Thymoglobulin (anti-thymocyte globulin (rabbit)) / Sanofi - LARVOL DELTA

Reduced-Dose Rabbit Anti-Thymocyte Globulin for GVHD Prophylaxis Following Haploidentical Peripheral Blood Stem Cell Transplantation with Reduced-Intensity Conditioning: A Single-Center Retrospective Analysis (HOPA 2026) - "Post-transplant cyclophosphamide (PTCy) has improved outcomes in haploHSCT by reducing GVHD incidence while preserving survival; however, rates of acute and chronic GVHD remain as high as 41% and 31%, respectively...Banner MD Anderson Cancer Center (BMDACC) utilizes a novel GVHD prophylaxis regimen combining PTCy, tacrolimus, sirolimus, and reduced-dose, non-pharmacokinetically driven rATG (1 mg/kg) administered as a single dose on day +5 post-haploHSCT following RIC...RIC regimens consisted of fludarabine and melphalan (70-140 mg/m2), with or without thiotepa (2.5-10 mg/kg)...Exclusion criteria included prior alloHSCT, prior autologous HSCT within two years, receipt of abatacept, or left ventricular ejection fraction (LVEF) <45% at the time of transplant, which would preclude PTCy utilization... To be determined upon study completion."

Preclinical • Retrospective data • Acute Graft versus Host Disease • Bone Marrow Transplantation • Chronic Graft versus Host Disease • Graft versus Host Disease • Hematological Malignancies • Immunology • Infectious Disease • Myelofibrosis • Transplantation

Impact of Adding Abatacept to Antithymocyte Globulin for Acute Graft-Versus-Host Disease Prophylaxis in Matched Unrelated Donor Hematopoietic Stem Cell Transplantation (HOPA 2026) - "Rabbit antithymocyte globulin (rATG) reduces the incidence of GVHD by depleting circulating T cells prior to donor engraftment and may be used as a component of prophylaxis in the matched unrelated donor setting. Prior to 2022, per center-specific guidelines, rATG was administered with calcineurin inhibitor and methotrexate prophylaxis for matched unrelated donor transplantation...Exclusion criteria include the use of clofarabine for pre-transplant conditioning, use of post-transplant cyclophosphamide, umbilical cord cell source, and primary graft failure. Conclusions pending."

Acute Graft versus Host Disease • Acute Myelogenous Leukemia • Aplastic Anemia • Bone Marrow Transplantation • Chronic Graft versus Host Disease • Cytomegalovirus Infection • Epstein-Barr Virus Infections • Graft versus Host Disease • Hematological Malignancies • Immunology • Leukemia • Myelodysplastic Syndrome • Transplantation

FREXERA: A Study to Investigate the Efficacy and Safety of Frexalimab Versus Tacrolimus in Adults Undergoing Kidney Transplantation (clinicaltrials.gov) - P2/3 | N=526 | Recruiting | Sponsor: Sanofi | Not yet recruiting ➔ Recruiting

Enrollment open • Immunology • Transplant Rejection • Transplantation

OUTCOME OF UNRELATED AND HAPLOIDENTICAL HSCT AMONG CHILDREN WITH MYELODYSPLASTIC SYNDROME (EBMT 2026) - "16 patients were treated using the αβ T-cell depletion platform, 17 patients were treated using post-transplant cyclophosphamide (PTCy), 3 – with conventional CNI-based GVHD prophylaxis (group characteristics, table 1).All patients had cytopenia: transfusion dependence, leukopenia with neutropenia moderate to severe, 5 patients had progression to acute myeloid leukemia (AML).17 cases had germline condition predisposing to MDS: 2 - Shwachman-Diamond syndrome (SDS), 2 - Severe Congenital Neutropenia, 2 unspecified primary immune deficiency, 1 patient due to Diamond-Blackfan anemia, 2 patients had Down syndrome, 1 patient had Noonan syndrome...5 patients with AML received various courses of hypomethylating agents.Preparative regimen included treosulfan 42 gr/m2, fludarabine 150 mg/kg, thiotepa 10 mg/kg or melphalan 140 mg/m2. Thymoglobulin 5mg/kg or ATGAM 100 mg/kg and rituximab 200mg/m2 were used in a proportion of cases.Post-transplant GVHD prophylaxis included for αβ..."

