Publications by authors named "Minoo Battiwalla"

Purpose: On the basis of the results of the ZUMA-3 trial, brexucabtagene autoleucel (brexu-cel), a CD19-directed chimeric antigen receptor T-cell therapy, gained US Food and Drug Administration approval in October 2021 for adults with relapsed/refractory (R/R) B-cell ALL (B-ALL). We report outcomes of patients treated with brexu-cel as a standard therapy.

Methods: We developed a collaboration across 31 US centers to study adults with B-ALL who received brexu-cel outside the context of a clinical trial.

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We analyzed access barriers to anti-CD19+ chimeric antigen receptor T-cells (CART) for non-Hodgkin lymphoma (NHL) within a community-based transplant and cell therapy network registry. 357 intended recipients of FDA-approved anti-CD19+ CART were identified in the study period (2018 to 2022). Results showed that the median age at referral was 61 years, referral year was 2018 (4%), 2019 (14%), 2020 (18%), 2021 (26%), and 2022 (38%).

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The treatment setting influences acute myeloid leukemia (AML) outcomes. Most cancer patients receive care in the community, yet few studies have evaluated the effectiveness of clinical programs outside of academic or National Cancer Institute (NCI)-designated cancer centers. This was a multi-level, case-controlled study of real-world outcomes for initial AML treatment in a community-based network for 1,391 patients with AML between 2011 and 2018.

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Blastic plasmacytoid dendritic cell neoplasm (BPDCN) is a rare hematological malignancy with a poor prognosis and considered incurable with conventional chemotherapy. Small observational studies reported allogeneic hematopoietic cell transplantation (allo-HCT) offers durable remissions in patients with BPDCN. We report an analysis of patients with BPDCN who received an allo-HCT, using data reported to the Center for International Blood and Marrow Transplant Research (CIBMTR).

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Management of higher-risk myelodysplastic syndromes (HR-MDS) is challenging in the real world. We studied 200 patients with HR-MDS within a large US community hospital network. We describe the clinical presentation, patient-related factors, prognostic characteristics, treatment patterns, clinical outcomes and resource utilization.

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Article Synopsis
  • Researchers created a prognostic model to predict outcomes for patients with myelofibrosis undergoing allogeneic hematopoietic cell transplantation by analyzing data from 623 patients in the U.S. (CIBMTR cohort) from 2000 to 2016.
  • They identified key factors that influence mortality, assigning weighted scores based on age, donor matching, hemoglobin levels, and donor compatibility, which resulted in differing survival rates among low, intermediate, and high score groupings.
  • The model was validated in a European cohort (EBMT), proving effective for predicting overall survival and transplant-related mortality, aiding clinicians in discussing transplantation prospects with myelofibrosis patients.
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  • Overall survival rates for multiple myeloma have greatly improved due to advanced treatments like proteasome inhibitors and autologous stem cell transplants.
  • However, there's an increased risk of developing second primary malignancies (SPM) and second hematological malignancies (SHM) after these treatments, affecting patients' post-transplant health.
  • A study showed that patients who developed SPM and SHM had significantly lower progression-free survival (PFS) and overall survival (OS), highlighting the need for ongoing monitoring of these risks in survivorship care.
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The landscape for evidence generation in hematologic malignancies is rapidly evolving. While randomized controlled trials (RCTs) remain the gold standard in support of drug efficacy, approval and use, the supplemental use of real-world data (RWD), generated as part of routine healthcare delivery, and real-world evidence (RWE), the insights derived from RWD, in this setting has become increasingly common. There is a wide variety of sources of RWD, each with its own strengths and weaknesses that need to be considered when determining its appropriate use in RWE generation.

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  • T cell prolymphocytic leukemia (T-PLL) is a rare and aggressive cancer with limited treatment options and poor survival rates, prompting this study to evaluate allogeneic hematopoietic cell transplantation (alloHCT) outcomes in affected patients.* -
  • The analysis utilized data from 266 T-PLL patients who underwent alloHCT from 2008 to 2018, revealing a 4-year overall survival rate of 30% and highlighting significant factors affecting survival, including the conditioning regimen and patient age.* -
  • Findings indicated that myeloablative conditioning and poor performance status lead to worse survival and increased treatment-related mortality, while stable disease or progression correlated with a higher relapse risk, suggesting
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Introduction: Cytokine release syndrome is a life-threatening hyper-inflammatory state induced by immune effector cell therapy. Anti-interleukin 6-(IL-6) therapy, such as tocilizumab, is the standard treatment for cytokine release syndrome since it reverses symptoms without compromising immune effector cell therapy efficacy. Glucocorticoids are reserved for refractory or severe cytokine release syndrome due to concern for attenuating antitumor activity.

