Image showing a PICO summary for: Phase I/II Trials of Donor Regulatory T Cells for the Treatment of Steroid-Refractory Chronic Graft versus Host Disease by Soares et al. featured in the Stem Cell Evidence Alert - March 2026

New from the #StemCellEvidence Alert:

Phase I/II Trials of Donor Regulatory T Cells for the Treatment of Steroid-Refractory Chronic Graft versus Host Disease
by Soares et al.
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#MedSky

Longitudinal Study of Late Acute and Chronic Graft-Versus-Host Disease after Allogeneic Hematopoietic Cell Transplantation: A Long-Term Follow-up Study from the Chronic Graft-Versus-Host Disease Consortium Late acute and chronic graft-versus-host disease (GVHD) contribute to morbidity and death after allogeneic hematopoietic cell transplantation (HCT). A prior national Chronic GVHD Consortium longitudinal study enrolled patients pre- or early post-HCT and identified the incidence of late acute GVHD, chronic GVHD, and chronic GVHD subtypes of bronchiolitis obliterans syndrome (BOS) and cutaneous sclerosis. We now report 10-yr follow-up from that study in a long-term follow-up analysis (N = 911 subjects). Late acute GVHD occurred in 11% at a median of 5.5 mo. Chronic GVHD in total occurred in 54% with median onset of 7.4 mo. BOS (4% of subjects, median onset 12.6 mo) and cutaneous sclerosis (10% of subjects, median onset of 17.2 mo) were less frequent and had later-onset. The long-term analysis demonstrated additional GVHD events occurring beyond 1 to 2 yr post-HCT. Inter-conversion between GVHD types was common, and notably included new development of BOS and cutaneous sclerosis after initial chronic GVHD presentation without these manifestations. GVHD-free, relapse-free survival (GVHD-DFS, survival without relapse or development of late acute or chronic GVHD) was 22% at 2 yr, 18% at 5 yr, and 15% at 10 yr post-HCT. Non-relapse mortality continued to increase beyond 2 yr, with 10-yr estimates up to 35% (late acute), 31% (chronic GVHD), 62% (BOS), and 36% (cutaneous sclerosis). Durable complete discontinuation of immune suppression was uncommon for all GVHD types. These data suggest that extended surveillance for late acute and chronic GVHD is needed post-HCT due to late occurrences, and that associated mortality is high through 10 yr post-GVHD onset.

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Longitudinal Study of Late Acute and Chronic Graft-Versus-Host Disease after Allogeneic Hematopoietic Cell Transplantation: A Long-Term Follow-up Study from the Chronic Graft-Versus-Host Disease Consortium
by Pidala et al.
www.stemcellevidence.com/alerts/artic...
#MedSky

Image showing a PICO summary for: Longitudinal Study of Late Acute and Chronic Graft-Versus-Host Disease after Allogeneic Hematopoietic Cell Transplantation: A Long-Term Follow-up Study from the Chronic Graft-Versus-Host Disease Consortium by Pidala et al. featured in the Stem Cell Evidence Alert - March 2026

From the #StemCellEvidence Alert:

Longitudinal Study of Late Acute and Chronic Graft-Versus-Host Disease after Allogeneic Hematopoietic Cell Transplantation: A Long-Term Follow-up Study from the Chronic Graft-Versus-Host Disease Consortium
by Pidala et al.
Link in the comments
#MedSky

The role of HLA transcription in unrelated hematopoietic cell transplantation Expression levels of human leukocyte antigen (HLA) are associated with susceptibility to various diseases and outcomes after allogeneic hematopoietic cell transplantation (allo-HCT). However, there has been no comprehensive analysis of the effect of the expression levels of the HLA-A, -B, -C, and -DRB1 alleles in unrelated (UR)-HCT. Using the Capture RNA-Seq method, we analyzed the gene expression of HLA alleles in 443 healthy donors and determined the allele-specific transcription levels (AST levels). We assigned median (M)-AST levels to HLA typing data of patients who received a transplant from HLA-A, -B, -C, and -DRB1-matched unrelated donors using transplant registry data. All 6,084 patients were divided into low, middle, and high tertile groups according to the distribution of the sum of the two alleles of the M-AST level for each HLA locus and the sum of the eight alleles of the M-AST level for all four loci. The risk of grade II-IV acute graft-versus-host disease (GVHD) was significantly higher in the middle group (hazard ratio, 1.11; P = 0.044) and high group (hazard ratio, 1.20; P < 0.001) than in the low group for the sum of the HLA-A, -B, -C, and -DRB1 loci. Similar results were observed at the HLA-A, -B, -C, and -DRB1 loci. Higher transcription levels were also associated with a lower risk of relapse at the HLA-B, -C, and -DRB1 loci. Our data suggest that a high transcription level of patient and/or donor HLA may evoke strong alloimmune responses and affect UR-HCT outcomes.