Clinical • Acute Myelogenous Leukemia • Bone Marrow Transplantation • Developmental Disorders • Genetic Disorders • Graft versus Host Disease • Hematological Disorders • Hematological Malignancies • Immunology • Infectious Disease • Leukemia • Leukopenia • Myelodysplastic Syndrome • Neutropenia • Primary Immunodeficiency • GATA1 • GATA2

MULTICENTER EXPERIENCE OF HSCT IN RAS-ASSOCIATED AUTOIMMUNE LEUKOPROLIFERATIVE DISEASE (RALD) – IEWP EBMT STUDY (EBMT 2026) - "Six patients received treosulfan/thiotepa-based myeloablative conditioning (MAC) and one busulfan-based MAC. Serotherapy included rabbit ATG in 4, alemtuzumab in 2 and none in 1. Four patients additionally received rituximab...For graft-versus-host disease (GVHD) prophylaxis, in haploidentitcal grafts TCRab/CD19 depletion was used in 3 and posttranspant cyclophosphamide with ruxolitinib in 1. In the remaining, ciclosporin alone and in combination with prednisolone/mycophenolate mofetil was used.Neutrophil engraftment occurred in 5 patients (median 16 days, range 11-24 days)...In second HSCT, conditioning regimens were switched to melphalan/cyclophosphamide and irradiation in two and to busulfan in 1 (after treosulfan) and to treosulfan in one (after busulfan)... HSCT is a feasible option to control symptoms of RALD. Our case series demonstrate a very high risk of graft failure, however, salvage HSCT is a viable option in these patients. Mixed chimerism may lead to..."

Clinical • Acute Graft versus Host Disease • Bone Marrow Transplantation • Chronic Graft versus Host Disease • Gastroenterology • Gastrointestinal Disorder • Glomerulonephritis • Graft versus Host Disease • Hematological Disorders • Immunology • Infectious Disease • Inflammation • Inflammatory Arthritis • Inflammatory Bowel Disease • Lupus • Lupus Nephritis • Myelodysplastic Syndrome • Nephrology • Neutropenia • Systemic Lupus Erythematosus • Thrombocytopenia • Transplant Rejection • Ulcerative Colitis • KRAS • NRAS

OUTCOMES OF ALLOGENEIC HSCT IN NIJMEGEN BREAKAGE SYNDROME WITH TREOSULFAN-BASED CONDITIONING REGIMEN - A MULTICENTER STUDY (EBMT 2026) - "We recently demonstrated experience of treosulfan 30g/m2 versus low doses of busulfan...All received fludarabine 150mg/m2, 24 cyclophosphamide (22–40mg/kg, 1–30mg/kg, 1–20mg/kg). In 37 patients, rabbit ATG (thymoglobulin, Genzyme) 3-7,5mg/kg, in 1 patient horse ATG (ATGAM) 100mg/kg, and in 2 patients no serotherapy was used.In 25 patients matched unrelated (19–10/10, 6–9/10 HLA-matched), in 9 mismatched related, in 6 matched sibling donor was used... Both doses of treosulfan 21g/m2 and 30g/m2 were well tolerated by NBS patients. However, lower dose of 21g/m2 was associated with higher risk of mixed chimerism and leukemia relapse versus increased incidence of secondary malignancy after higher dose of 30g/m2."

Clinical • Acute Graft versus Host Disease • Bone Marrow Transplantation • Chronic Graft versus Host Disease • Graft versus Host Disease • Hematological Malignancies • Immunology • Infectious Disease • Leukemia • Lymphoma • Metabolic Disorders • Primary Immunodeficiency • Rhabdomyosarcoma • Sarcoma • Solid Tumor • Transplant Rejection