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  • Bortezomib-based triplet regimens, VRD (bortezomib, lenalidomide, dexamethasone) and VCD (bortezomib, cyclophosphamide, dexamethasone), are commonly used for treating newly diagnosed multiple myeloma in patients eligible for transplantation, but their relative effectiveness is debated.
  • A study comparing long-term outcomes of these regimens found that while patients receiving VCD were generally worse off initially, the efficacy between VRD and VCD in terms of response rates before and after autologous stem cell transplantation was similar.
  • However, patients treated with VRD had better overall and progression-free survival rates post-transplant, suggesting
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Cytogenetic and molecular abnormalities are known to influence post-transplant outcomes in acute myeloid leukemia (AML) but data assessing the prognostic value of combined genetic models in the HCT setting are limited. We developed an adapted European LeukemiaNet (aELN) risk classification based on available genetic data reported to the Center for International Blood and Marrow Transplant Research, to predict post-transplant outcomes in 2289 adult AML patients transplanted in first remission, between 2013 and 2017. Patients were stratified according to aELN into three groups: favorable (Fav, N = 181), intermediate (IM, N = 1185), and adverse (Adv, N = 923).

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Young adult (YA) survivors of allogeneic hematopoietic cell transplantation (HCT) are at risk for late psychosocial challenges, including the inability to return to work post-HCT. Work-related outcomes in this population remain understudied, however. We conducted this study to assess the post-HCT work status of survivors of allogeneic HCT who underwent HCT as YAs and to analyze the patient-, disease-, and HCT-related factors associated with their work status at 1 year post-HCT.

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Minor histocompatibility antigens (mHAs), recipient-derived peptide epitopes presented on the cell surface, are known to mediate graft-versus-host disease (GVHD); however, there are no current methods to associate mHA features with GVHD risk. This deficiency is due in part to the lack of technological means to accurately predict, let alone confirm, the tremendous number of potential mHAs in each individual transplant. Previous studies have shown that different HLA molecules present varying fractions of candidate peptide epitopes; however, the genetic "distance" between HLA-matched donors and recipients is relatively constrained.

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This longitudinal cohort study compared ocular surface indicators in forty allogeneic hematopoietic stem cell transplant (HSCT) subjects with twenty healthy controls at baseline and identified changes in ocular graft-versus-host disease (oGVHD). Outcome measures included: Ocular Surface Disease Index (OSDI), tear osmolarity, Schirmer's test, Oxford corneal staining score, tear break-up time (TBUT), and tear and serum biomarkers (IFN-γ, IL-10, MMP-9, IL-12, IL-13, IL-17α, IL-1β, IL-2, IL-4, IL-6, IL-8, CXCL10, MCP-1, MIP-1α, RANTES, TNF-α). At baseline the HSCT group had higher median Oxford corneal staining score (1.

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We compared outcomes in 603 patients with myelodysplastic syndrome (MDS) after HLA-haploidentical relative (n = 176) and HLA-matched unrelated (n = 427) donor hematopoietic cell transplantation (HCT) from 2012 to 2017, using the Center for International Blood and Marrow Transplant Research database. All transplantations used reduced-intensity conditioning regimens. Total-body irradiation plus cyclophosphamide and fludarabine was the predominant regimen for HLA-haploidentical relative donor HCT, and graft-versus-host disease (GVHD) prophylaxis was uniformly posttransplantation cyclophosphamide, calcineurin inhibitor, and mycophenolate.

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Social determinants of health, including poverty, contribute significantly to health outcomes in the United States; however, their impact on pediatric hematopoietic cell transplantation (HCT) outcomes is poorly understood. We aimed to identify the association between neighborhood poverty and HCT outcomes for pediatric allogeneic HCT recipients in the Center for International Blood and Marrow Transplant Research database. We assembled 2 pediatric cohorts undergoing first allogeneic HCT from 2006 to 2015 at age ≤18 years, including 2053 children with malignant disease and 1696 children with nonmalignant disease.

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Background: Upfront autologous hematopoietic stem cell transplantation (AHCT) remains an important therapy in the management of patients with multiple myeloma (MM), a disease of older adults.

Methods: The authors investigated the outcomes of AHCT in patients with MM who were aged ≥70 years. The Center for International Blood and Marrow Transplant Research (CIBMTR) database registered 15,999 patients with MM in the United States within 12 months of diagnosis during 2013 through 2017; a total of 2092 patients were aged ≥70 years.

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