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The role of HLA transcription in unrelated hematopoietic cell transplantation
by Morishima et al.
www.stemcellevidence.com/alerts/artic...
#MedSky

Image showing a PICO summary for: The role of HLA transcription in unrelated hematopoietic cell transplantation by Morishima et al. featured in the Stem Cell Evidence Alert - March 2026

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The role of HLA transcription in unrelated hematopoietic cell transplantation
by Morishima et al.
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#MedSky

Bone marrow transplantation for sickle cell disease using post-transplantation cyclophosphamide and 400 cGy TBI Reduced intensity conditioning (RIC), haploidentical donors, and post-transplantation cyclophosphamide (PTCy) have overcome many barriers associated with bone marrow transplantation (BMT) for sickle cell disease (SCD). However, initial approaches had high graft failure rates. Our preliminary data suggested increasing the total body irradiation (TBI) dose from 200 to 400 cGy could improve engraftment. This study included SCD patients aged 2-70 undergoing BMT from November 2014 - January 2025. The regimen included anti-thymocyte globulin, fludarabine, cyclophosphamide, and single fraction 400 cGy TBI. Graft-vs-host disease (GVHD) prophylaxis included PTCy, mycophenolate mofetil, and sirolimus. Outcomes measured were disease-free survival (DFS), graft failure, overall survival (OS), and GVHD incidence. Forty-three patients (median age 23) were transplanted. Thirty-four (79%) had >2 indications for BMT and all had >2 SCD comorbidities. Five-year OS probability was 95.5% (CI 0.87 - 1.0) at a median follow-up of 2.43 years. DFS probability at 2 years was 94.5% (CI 0.87 - 1.0) with only 2 (5%) graft failures. Two patients died late (2.5 and 6 years) after BMT. The cumulative incidence of grades 3-4 acute GVHD was 2.4% (CI 0 - 0.07) and moderate-severe chronic GVHD was 7.3% (CI 0 - 0.15). Median time to discontinuation of immunosuppression was 354 days. Of the 27 female patients, 12 had return of menses and/or normalized gonadal function. RIC haploidentical BMT with 400 cGy maintained a low toxicity profile, provides high rates of durable engraftment, and may preserve fertility. This regimen expands the availability of curative therapy for severe SCD. NCT00489281.

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Bone marrow transplantation for sickle cell disease using post-transplantation cyclophosphamide and 400 cGy TBI
by Goldenberg et al.
www.stemcellevidence.com/alerts/artic...
#MedSky

Image showing a PICO summary for: Bone marrow transplantation for sickle cell disease using post-transplantation cyclophosphamide and 400 cGy TBI by Goldenberg et al. featured in the Stem Cell Evidence Alert - March 2026

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Bone marrow transplantation for sickle cell disease using post-transplantation cyclophosphamide and 400 cGy TBI
by Goldenberg et al.
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#MedSky

The BIOPREVENT machine-learning algorithm predicts chronic graft-versus-host disease and mortality risk using posttransplant biomarkers BACKGROUNDChronic graft-versus-host disease (cGVHD) is a major contributor to nonrelapse mortality (NRM) following hematopoietic cell transplantation (HCT). Whether machine-learning (ML) models with biomarkers improve the accuracy for predicting future cGVHD/NRM is not established.METHODSWe developed BIOPREVENT (BIOmarkers PREVENTion), a ML algorithm using data from 1,310 HCT recipients, incorporating 7 plasma proteins measured at Day 90/100 post-HCT and 9 clinical variables. Patients were divided into training and validation datasets. ML models - including CoxXGBoost, Group SCAD, Adaptive Group Lasso, Random Survival Forests, and Bayesian Additive Regression Trees (BART) - were used to estimate time-varying Area Under the ROC Curve (AUCt) at Days 180, 270, 360, and 540. Deep learning models were also evaluated.RESULTSML models with biomarkers outperformed clinical-only models for predicting cGVHD, with BART and CoxXGBoost achieving AUCt greater than 0.65 at 1 year. For NRM, models with biomarkers achieved AUCt ranging from 0.75-0.91. Deep learning did not outperform other ML approaches. BART consistently demonstrated high predictive accuracy and was selected for the final BIOPREVENT model. Calibration curves aligned with observed values. Variable importance analysis identified MMP3 and CXCL9 as key for cGVHD prediction and IL1RL1 and sCD163 for NRM. Cumulative incidences of cGVHD and NRM differed significantly based on BIOPREVENT-defined cutpoints.CONCLUSIONBIOPREVENT accurately predicts individual risk of future cGVHD and NRM using biomarkers at 3 months post-HCT. A publicly available R Shiny web application supports its clinical use. Further studies are needed to explore its role in guiding preemptive therapy.TRIAL REGISTRATIONBMTCTN 0201, BMTCTN 1202, and NCT02194439.FUNDINGR01CA264921, U10HL069294, U24HL138660, R01HD074587, and P01HL158505.