T CELL-DEPLETED HAPLOIDENTICAL AND MATCHED UNRELATED DONOR HEMATOPOIETIC STEM CELL TRANSPLANTATION REPRESENT SAFE AND EFFICIENT THERAPEUTIC APPROACHES FOR PEDIATRIC PATIENTS WITH BONE MARROW FAILURE SYNDROMES (EBMT 2026) - "Five patients received a conditioning regimen based on fludarabine and cyclophosphamide (FC) and three fludarabine, thiotepa, treosulfan (FTT), along with ATG-Grafalon® or thymoglobulin. One SD patient received a MUD-PBSC, conditioned with fludarabine, thiotepa and melphalan (FTM). Immunosuppression (IST) consisted of tacrolimus (or cyclosporine) and mycophenolate mofetil (MMF) in all nine patients...One case of veno-occlusive disease (VOD) in a T-haplo-HSCT patient with FA resolved completely after treatment with defibrotide... This single-center, retrospective series supports feasibility and safety of T-haplo-HSCT in rare BMF syndromes and indicates favorable outcomes in T-haplo-HSCT with fast engraftment, low GVHD, and a reduced risk of severe toxicity compared to MUD-HSCT in children."

Clinical • Acute Graft versus Host Disease • Aplastic Anemia • Bone Marrow Transplantation • Chronic Graft versus Host Disease • Dermatitis • Genetic Disorders • Graft versus Host Disease • Hematological Disorders • Hepatology • Immunology • Mucositis • Neutropenia • Pediatrics • Transplantation • CD34

HLA-MATCHED RELATED DONOR TRANSPLANTATION IN CHILDREN WITH TRANSFUSION-DEPENDENT THALASSAEMIA: A SINGLE-CENTRE EXPERIENCE IN A RESOURCE-LIMITED SETTING – LADY RIDGEWAY HOSPITAL FOR CHILDREN, SRI LANKA (EBMT 2026) - "Class 1 and 2 children received oral Busulfan/Cyclophosphamide either with rabbit or horse Antithymocyte globulin. Class 3 received Thioptepa/Treosulfan/Fludarabin.All received graft vs host disease prophylaxis with Intravenous Cyclosporin and short course of Methotrexate... This report represents a single-centre experience of HLA-matched sibling donor HSCT in children with TDT in a government-funded transplant centre in Sri Lanka. Our findings confirm that HLA-matched related donor HSCT is a safe, and cost-efficient curative option for children with TDT, even in low-resource settings. Careful patient selection, appropriate conditioning regimens,and experienced multidisciplinary care, enabled excellent outcomes.Scaling up access to HSCT for thalassaemia in Sri Lanka and similar settings will require increased infrastructure, training, and early identification of suitable patients before irreversible organ damage occurs."

Clinical • Bone Marrow Transplantation • Gene Therapies • Graft versus Host Disease • Immunology • Transplantation • CD34

POST-TRANSPLANT CYCLOPHOSPHAMIDE VERSUS ATG AS GVHD PROPHYLAXIS IN MATCHED SIBLING DONOR PBSC TRANSPLANTATION FOR Β-THALASSEMIA MAJOR – A SINGLE CENTER EXPERIENCE (EBMT 2026) - " Aim: To evaluate whether PT-Cy can be used(in cost-constrained countries)as an alternative to ATG for GvHD prophylaxis in MSD-PBSC-HCT for TDT, focusing on Engraftment, Thalassemia-Free Survival and GvHDStudy design: In a comparative study, a prospective group of 41 consecutive patients transplanted between August 2023-January 2025(Group A) with TDT who underwent MSD-PBSC-HCT, conditioned with BU/CY and GvHD prophylaxis using PT-CY 40mg/kg/day(Day+3,D+4) plus CSA were compared to a retrospective historical control of 55 consecutive patients transplanted between 2021-2023(Group B),conditioned with BU/CY+rabbit ATG 7mg/kg/day(Day-3-Day-1)and GvHD prophylaxis using CSA/MTX. Replacing ATG by PT-CY has significantly reduced aGvHD/cGvHD risks together with reducing CMV reactivation while maintaining comparable OS. However, the higher rates of MC and late graft rejection were observed. The clinically relevant trend toward lower TFS and graft instability in the PT-Cy group..."

