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.

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.

Treatment of post-transplant lymphoproliferative disorders in kidney transplant recipients: a single-center retrospective analysis.

Post-transplant lymphoproliferative disorder (PTLD) is a significant complication of immunosuppression after kidney transplantation and has no established standard therapy. Achieving favorable treatment outcomes and preserving renal function in patients with PTLD remains challenging, particularly when the central nervous system (CNS) is involved. Here we describe our experience with 8 patients who developed PTLD after kidney transplantation at our institution.

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).

Refinement of Day 28 Treatment Response Criteria for Acute GVHD: A Collaboration Study of the JSTCT and MAGIC.

Overall response (OR) that combines complete (CR) and partial responses (PR) at day (D) 28 is the conventional endpoint for acute GVHD trials. Since PR includes heterogeneous clinical presentations, reclassifying PR could produce a better endpoint. Patients in the primary treatment cohort from JSTCT were randomly divided into training and validation sets.

Augmented use of L-asparaginase markedly improves AYA ALL outcomes: FBMTG prospective MRD2014 study.

The enhanced utilization of native L-asparaginase (L-Asp) aims to improve treatment outcomes for adult patients with non-Philadelphia chromosome (Ph) acute lymphoblastic leukemia (ALL). In this measurable residual disease 2014 (MRD2014) study, we modified our protocol to include an augmented dose of native L-Asp. Compared with former MRD2008, the total dose of L-Asp was raised from 36 000 U/m to 232 000 U/m in patients aged 16 to 35 and from 36 000 U/m to 132 000 U/m in patients aged 36 to 65 years.

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.

Refractory invasive pulmonary aspergillosis caused by two Aspergillus species in an allogeneic hematopoietic stem cell transplantation recipient.

Invasive fungal infections are critical complications in patients with hematological malignancies. Herein, we report a case of refractory invasive pulmonary aspergillosis (IPA) caused by two Aspergillus species in a recipient of hematopoietic stem cell transplantation. Repeated sputum cultures revealed causative pathogens, which were identified as Aspergillus terreus and Aspergillus flavus.

Whole-Genome Sequencing and Comparative Genomic Analysis of Three Clinical Bloodstream Infection Isolates of .

is a recently described yeast species related to and exclusively isolated from clinical specimens. However, its genomic features and pathogenic potential remain poorly understood. In this study, we performed whole-genome sequencing on three blood-derived isolates from patients with invasive fungal infections and comparative analyses with 13 related species.

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]).

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.

Improved Transplant Outcomes With Alternative Donors in Myelofibrosis: A 20-Year Japanese Registry Analysis of Donor Sources and the Impact of Ruxolitinib.

This study of 308 myelofibrosis patients shows that in recent years (2013-2019), alternative donors (mismatched unrelated donors and cord blood) achieved survival rates comparable to HLA-matched donors-a significant improvement compared to earlier years (2000-2012) when outcomes differed substantially. Ruxolitinib showed significant benefits in older patients (≥ 57), particularly with mismatched unrelated donors. Cord blood transplantation outcomes improved with MMF-based GVHD prophylaxis.

Heterogeneity of Survival Benefit Conferred By Letermovir.

Variation in treatment effects based on individual patient characteristics-known as treatment effect heterogeneity or effect modification-has recently gained significant attention. A previous clinical trial and its post hoc analysis suggested that letermovir (LTV) may reduce mortality more in some patients than in others. We hypothesized that the survival benefit of LTV differs according to each patient's specific characteristics.

The impact of the SARS-CoV-2 pandemic on umbilical cord blood transplantation in Japan: insights from an interrupted time series analysis.

The SARS-CoV-2 pandemic disrupted healthcare systems worldwide, particularly affecting hematopoietic stem cell transplantation (HSCT) activities. Understanding the impact of the SARS-CoV-2 pandemic on transplant practices, especially in Japan, where cord blood transplantation (CBT) is prevalent, is crucial. A total of 40,444 allogeneic HSCT cases in Japan between 2011 and 2021 were examined using an interrupted time series analysis to assess the impact of COVID-19 on CBT utilization.

Comparative analysis of allogeneic hematopoietic cell transplantation in patients with therapy-related and de novo acute promyelocytic leukemia A retrospective study.

Although allogeneic hematopoietic cell transplantation (HCT) is an alternative treatment for relapsed or refractory (R/R) acute promyelocytic leukemia (APL), little is known regarding the utility of allogeneic HCT for R/R therapy-related APL (t-APL). We retrospectively analyzed data for 144 patients with APL (t-APL, n = 20 and de novo APL, n = 124) who received a first allogeneic HCT between 2008 and 2020. We found no significant differences in survival between the t-APL and de novo APL groups.