#StemCellEvidence link to:
The BIOPREVENT machine-learning algorithm predicts chronic graft-versus-host disease and mortality risk using posttransplant biomarkers
by Martens et al.
www.stemcellevidence.com/alerts/artic...
#MedSky

Image showing a PICO summary for: The BIOPREVENT machine-learning algorithm predicts chronic graft-versus-host disease and mortality risk using posttransplant biomarkers by Martens et al. featured in the Stem Cell Evidence Alert - March 2026

From the #StemCellEvidence Alert:

The BIOPREVENT machine-learning algorithm predicts chronic graft-versus-host disease and mortality risk using posttransplant biomarkers
by Martens et al.

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Baseline IPSS-M vs Pretransplant Risk Downstaging as Prognostic Determinants in MDS Undergoing Allogeneic Transplantation Risk stratification in myelodysplastic syndromes (MDS) is essential for clinical decision-making, yet the optimal approach to estimate risk for patients undergoing allogeneic stem cell transplantation (alloHSCT) remains uncertain. Whether dynamic changes in risk between diagnosis and post-hypomethylating agent (HMA) therapy improve prognostic accuracy beyond baseline evaluation has not been established. We retrospectively studied 176 HMA-treated patients who underwent alloHSCT, applying the Molecular International Prognostic Scoring System (IPSS-M) at both diagnosis and pre-transplant. The primary endpoint was 4-year progression-free survival (PFS). Overall, dynamic assessment did not improve prognostic performance compared with baseline evaluation. For 4-year PFS, C-indices at diagnosis versus at alloHSCT were 0.6406 versus 0.6377 (p=0.82). Patients with worsening risk after HMA experienced notably inferior outcomes, while those with apparent improvement fared no better than patients with unchanged risk (4-year PFS: 50%, 50%, and 31% for improved, unchanged, and worsening risk, respectively). Apparent IPSS-M improvement before alloHSCT yielded no gains in survival and no reduction in relapse relative to unchanged risk, a pattern consistent among TP53 wild-type patients. Moreover, clearance of TP53 mutations following HMA therapy did not translate into improved post-transplant outcomes. In summary, dynamic reassessment with IPSS-M prior to alloHSCT offers no prognostic advantage over baseline evaluation at diagnosis in HMA-treated MDS patients. Accordingly, risk reduction should not be regarded as a therapeutic goal or trial endpoint, whereas risk progression constitutes an adverse marker that may inform incorporation of post-transplant maintenance strategies or intensified conditioning regimens to improve survival.

#StemCellEvidence link to:

Baseline IPSS-M vs Pretransplant Risk Downstaging as Prognostic Determinants in MDS Undergoing Allogeneic Transplantation
by Aguirre et al.
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#MedSky

Image showing a PICO summary for: Baseline IPSS-M vs Pretransplant Risk Downstaging as Prognostic Determinants in MDS Undergoing Allogeneic Transplantation by Aguirre et al. featured in the Stem Cell Evidence Alert - March 2026

From the #StemCellEvidence Alert:

Baseline IPSS-M vs Pretransplant Risk Downstaging as Prognostic Determinants in MDS Undergoing Allogeneic Transplantation
by Aguirre et al.
🔗Link in the comments.