Clinical • Post-transplantation • Acute Graft versus Host Disease • Beta-Thalassemia • Bone Marrow Transplantation • Chronic Graft versus Host Disease • Genetic Disorders • Graft versus Host Disease • Hematological Disorders • Immunology • Infectious Disease • Transplant Rejection • Transplantation

HAPLOIDENTICAL STEM CELL TRANSPLANTATION AS A CURATIVE STRATEGY FOR SEVERE SICKLE CELL DISEASE: A SINGLE-CENTER REPORT (EBMT 2026) - "We report our single-center experience using post-transplant cyclophosphamide (PTCy) platform...Conditioning: thiotepa, fludarabine, rabbit antithymocyte globulin (ATG) and 2 Gy TBI, except for the first patient, who received treosulfan. Graft versus host disease (GVHD) prophylaxis: mycophenolate mofetil with tacrolimus or sirolimus and PTCy, abatacept was added in the last two patients. All patients received hydroxyurea before transplantation and peri-transplant transfusion support. The first patient additionally received azathioprine for two months andrequired rituximab and plasmapheresis due to alloimmunization...Three patients developed overlap syndrome with intestinal involvement (no biopsy confirmation in 66.7% cases), treated with photopheresis and ruxolitinib: one is off treatment, one is tapering immunosuppression, and one remains on immunosuppressive therapy... Haplo-HSCT using PTCy was feasible in this small cohort of adolescents with severe SCD, resulting in..."

Clinical • Acute Graft versus Host Disease • Acute Kidney Injury • Bone Marrow Transplantation • Cardiovascular • Chronic Graft versus Host Disease • CNS Disorders • Epilepsy • Genetic Disorders • Graft versus Host Disease • Hemophagocytic lymphohistiocytosis • Hypertension • Immunology • Mucositis • Nephrology • Rare Diseases • Sickle Cell Disease • Subarachnoid Hemorrhage • Transplant Rejection • Transplantation

MMR SEROLOGIC STATUS IN MULTIPLE SCLEROSIS AFTER AUTOLOGOUS STEM CELL TRANSPLANTATION (EBMT 2026) - "Background: Multiple sclerosis (MS) patients undergoing autologous stem cell transplantation (ASCT) after carmustine, cytarabine, etoposide, melphalan (BEAM) or cyclophosphamide (Cy) chemotherapy plus rabbit anti-thymocyte globulin (rATG) are considered as naïve to vaccines and offered revaccination; live vaccines as measles/mumps/rubella (MMR) are not recommended before 24 months from ASCT with concern about vaccine-induced infectious disease...The two patients who lost either MMR (ID23) or rubella immunity (ID35) had both low titles at baseline; prior to ASCT, they had received interferon and 3 lines of therapy (interferon, fingolimod, ocrelizumab), respectively... In our real-life population of ASCT-treated MS, the vast majority of patients retained MMR immunity with a slight decline of vaccine titres over time confirming that MMR revaccination could be safely postponed after 24 months from ASCT and probably offered only in selected patients at high risk (i.e...."

CNS Disorders • Infectious Disease • Measles • Multiple Sclerosis • Mumps • Rubella • Transplantation

HETEROGENEOUS NON-HEMATOLOGIC AUTOIMMUNE COMPLICATIONS AFTER ALLOGENEIC HEMATOPOIETIC STEM CELL TRANSPLANTATION IN PEDIATRIC LEUKEMIA: INCIDENCE, SPECTRUM, AND OUTCOMES IN A SINGLE-CENTER COHORT (EBMT 2026) - "Observed ADs included Nephrotic Syndrome (n=3), Autoimmune Hepatitis accompanied by nephrotic syndrome (n=1), Vitiligo (n=2), Scleroderma (n=1), and Transverse myelitis (n=1).All patients underwent a non-TBI myeloablative conditioning regimen (Busulfan, Cyclophosphamide, +/- rabbit-ATG)...cGVHD was significantly higher in the Autoimmune Group (75% vs. 36.4%, p=0.05).Three patients with nephrotic syndrome were treated with systemic steroids and Mycophenolate in 2 cases, and cyclosporin in 1. The patient with autoimmune hepatitis and nephrotic syndrome received steroids, tacrolimus, and azathioprine. Two patients with vitiligo, initially resistant, were finally treated with prednisolone and Ruxolitinib...The patient with Scleroderma was treated with systemic steroids and sirolimus... Post-HSCT non-hematologic ADs in pediatric leukemia patients are diverse and differ from those in non-malignant diseases. The rate of AD incidence is consistent with prior research findings...."

