Risk Stratification Using DIPSS and Splenomegaly in Myelofibrosis Treated with Pre-Transplant JAK inhibitors.

The Dynamic International Prognostic Scoring System for primary myelofibrosis (DIPSS) has been reported to predict transplant outcomes in myelofibrosis (MF) patients. Recently, the pre-transplant use of JAK inhibitors has become common in clinical practice, but it is unclear whether DIPSS is also useful for predicting transplant outcomes for these patients. In this study, we compared the prognostic impact of DIPSS between MF patients with and without pre-transplant Ruxolitinib therapy.

Loss-Prone HLA Class I Alleles Inform Outcomes of Early Hematopoietic Cell Transplantation in Acquired Aplastic Anemia.

HLA class I allele loss in acquired aplastic anemia (AA) represents an immune escape from the T cell-mediated pathogenesis. We investigated the impact of loss-prone HLA alleles on the hematopoietic cell transplantation (HCT) outcomes using registry data of 875 Japanese patients with acquired AA. HLA associations were evident exclusively among 399 patients who received HCT within 1 year of the diagnosis, consistent with the predominance of HLA loss in this group.

Allogeneic hematopoietic stem cell transplantation for older patients with adult T-cell leukemia/lymphoma: A nationwide retrospective study.

Background: Adult T-cell leukemia/lymphoma (ATL) is an aggressive malignancy with a poor prognosis. Allogeneic hematopoietic stem cell transplantation (allo-HSCT) is the only curative option but has been limited in older patients due to toxicity risks. With Japan's aging population and evolving treatment options, reassessing allo-HSCT in patients aged ≥65 is essential.

Dasatinib is superior to imatinib in adult Ph + ALL: a propensity score-matched analysis of pooled JALSG trial data.

This study compared dasatinib and imatinib in adult Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph + ALL). Using pooled data from three JALSG prospective trials (Ph + ALL202, Ph + ALL208, Ph + ALL213), we analyzed outcomes for 206 patients aged 15-64 years treated with dasatinib (n = 74) or imatinib (n = 132) in combination with chemotherapy. We applied propensity score matching (1:1) and inverse probability of treatment weighting to minimize selection bias and balance baseline characteristics.

Prevalence and outcome of VEXAS syndrome in unrelated hematopoietic cell transplantation for bone marrow failure.

VEXAS syndrome (vacuoles, E1 enzyme, X-linked, autoinflammatory, and somatic) is a recently identified clonal disorder caused by somatic UBA1 mutations in hematopoietic stem cells, leading to bone marrow failure (BMF) and systemic inflammation. We screened 1771 patients with BMF who underwent unrelated hematopoietic cell transplantation in Japan between 1995 and 2020 using multitarget real-time PCR. The diagnoses included myelodysplastic syndrome (MDS, n = 1139), myeloproliferative neoplasms (n = 125), plasma cell neoplasms (n = 23), acquired BMF (n = 395), and congenital BMF (n = 89).

The impact of age on survival and excess mortality after autologous hematopoietic cell transplantation in newly diagnosed multiple myeloma patients.

Despite the availability of novel agents, autologous hematopoietic cell transplantation (auto-HCT) remains the standard of care in newly diagnosed multiple myeloma (MM) patients. The impact of age on overall survival (OS), progression-free survival (PFS), relapse incidence, non-relapse mortality (NRM), and excess mortality (taking account of general population mortality) was investigated using information on 61,797 MM patients transplanted between 2013 and 2017. The median age at auto-HCT was 60.

Has early mortality following single-unit unrelated cord blood transplantation improved in recent years? A nationwide registry data analysis from 2003 to 2022 in Japan.

Unrelated single-unit cord blood transplantation (CBT) is a valuable alternative donor source for patients without matched related or unrelated donors. Although initial concerns included limited cell dose, delayed haematopoietic recovery and higher early mortality, advancements in transplant practices may have led to improved outcomes. However, it remains uncertain whether these improvements extend to the most recent years.

Machine Learning Algorithm to Explore Patients With Heterogeneous Treatment Effects of Clinically Significant CMV Infection and Non-Relapse Mortality After HSCT.