Comparable outcomes of HSCT up-front and after failure of IST in pediatric aplastic anemia in recent years We investigated the outcomes of 179 pediatric patients with severe aplastic anemia (SAA) who received up-front hematopoietic stem cell transplantation (HSCT; n = 87) or received HSCT after failure of immunosuppressive therapy (IST; n = 92). With a median follow-up of 4.0 years (range, 0.2-10.7) after HSCT, 161 patients were alive at last follow-up. The cumulative incidence of graft failure, grade 2 to 4 acute and chronic graft-versus-host disease (GVHD) was 10%, 18%, and 8%, respectively. Five-year overall survival (OS) and GVHD-free, relapse/rejection-free survival (GRFS) were 89% and 79%, respectively. Patients who received up-front HSCT from a matched sibling donor (MSD) or ≥9/10 HLA-matched unrelated donor (MUD) both achieved an OS of 95%. OS was superior in patients who received up-front HSCT than those who received HSCT after failure of IST (up-front, 95% [95% CI, 89-100] vs after IST failure, 83% [95% CI,75-91]; P < .01). However, OS in the HSCT group after IST failure improved over time, resulting in comparable OS between up-front HSCT and HSCT after IST failure from 2015 (97% vs 89%; P = .09). Moreover, there was no difference in GRFS of MUD-HSCT comparing patients who received a transplant up front or after IST failure (up-front, 77% vs after IST failure, 76%; P value not significant). Our data highlight the good transplant outcomes of a consecutive pediatric SAA cohort. Although up-front HSCT from a readily available MUD is a viable option if an MSD is not available, HSCT after IST failure from a well-matched unrelated donor also yields favorable outcomes.

#StemCellEvidence link 🔗to:

Comparable outcomes of HSCT up-front and after failure of IST in pediatric aplastic anemia in recent years
by Andresen et al.
www.stemcellevidence.com/alerts/artic...

Image showing a PICO summary for: Comparable outcomes of HSCT up-front and after failure of IST in pediatric aplastic anemia in recent years by Andresen et al. featured in the Stem Cell Evidence Alert - March 2026

From the #StemCellEvidence Alert:

Comparable outcomes of HSCT up-front and after failure of IST in pediatric aplastic anemia in recent years
by Andresen et al.

🔗Link in the comments

Image showing a PICO summary for: HCT Frailty Scale (HCT-FS) for assessing frailty in adult candidates for allogeneic haematopoietic cell transplantation: an international prospective, observational cohort study by Salas et al. featured in the Stem Cell Evidence Alert - March 2026

New from the #StemCellEvidence Alert:

HCT Frailty Scale (HCT-FS) for assessing frailty in adult candidates for allogeneic haematopoietic cell transplantation: an international prospective, observational cohort study
by Salas et al.
🔗Link in the comments.

Stem Cell Evidence Stem Cell Evidence is a comprehensive collection of high quality research relevant to haematopoietic stem cell transplantation. Articles are pre-selected for clinical relevance and methodological qual...

🔗Link to top articles featured in the #StemCellEvidence Alert - March 2026:
www.stemcellevidence.com/alerts/latest

Image showing an extract of the Stem Cell Evidence Alert - March 2026

The #StemCellEvidence alert for March is out!

See all the top 10 articles; each one has an easy-to-read PICO summary.
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GVHD prophylaxis after cord blood transplantation in patients with high-risk AML: a nationwide Japanese cohort study Cord blood transplantation (CBT) is a curative option for patients with high-risk acute myeloid leukemia (AML), including therapy-related AML (t-AML). However, the optimal prophylaxis for graft-versus...

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GVHD prophylaxis after cord blood transplantation in patients with high-risk AML: a nationwide Japanese cohort study
by Yamasaki et al.
www.stemcellevidence.com/alerts/artic...
#MedSky

Image showing a PICO summary for: GVHD prophylaxis after cord blood transplantation in patients with high-risk AML: a nationwide Japanese cohort study by Yamasaki et al. featured in the Stem Cell Evidence Alert - February 2026

New from the #StemCellEvidence Alert:

GVHD prophylaxis after cord blood transplantation in patients with high-risk AML: a nationwide Japanese cohort study
by Yamasaki et al.
#MedSky

Efficacy and safety of ruxolitinib for graft-versus-host disease prophylaxis in allogeneic hematopoietic stem cell transplantation: a systematic review and meta-analysis INTRODUCTION: Graft-versus-host disease (GVHD) remains a major complication after allogeneic hematopoietic stem cell transplantation (allo-HSCT). Despite standard prophylaxis, acute and chronic GVHD i...

#StemCellEvidence link to:

Efficacy and safety of ruxolitinib for graft-versus-host disease prophylaxis in allogeneic hematopoietic stem cell transplantation: a #systematicreview and #metaanalysis
by Xie e al.
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#MedSky

Image showing a PICO summary for: Efficacy and safety of ruxolitinib for graft-versus-host disease prophylaxis in allogeneic hematopoietic stem cell transplantation: a #systematicreview and #metaanalysis by Xie e al. featured in the Stem Cell Evidence Alert - February 2026

From the #StemCellEvidence Alert:

Efficacy and safety of ruxolitinib for graft-versus-host disease prophylaxis in allogeneic hematopoietic stem cell transplantation: a #systematicreview and #metaanalysis
by Xie e al.
#MedSky

Real-world outcomes of tisagenlecleucel treatment in patients with relapsed or refractory large B-cell lymphomas: a nationwide registry BACKGROUND: Chimeric antigen receptor (CAR) T-cell therapy has transformed treatment for patients with relapsed or refractory large B-cell lymphoma (LBCL). However, real-world data in Japan remain lim...