Clinical • Heterogeneity • Autoimmune Hepatitis • Bone Marrow Transplantation • Chronic Graft versus Host Disease • CNS Disorders • Cytomegalovirus Infection • Glomerulonephritis • Graft versus Host Disease • Hepatology • Immunology • Nephrology • Pediatrics • Scleroderma • Systemic Sclerosis • Transplant Rejection • Transplantation • Vitiligo

AUTOLOGOUS STEM CELL TRANSPLANTATION FOR ANTI-SYNTHETASE SYNDROME: LONG-TERM OUTCOMES FROM TWO REFRACTORY CASES (EBMT 2026) - "Over the following 18 months, he experienced multiple relapses despite sequential immunosuppressive strategies including mycophenolate mofetil, rituximab, ciclosporin, tacrolimus, and repeated corticosteroid pulses...Disease response to various treatments is summarised in Figure 1.Figure 1: Creatine Kinase trend with treatmentsCase 2: A 46-year-old woman with Jo-1–positive ASSD initially responded to glucocorticoids, cyclophosphamide, and hydroxychloroquine but experienced multiple relapses requiring escalation to IVIg, tofacitinib, and rituximab...Two later flares were controlled with lower-dose prednisone, maintenance IVIg, and six-monthly rituximab... Our cases support ASCT as a viable option for selected ASSD patients amid growing interest in advanced cellular therapies such as CAR-T. While CAR-T shows promise, its long-term outcomes and accessibility remain uncertain. In contrast, ASCT offers multi-year follow-up data and comparatively lower cost, at approximately..."

Clinical • IO biomarker • Immunology • Interstitial Lung Disease • Myositis • Pulmonary Disease • Rare Diseases • Respiratory Diseases • Transplantation • CD4 • CD8

A SYSTEMATIC REVIEW OF REAL-WORLD EVIDENCE ON THERAPIES FOR MODERATE APLASTIC ANAEMIA (EBMT 2026) - "Treatment choices depend on blood counts, transfusion dependency or other significant symptoms and include supportive care, monotherapy (cyclosporine [CSA], tacrolimus, daclizumab, thrombopoietin receptor agonists [TPO-RA], androgens), double or triple therapy, (combining horse or rabbit anti-thymocyte globulin [hATG or rATG], CSA/tacrolimus, and eltrombopag [EPAG]/TPO-RA or androgens), and/or haematopoietic stem-cell transplantation (HSCT)... Across heterogenous studies, OS and ORR have been documented for monotherapies, double, and triple therapies; HSCT was only reported in small, heterogeneous cohorts. Due to varied timepoints, missing data, and cohort selection, there was insufficient homogenous data for quantitative analysis. This SLR demonstrates a clear need for uniform definition of mAA and for comparative, prospective studies to enable the comparison of therapeutic effectiveness and to develop sufficient treatment algorithms."

Clinical • HEOR • Real-world • Real-world evidence • Review • Anemia • Aplastic Anemia • Bone Marrow Transplantation • Hematological Disorders

HIGH CD34⁺ CELL DOSE SIGNIFICANTLY REDUCES EBV REACTIVATION AND IMPROVES SURVIVAL IN APLASTIC ANEMIA PATIENTS UNDERGOING UCBT (EBMT 2026) - "ATG formulations included Rabbit Anti-human T Lymphocyte Immunoglobulin (ATLG) (14.3%) and Rabbit Anti-human Thymocyte Immunoglobulin (rATG) (85.7%)... Infused CD34⁺ dose >0.16×10⁶/kg emerged as the strongest independent predictor of reduced early EBV reactivation and showed favorable trends in long-term survival in pediatric AA undergoing UCBT. This data-driven cutoff remained robust in multivariable analyses and was accompanied by a modest advantage in late CD4⁺ immune recovery."