Introduction: Clinically significant cytomegalovirus infection (csCMVi) and non-relapse mortality (NRM) remain serious concerns after allogeneic hematopoietic stem cell transplantation (HSCT), but subpopulations with heterogeneous treatment effects (HTEs) is unclear. Although machine learning (ML) algorithms have recently been applied to HSCT, the methodology has not been well elucidated.

Methods: We developed a ML algorithm which combined weighting procedures and left-truncated and right-censored trees based on classification and regression tree algorithms to fit survival data with time-varying covariates and competing risks comprehensively.

Autologous stem cell transplantation from 2011 to 2022 in Japanese patients aged ≥ 65 years with relapsed or refractory diffuse large B-cell lymphoma.

High-dose chemotherapy with autologous stem cell transplantation (ASCT) is an option for patients aged ≥ 65 years with relapsed or refractory (R/R) diffuse large B-cell lymphoma (DLBCL). Few data are available to select patients suitable for chimeric antigen receptor T-cell (CAR-T) therapy or bispecific antibodies. We retrospectively analyzed the risk factors for poor outcomes for 451 Japanese patients aged ≥ 65 years with R/R DLBCL who received ASCT at either second complete remission or first partial remission (n = 336 and 115, respectively) between 2011 and 2022.

A win ratio approach for comparing graft-versus-host disease-free, relapse-free survival among alternative donors.

The optimal alternative donor type for patients lacking human leucocyte antigen (HLA)-matched related or unrelated donors remains unclear. In comparative studies evaluating donor types, graft-versus-host disease (GVHD)-free, relapse-free survival (GRFS) represents a well-established end-point but has limitations. The win ratio approach addresses these limitations by analysing multiple end-points with varying severities to account for the relative component priorities.

Prognostic Impact of Cytomegalovirus Reactivation After Transplantation From Cord Blood Compared to Other Donor Sources in Patients With Adult T-Cell Leukemia/Lymphoma in the Pre-Letermovir Era.

Background: Cytomegalovirus reactivation (CMV-react) is an indicator for the worse non-relapse mortality (NRM) and overall survival (OS) after allogeneic hematopoietic stem cell transplantation using HLA-matched related donor (MRD) and unrelated donor (URD) for adult T-cell leukemia/lymphoma (ATL). However, it remains unclear whether CMV-react correlates with outcomes after unrelated cord blood (U-CB) transplantation.

Methods: We conducted a retrospective nationwide study to evaluate the impact of CMV-react on the outcomes after posttransplant 100 days.

Outcomes of Allogeneic Hematopoietic Stem Cell Transplantation in Elderly Patients with Myeloid Malignancies Over 70 Years Old: A Retrospective Analysis of the Japanese Nationwide Registry.

Although allogeneic hematopoietic stem cell transplantation (SCT) remains a potentially curative treatment option for several myeloid malignancies, despite the fact that the average age at disease onset for myeloid malignancies is approximately 70 years of age, its applicability in elderly patients is challenging. We retrospectively evaluated the outcomes of elderly SCT patients with acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS), using a nationwide Japanese registry database. We analyzed the data of 3609 patients ranging from 65 to 79 years of age with AML or MDS who underwent initial SCT between 2003 and 2022, while focusing on those 70 to 74 (n = 645) and 75 to 79 years old (n = 65).

Prognostic factors for allogeneic haematopoietic cell transplantation outcomes in primary refractory acute myeloid leukaemia (2013-2022): A retrospective study by the adult acute myeloid leukaemia working group of the Japanese Society for Transplantation and Cellular Therapy.

Primary refractory acute myeloid leukaemia (AML) remains a major clinical challenge, with poor outcomes despite salvage chemotherapy. Allogeneic haematopoietic cell transplantation (HCT) continues to be a potentially curative option for these patients. However, recent data on outcomes and prognostic factors specific to primary refractory AML remain limited.

An ASTCT, CIBMTR, EBMT, and APBMT Consensus Statement Defining Response Criteria for Hematopoietic Cell Transplantation Associated Thrombotic Microangiopathy (TA-TMA) Directed Therapy.

Background: Transplant associated thrombotic microangiopathy (TA-TMA) confers significant morbidity and mortality in hematopoietic cell transplant recipients. The safety and efficacy of multiple TA-TMA directed therapeutic agents are being tested in ongoing clinical trials. In the absence of approved drugs, several treatments are used off-label.