#StemCellEvidence link to:

Real-world outcomes of tisagenlecleucel treatment in patients with relapsed or refractory large B-cell lymphomas: a nationwide registry
by Sakurai et al.
www.stemcellevidence.com/alerts/artic...
#MedSky

Image showing a PICO summary for: Real-world outcomes of tisagenlecleucel treatment in patients with relapsed or refractory large B-cell lymphomas: a nationwide registry by Sakurai et al. featured in the Stem Cell Evidence Alert - February 2026

New from the #StemCellEvidence Alert:

Real-world outcomes of tisagenlecleucel treatment in patients with relapsed or refractory large B-cell lymphomas: a nationwide registry
by Sakurai et al.
#MedSky

Modeling donor age and type trade-offs for personalized selection in allogeneic hematopoietic cell transplantation with post-transplant cyclophosphamide prophylaxis The prevailing fixed-hierarchy approach to donor selection for allogeneic hematopoietic cell transplantation (HCT) fails to capture critical interactions between donor age and type. We addressed this ...

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Modeling donor age and type trade-offs for personalized selection in allogeneic hematopoietic cell transplantation with post-transplant cyclophosphamide prophylaxis
by Mehta et al.
www.stemcellevidence.com/alerts/artic...
#MedSky

Image showing a PICO summary for: Modeling donor age and type trade-offs for personalized selection in allogeneic hematopoietic cell transplantation with post-transplant cyclophosphamide prophylaxis by Mehta et al. featured in the Stem Cell Evidence Alert - February 2026

From the #StemCellEvidence Alert:

Modeling donor age and type trade-offs for personalized selection in allogeneic hematopoietic cell transplantation with post-transplant cyclophosphamide prophylaxis
by Mehta et al.
#MedSky

Image showing a PICO summary for: Re-Evaluating Donor Selection Priorities for Hematopoietic Cell Transplantation With Post-Transplant Cyclophosphamide by Mehta et al. featured in the Stem Cell Evidence Alert - February 2026

New from the #StemCellEvidence Alert:

Re-Evaluating Donor Selection Priorities for Hematopoietic Cell Transplantation With Post-Transplant Cyclophosphamide
by Mehta et al.

#MedSky

Image showing a PICO summary for: Donor age and type in allogeneic haematopoietic stem cell transplantation: Strong effect in MDS, limited in AML by Kim et al. featured in the Stem Cell Evidence Alert - February 2026

From the #StemCellEvidence Alert:

Donor age and type in allogeneic haematopoietic stem cell transplantation: Strong effect in MDS, limited in AML
by Kim et al.
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#MedSky

Image showing a PICO summary for: Harnessing HLA Divergence for Improved Donor Selection in Haploidentical Haematopoietic Stem Cell Transplantation by Cordeiro et al. featured in the Stem Cell Evidence Alert - February 2026

New from the #StemCellEvidence Alert:

Harnessing HLA Divergence for Improved Donor Selection in Haploidentical Haematopoietic Stem Cell Transplantation
by Cordeiro et al.
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#MedSky

Image showing a PICO summary for: RBC Alloimmunization Impacts Pediatric Sickle Cell Disease Transplant Outcomes and Transfusion Burden: A STAR Registry Report by Inam et al. featured in the Stem Cell Evidence Alert - February 2026

From the #StemCellEvidence Alert:

RBC Alloimmunization Impacts Pediatric Sickle Cell Disease Transplant Outcomes and Transfusion Burden: A STAR Registry Report
by Inam et al.
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#MedSky

Image showing a PICO summary for: The impact of DNMT3A mutation on survival of AML patients receiving allotransplant in first remission depends on the karyotype and co-occurring mutations Abou Dalle et al. featured in the Stem Cell Evidence Alert - February 2026

New from the #StemCellEvidence Alert:

The impact of DNMT3A mutation on survival of AML patients receiving allotransplant in first remission depends on the karyotype and co-occurring mutations
by Abou Dalle et al.
www.stemcellevidence.com/alerts/artic...
#MedSky