Clinical • Anemia • Aplastic Anemia • Graft versus Host Disease • Immunology • CD14 • CD8

THIOTEPA-BASED BUSULFAN-SPARING CONDITIONING OPTIMIZES ENGRAFTMENT AND MITIGATES CHRONIC GVHD IN PEDIATRIC HAPLOIDENTICAL HSCT FOR SAA: A MATCHED CASE-CONTROL STUDY (EBMT 2026) - "All patients received a standard conditioning backbone of Fludarabine, Cyclophosphamide, and Rabbit ATG. Thiotepa intensification facilitates a Busulfan-sparing strategy that ensures rapid, stable engraftment and significantly reduces chronic GVHD in pediatric SAA. Despite a signal for manageable endothelial toxicity, this regimen establishes a compelling new paradigm with a superior efficacy-safety profile, effectively trading short-term, reversible toxicity for enhanced long-term immunological recovery and survival. These findings strongly support the design of prospective, randomized trials to validate this regimen as a standard of care."

Clinical • Acute Graft versus Host Disease • Aplastic Anemia • Bone Marrow Transplantation • Chronic Graft versus Host Disease • Graft versus Host Disease • Hematological Disorders • Hepatology • Immunology • Liver Failure • Pediatrics

IMPACT OF EBV-DRIVEN POST-TRANSPLANT LYMPHOPROLIFERATIVE DISEASE (EBV-PTLD) ON DONOR T-CELL CHIMERISM IN ALLOGRAFT RECIPIENTS RECEIVING THYMOGLOBULIN AS GVHD PROPHYLAXIS (EBMT 2026) - "GVHD prophylaxis consisted of thymoglobulin 4.5 mg/kg in divided doses (days –3 to –1), predominantly with methotrexate and cyclosporine (n=395)... csEBVR occurred in 161 patients (40%), managed with immunosuppression reduction alone (n=54) and/or rituximab (n=107)... EBV-driven PTLD is independently associated with loss of donor T cell engraftment, conferring four times the risk of major chimerism loss. While the causal mechanism remains unclear, subsequent CD3⁺ chimerism status—rather than PTLD itself—is a strong independent predictor of survival. These findings highlight the critical importance of intensive chimerism monitoring in EBV-driven PTLD post allograft"

Post-transplantation • Graft versus Host Disease • Immunology • Respiratory Diseases • Transplantation

HARMONIZING HAPLOIDENTICAL STRATEGIES: A FOUR-YEAR COMPARATIVE ANALYSIS OF T-CELL–DEPLETED AND REPLETE PEDIATRIC TRANSPLANTS FROM A DEVELOPING COUNTRY (EBMT 2026) - "Advances such as TCRαβ/CD19 depletion (TCD) and post-transplant cyclophosphamide (PTCy)-based T-cell–replete (TCR) platforms have significantly reduced immunologic toxicity, yet comparative pediatric data from India remain sparse...Rabbit ATG was administered to all, with additional rituximab in TCD recipients... Pediatric haploidentical HSCT using either TCD or PTCy-based TCR approaches yields robust engraftment and acceptable toxicity. However, TCRαβ-depleted haplo-HSCT provides the most favorable profile, with markedly reduced GVHD, no significant increase in relapse rate, minimal TRM, and excellent survival. These results support broader adoption of TCD haplo-HSCT in resource-limited settings and warrant future comparative studies with matched unrelated donor transplants."

Clinical • Acute Graft versus Host Disease • Aplastic Anemia • Beta-Thalassemia • Bone Marrow Transplantation • Chronic Graft versus Host Disease • Genetic Disorders • Graft versus Host Disease • Immunology • Infectious Disease • Juvenile Myelomonocytic Leukemia • Pediatrics • Primary Immunodeficiency • Sickle Cell Disease • Transplantation

TREOSULFAN-FLUDARABINE AS A REDUCED INTENSITY CONDITIONING ALTERNATIVE TO BUSULFAN-FLUDARABINE FOR ADULT ALLOGENEIC STEM CELL TRANSPLANTATION: SINGLE-CENTER, EARLY REAL-LIFE CLINICAL AND ECONOMICAL COMPARATIVE OUTCOMES (EBMT 2026) - " We retrospectively reviewed all allo-HCTs performed between 2021 and 2025 using FT10 or FB2 conditioning, both combined with anti-lymphocyte globulin ((ATG) Thymoglobulin 5 mg/kg or Grafalon 30 mg/kg), for adults with AML or MDS. Transplant practices were identical across the study period: peripheral blood stem cell grafts, ciclosporine + methotrexate for GVHD prophylaxis, letermovir prophylaxis, and unchanged center-level procedures and teams... Despite being used in a substantially higher-risk population, FT10 achieved early post-transplant outcomes comparable to FB2, with a lower burden of early unplanned healthcare costs. These findings support the feasibility and cost effectiveness of FT10 as a reduced-toxicity RIC option for AML/MDS."