Efficacy of Total-Body Irradiation-based Intensified Myeloablative Regimens for Acute Leukemia-An International Collaborative Study.

Background: In this study, we compared outcomes of intensified myeloablative conditioning regimens using large registry data from Japan (Japanese Society for Transplantation and Cellular Therapy) and the United States (Center for International Blood and Marrow Transplant Research).

Methods: Adult patients who underwent their first myeloablative allogeneic hematopoietic stem cell transplantation (HSCT) for acute leukemia in remission between 2010 and 2018 using conditioning regimens of cyclophosphamide plus total-body irradiation (CY/TBI), CY/TBI+cytarabine (AraC), or CY/TBI+etoposide (VP16) were included.

Results: The acute myeloid leukemia (AML) cohort ( = 480, 38.

Impact of Conditioning Intensity on Clinical Outcomes of Second Allogeneic Hematopoietic Cell Transplantation for Relapse After First Transplantation.

Although second allogeneic hematopoietic cell transplantation HCT (HCT2) is a potentially curative treatment for patients relapsing after their first HCT (HCT1), it is associated with higher non-relapse mortality (NRM) compared with HCT1. Furthermore, while reduced-intensity conditioning (RIC) in HCT2 might decrease NRM, there is no consensus on which patients may benefit from RIC. We retrospectively analyzed 2478 patients who underwent HCT2 for relapse of hematologic malignancies after HCT1.

Impact of graft-versus-host disease on outcomes of cord blood transplantation according to HLA disparity and its prophylaxis type.

This study evaluated the impact of acute graft-versus-host disease (aGVHD) on cord blood transplantation (CBT) outcomes based on human leukocyte antigen (HLA) disparity and GVHD prophylaxis type. Data from 4,196 adult patients with acute myeloid leukemia, acute lymphoblastic leukemia, or myelodysplastic syndrome were analyzed. Patients were classified by HLA mismatch (8/8-6/8, 5/8, and 4/8-2/8) and further by GVHD prophylaxis type (methotrexate [MTX] or mycophenolate mofetil [MMF]).

Safety of hematopoietic stem cell donation from young related donors following the lowering of the age of legal adulthood in Japan.

The Japanese government's recent lowering of the legal adult age to 18 may prompt reconsideration of the minimum age for donors in the Japan Marrow Donor Program. To provide foundational data for decision-makers, this retrospective study analyzed the safety of hematopoietic stem cell donation among 1999 related donors aged 18 to 24 years, using data from the Japanese Data Center for Hematopoietic Cell Transplantation. Severe adverse events (SAEs) occurred in 1.

Impact of center volume on acute graft versus host disease in allogeneic stem cell transplant recipients.

Prophylactic, diagnostic, and treatment strategies for acute graft-versus-host disease (aGVHD) may vary across medical centers and physicians. We aimed to investigate the relationship between center volume and the incidence and outcomes of aGVHD. This retrospective study included 28,786 patients who underwent their first hematopoietic stem cell transplantation (HSCT) (entire cohort) and 9498 patients who developed grade II-IV aGVHD (aGVHD cohort).

Hematopoietic cell transplantation in pediatric EB virus-associated hemophagocytic lymphohistiocytosis.

Background: Few reports exist on the transplant outcomes of Epstein-Barr virus-associated hemophagocytic lymphohistiocytosis (EBV-HLH). This nationwide survey evaluated the clinical outcomes of pediatric patients with EBV-HLH who underwent allogeneic HCT.

Methods: Data from 32 pediatric patients with EBV-HLH who underwent their first allogeneic HCT between 2000 and 2020 were reviewed retrospectively.

Classical and Late-Onset SOS/VOD After Allogeneic HSCT: A Japanese Transplant Registry Analysis.

Sinusoidal obstruction syndrome/veno-occlusive disease (SOS/VOD) is a lethal complication of allogeneic hematopoietic stem cell transplantation (allo-HSCT). According to the 2016 European Society for Blood and Marrow Transplantation criteria, SOS/VOD is classified into classical SOS/VOD and late-onset SOS/VOD, but their similarities and differences remain unclear. Here we retrospectively investigated the incidence, risk factors, and impact on transplant outcomes of classical and late-onset SOS/VOD in 16 518 allo-HSCT recipients using the Japanese nationwide registry data.