, but their prognostic influence in veterans with AML is unclear. The aim of this study was to measure the prevalence of psychiatric and SUDs in veterans with AML treated with VEN combinations, determining the impact of these conditions on treatment response and outcomes. Dr. Lee and team found that psychiatric diagnoses and SUD were not only prevalent among veterans with AML, but they also negatively impacted treatment response, early mortality, and overall survival—independently of age, sociodemographic variables, markers of disease risk, and VEN combination. Veterans with SUD also had significantly higher ICU admissions after treatment initiation for AML. Compared to published clinical trials data, veterans experienced inferior outcomes. Further studies that explore specific factors contributing to health disparities and targeting these comorbidities with supportive care are warranted and may improve outcomes.
Phase 1 Study of CAR-T-ddbcma for the Treatment of Patients with Relapsed and/or Refractory Multiple Myeloma: Results from at Least 1-Year Follow-up in All Patients
When: December 11, 8 p.m. ET/5 p.m. PT
Who: Matthew J. Frigault, MD, MS
What: CAR-T-ddBCMA, an autologous anti-B-cell maturation antigen (BCMA) chimeric antigen receptor (CAR) T-cell therapy with a unique, synthetic binding domain, is being studied in a first-in-human clinical trial in patients with relapsed &/or refractory multiple myeloma (RRMM). Dr. Frigault will present one-year or more clinical data from all patients, which found that adverse events with CAR-T-ddBCMA, including cytokine release syndrome and immune effector cell-associated neurotoxicity syndrome, were manageable, and no off-tumor tissue-targeted toxicity, delayed neurotoxicity, or Parkinsonian-like events were observed in the entire cohort at the time of data-cut. Ongoing efficacy results are encouraging, with 100% tumor objective response rate, including 35 (92%) response of at least a very good partial response and 29 (76%) with complete response. More importantly, clinical responses were durable, with an overall estimated 18-month progression-free survival rate of 67% with comparable clinical responses seen in “high-risk” patients known to have poor prognoses.
Multi-Site Randomized Trial of Inpatient Palliative Care for Hospitalized Patients Undergoing Hematopoietic Stem Cell Transplantation
When: December 11, 5:45 p.m. ET/2:45 p.m. PT
Who: Areej El-Jawahri, MD
What: Patients with hematologic malignancies experience a substantial decline in their quality of life (QOL) and psychological health during their hospitalization for hematopoietic stem cell transplantation (HSCT). Early single center studies established the feasibility and promising preliminary efficacy of integrating palliative care during the HSCT hospitalization. However, data to test the efficacy of this care model across diverse care settings are lacking. In this multi-site randomized clinical trial, Dr. El-Jawahri and team found that inpatient palliative care led to substantial improvements in patients’ QOL, depression and PTSD symptoms, symptom burden, and fatigue during HSCT hospitalization compared to usual care. Integrated palliative care should be considered a new standard of care for patients hospitalized for HSCT.
Phase II Multicenter Trial of Ruxolitinib to Treat Bronchiolitis Obliterans Syndrome after Allogeneic Hematopoietic Cell Transplantation
When: December 11, 1:30 p.m. ET/10:30 a.m. PT
Who: Zachariah DeFilipp, MD
What: Bronchiolitis obliterans syndrome (BOS), occurring after allogeneic HCT, is a pulmonary manifestation of chronic graft versus host disease (cGVHD) that is associated with poor outcomes. Ruxolitinib, an oral selective JAK1/2 inhibitor, is approved for the treatment of recurrent/refractory cGVHD. However, efficacy of ruxolitinib for BOS is not well-characterized. Dr. DeFilipp and team found that ruxolitinib is associated with clinical responses in BOS, which were observed across all severity of disease. Ruxolitinib was associated with few severe adverse events, including severe infections. Most subjects were able to taper or discontinue corticosteroids while on trial treatment. These results support the use of ruxolitinib in the management of BOS after allogeneic HCT.
PATH-HHT, a Double-Blind, Randomized, Placebo-Controlled Trial in Hereditary Hemorrhagic Telangiectasia Demonstrates That Pomalidomide Reduces Epistaxis and Improves Quality of Life
When: December 12, 12 p.m. ET/9 a.m. PT
Who: Hanny Al-Samkari, MD
What: Hereditary hemorrhagic telangiectasia (HHT), the second most common bleeding disorder worldwide, affects 1-in-5,000 people and is characterized by extensive formation of telangiectasia and arteriovenous malformations (AVM) in mucosa, lung, brain, and liver. The primary clinical manifestation is recurrent, often severe nosebleeds, which commonly results in iron deficiency anemia, emergency room visits, hospitalizations, and reduced quality of life (QOL). Dr. Al-Samkari and team’s study is the largest and the first randomized trial of systemic therapy in HHT. The study demonstrated a significant and highly clinically relevant reduction in nosebleeds in pomalidomide-treated patients as well as an improvement in the HHT-specific QOL score. Pomalidomide may offer a novel approach to management of HHT. Since plasma was collected from patients in this study before and at the end of treatment, additional studies may identify biomarkers predicting pomalidomide responses.
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