Clinical • Acute Graft versus Host Disease • Graft versus Host Disease • Hepatology • Immunology • Transplantation

HAPLOIDENTICAL VERSUS FULLY MATCHED RELATED DONOR STEM CELL TRANSPLANTATION IN PEDIATRIC AND YOUNG ADULTS WITH SEVERE APLASTIC ANEMIA: A SINGLE-CENTER EXPERIENCE FROM A RESOURCE-LIMITED SETTING (EBMT 2026) - "The single patient previously treated with rabbit ATG and cyclosporine developed posterior reversible encephalopathy syndrome (PRES), which resolved with supportive management and treatment adjustment.Sixteen transplants were performed in 15 patients (median age 7 years; range 3–20; males n=8)...Conditioning consisted of fludarabine (150 mg/m²), cyclophosphamide (29 mg/kg), antithymocyte globulin (2.5 mg/kg), and TBI (4 Gy) for haploidentical transplants, while fully matched related donor transplants received reduced-dose TBI. GVHD prophylaxis included a calcineurin inhibitor and methotrexate for matched related donors, and post-transplant cyclophosphamide with a calcineurin inhibitor and mycophenolate mofetil for haploidentical donors...Day +30 chimerism (n=12) demonstrated high donor fractions (median ~99%).Acute GVHD occurred in two haploidentical recipients: one with hyperacute steroid-refractory gastrointestinal GVHD successfully treated with etanercept and..."

Clinical • Acute Graft versus Host Disease • Anemia • Aplastic Anemia • Chronic Graft versus Host Disease • CNS Disorders • Graft versus Host Disease • Hematological Disorders • Immunology • Infectious Disease • Pediatrics • Transplantation • DDX41

IMPORTANCE OF GRAFT COMPOSITION ON EBV REACTIVATION RISK FOLLOWING MODERN GVHD PROPHYLAXIS (EBMT 2026) - "Patients received either rabbit ATG or post-transplant cyclophosphamide for GVHD prophylaxis...Rituximab (RTX) had no impact on OS or PFS but was associated with sustained B-cell depletion, hypogammaglobulinemia, impaired hepatitis B immunity after vaccination with less patients achieving protective anti-HBsAg antibody levels (≥ 10 IU/mL) (p = 0.0176) and reduced anti-HBsAg antibody titers (p = 0.0064)... A high CD19+/CD8+ ratio in infused grafts was associated with a higher risk of high-level EBV reactivation in ATG-based GVHD prophylaxis but not after PTCy, highlighting distinct immune regulation and depletion patterns, as well as divergent early T-cell reconstitution dynamics. This new predictive factor may help identify patients who might benefit from more aggressive management such as increased monitoring frequency, lower treatment threshold, or even primary prophylaxis."

Acute Graft versus Host Disease • Chronic Graft versus Host Disease • Epstein-Barr Virus Infections • Graft versus Host Disease • Hepatitis B • Hepatology • Immunology • Infectious Disease • Inflammation • CD4 • CD8

TRANSPLANTING THROUGH UNCERTAINTY: SINGLE CENTER EXPERIENCE OF HEMATOPOIETIC STEM CELL TRANSPLANTATION IN CHILDREN WITH SEVERE APLASTIC ANEMIA AND POSSIBLE PULMONARY INVASIVE FUNGAL INFECTION (EBMT 2026) - "Two patients received matched sibling donor HSCT conditioned with Cyclophosphamide (200 mg/kg) and rATG (7.5 mg/kg), while the other two underwent T-replete haploidentical HSCT with post-infusion Cyclophosphamide (100 mg/kg) and conditioning with rATG (5 mg/kg), Fludarabine (150 mg/kg), Cyclophosphamide (29 mg/kg) and 300 cGy single fraction total body irradiation... Our findings suggests that proceeding with HSCT in carefully selected pediatric patients with SAA and radiological evidence of possible pulmonary IFI is feasible and associated with favorable outcomes. Customized HSCT and antifungal strategies appear to be key factors in mitigating infection-related risks. Larger multicenter pediatric studies are needed to better define risk stratification and optimize management strategies in this high-risk population."

Clinical • Anemia • Aplastic Anemia • Bone Marrow Transplantation • Hematological Disorders • Infectious Disease • Transplantation

FLUDARABINE AUC EXPOSURE IS NOT PREDICTIVE OF MORTALITY IN THE CONTEXT OF FLUDARABINE BUSULFAN AND ATG CONDITIONED TRANSPLANTATION (EBMT 2026) - "We evaluated the association between Flu AUC and outcomes in patients receiving a uniform reduced-intensity conditioning (RIC) regimen of Flu/Busulfan/rabbit anti-thymocyte globulin (Flu/Bu/rATG), using a population pharmacokinetic (popPK) model to assess whether AUC-guided dosing could provide clinical benefit...Graft-versus-host disease (GVHD) prophylaxis included ciclosporin and mycophenolate mofetil... In this single-centre cohort treated with homogeneous Flu/Bu/rATG RIC, we did not identify a Flu AUC threshold associated with increased NRM or inferior OS, contrasting with studies in myeloablative, T-replete or haplograft settings. These findings suggest that in RIC regimens incorporating rATG, optimising Flu exposure alone may not substantially influence outcomes. Further validation in external cohorts is needed before adopting AUC-guided Flu dosing in this context."

Graft versus Host Disease • Hematological Malignancies • Immunology • Transplant Rejection • Transplantation • EGFR

A SIMULATED PK/PD INVESTIGATION OF RATG, FLUDARABINE, AND THIOTEPA IN HAPLO-IDENTICAL TRANSPLANT FOR SICKLE CELL DISEASE; A VANDERBILT GLOBAL HAPLO-IDENTICAL BMT LEARNING COLLABORATIVE (VGC2) ANALYSIS (EBMT 2026) - "They received the reduced-toxicity conditioning including rabbit anti-thymocyte globulin [rATG], Thiotepa [TT], Fludarabine, and 200cGy TBI. GVHD prophylaxis consisted of post-HCT cyclophosphamide (PTCy), sirolimus, and mycophenolate mofetil... This preliminary analysis suggests that pre-HCT rATG underexposure is associated with GF, while post-HCT rATG overexposure is associated with cGVHD. Inadequate recipient immune-ablation, and interference of rATG with alloreactive T-cell depletion by PTCy might be the cause for this. A larger dataset, potentially including patients from the BMT-CTN 1507 trial, is needed to confirm these findings."

Clinical • PK/PD data • Chronic Graft versus Host Disease • Genetic Disorders • Graft versus Host Disease • Hematological Disorders • Immunology • Sickle Cell Disease • Transplantation

IMLIFIDASE EFFICIENTLY ABROGATES THE EFFECT OF ATLG AND THYMOGLOBULIN ON T- AND NK-CELL VIABILITY AND FUNCTION (EBMT 2026) - "Background: ATLG (Grafalon) and Thymoglobulin are long-established agents in allogeneic hematopoietic stem cell transplantation (allo-HSCT), effectively preventing both graft rejection and graft-versus-host disease (GvHD)... Imlifidase markedly attenuates the impact of ATLG/Thymoglobulin on NK-cell function and abrogates ATLG-/Thymoglobulin-mediated cytotoxicity on T-/NK-cells. This effect of imlifidase is to be evaluated in a clinical study in the context of T-/B-cell-depleted allo-HSCT in order to determine whether pre-transplant application of imlifidase (e.g. on day-2) results in effective inactivation of ATLG/Thymoglobulin in vivo and thereby accelerates post-transplant immune reconstitution."

Bone Marrow Transplantation • Graft versus Host Disease • Immunology • Transplant Rejection • IL2 • NCAM1