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

Effect of post-transplant cyclophosphamide on outcomes in stem cell transplant recipients with myelofibrosis (ASH 2025) - "Our analysis shows that pts with MMD had significantly higher risk of death, disease relapse, and NRM than those with MD. In addition, pts with KPS<80 had significantly higher risk of developing aGVHD. With a limited number of pts who received post-transplant cyclophosphamide, we did not observe a significant difference in aGVHD, cGVHD, NRM, relapse, RFS or OS compared to other GVHD prevention strategies."

Clinical • Post-transplantation • Acute Graft versus Host Disease • Bone Marrow Transplantation • Chronic Graft versus Host Disease • Graft versus Host Disease • Immunology • Myelofibrosis • Transplantation

Outcomes of allogeneic hematopoietic stem cell transplantations (HSCT) for treatment of secondary primary malignancies arising post-autologous HSCT for multiple myeloma (ASH 2025) - "As conditioning regimens, 12 patients received Fludarabine/Busulfan, and 5 patients received Fludarabine/Melphalan...For GVHD prophylaxis, 6 patients received Cyclophosphamide, Tacrolimus, and Mycophenolate Mofetil (MMF); 9 patients received Thymoglobulin, Tacrolimus, and MMF; 1 patient received Thymoglobulin, Sirolimus, and MMF; 1 patient received Tacrolimus, Methotrexate, and Abatacept...Limitations of the outcome data include a relatively small population that underwent Allo-HCST, likely lower than the true number of patients with SPM following Auto-HSCT due to them either not returning to the center or opting against Allo-HSCT for treatment. This data adds to the collective understanding of the associated risks and benefits of Allo-HSCT for treatment of hematological SPM following Auto-HSCT for MM."

Acute Graft versus Host Disease • Acute Lymphocytic Leukemia • Acute Myelogenous Leukemia • Bone Marrow Transplantation • Chronic Graft versus Host Disease • Graft versus Host Disease • Hematological Malignancies • Immunology • Leukemia • Multiple Myeloma • Myelodysplastic Syndrome • Oncology • Transplantation • TP53

Epidemiology, risk factors, and outcomes of cytomegalovirus (CMV) reactivation in allogeneic hematopoietic stem cell transplantation: A retrospective observational single-centre study from western India (ASH 2025) - "While pre-emptive therapy has reduced CMV disease, reactivation remains common, particularly in alternate donor transplants and resource-limited settings where access to newer antivirals like letermovir is limited...Serotherapy type did not impact overall reactivation, but early reactivation was significantly higher with Grafalon vs. Thymoglobulin (90% vs. 33.6%, p<0.001)...Adverse effects included neutropenia (ganciclovir/valganciclovir) and nephrotoxicity (foscarnet/cidofovir)... This single-centre study demonstrates a high CMV reactivation rate (89.7%) in a high-seroprevalence Indian cohort, especially in alternate donor and PTCy-treated patients. While CMV disease was rare due to effective surveillance and pre-emptive therapy, recurrence and antiviral toxicity were common. The type of serotherapy influenced early reactivation risk."

Retrospective data • Acute Graft versus Host Disease • Bone Marrow Transplantation • Cytomegalovirus Infection • Gastrointestinal Disorder • Graft versus Host Disease • Hematological Disorders • Immunology • Neutropenia • Transplantation

A novel reduced-toxicity conditioning regimen with busulfan/fludarabine/cyclophosphamide/anti-thymocyte globulin for severe aplastic anemia (ASH 2025) - "The conditioning regimen comprised Bu (0.8 mg/kg every 6 hours, 1 day, weight-adjusted dose for pediatric patients), Flu (30 mg/m²/day, 4 days), Cy (500 mg/m²/day, 4 days), and ATG with Thymoglobulin 2 mg/kg/day, 4 days in 3 patients or ATG-Fresenius 5 mg/kg/day, 4 days in 7 patients. Graft-versus-host disease (GVHD) prophylaxis included cyclosporine and mycophenolate mofetil for all patients, supplemented with either: short-term methotrexate(MTX, +1d 15mg/m², +4d, +8d, +11d 10mg/m², n=2); only CD25 monoclonal antibody(Basiliximab, +3d 20mg, n=1); or both(MTX as above plus Basiliximab, +3d 20mg, n=2; or MTX as above plus Recombinant humanized anti-CD25 monoclonal antibody, +4d, +8d, 1mg/kg, n=5)...Majority of the patients are with good quality of life. Longer follow-up and larger series are needed to evaluate fertility and transplant outcomes with current protocol."

Acute Graft versus Host Disease • Anemia • Aplastic Anemia • Bone Marrow Transplantation • Chronic Graft versus Host Disease • Graft versus Host Disease • Immunology

Transplant outcomes for children and adolescents with severe aplastic anemia comparing matched sibling donor and haploidentical transplant approaches. (ASH 2025) - "Introduction Recent studies comparing matched sibling donor (MSD) and haploidentical donor with post-transplant cyclophosphamide (haplo-PTCy) hematopoietic stem cell transplantation (HSCT) for children and adolescents with severe aplastic anemia (SAA) show promising results...GVHD prophylaxis included cyclosporine A (3–5 mg/kg) and mini-methotrexate...The conditioning regimen included ATG 4.5 mg/kg, fludarabine 150 mg/m², and total body irradiation 400 cGy. GVHD prophylaxis consisted of Cy 50 mg/kg on +3 and +4, followed by tacrolimus 0.06 mg/kg and mycophenolate mofetil 30 mg/kg starting on +5...Discussion and Conclusions Health-related quality of life outcomes is comparable between the two approaches, with no significative differences in overall survival between both transplant models. These results suggest that haplo-HSCT with PT-Cy is a viable alternative when an MSD is unavailable and may open the possibility of further research studies in which this transplant..."

Clinical • Acute Graft versus Host Disease • Anemia • Aplastic Anemia • Bone Marrow Transplantation • Cytomegalovirus Infection • Graft versus Host Disease • Hematological Disorders • Immunology • Infectious Disease • Respiratory Diseases • Transplantation

An evolving landscape – how changing conditioning regimes can improve myelofibrosis transplant outcomes. (ASH 2025) - "Of the 16 cases, seven received Fludarabine/Busulphan/Antithymocyte globulin (Flu/Bu/ATG) conditioning (Fludarabine 30mg/m 2 day -9 to day -5, Busulphan 3.2mg/kg on day -4 and day -3 and rabbit ATG 2.5mg/kg on day -2 and day -1), seven underwent Fludarabine/Cyclophosphamide (Flu/Cy) conditioning (Fludarabine 25mg/m 2 day -6 to day -2 and Cyclophosphamide 1g/m 2 on day -3 and day -2), and two received Fludarabine/Melphalan (Flu/Mel) (Fludarabine 25mg/m 2 day -6 to day -2 and Melphalan 100mg/m 2 on day -2)...Therapies included Ruxolitnib, Momelotinib, splenic irradiation, Hydroxycarbamide, Danazol, Interferon, Cyclophosphamide, Anagrelide, Thalidomide, Panobinostat and Navitoclax...Although limited by a small sample size, these findings indicate that the Flu/Bu/ATG conditioning regimen may be associated with improved outcomes, thereby supporting its adoption as the preferred approach for patients undergoing allogeneic stem cell transplantation for myelofibrosis as per BSH..."

Acute Graft versus Host Disease • Chronic Graft versus Host Disease • Graft versus Host Disease • Immunology • Infectious Disease • Myelofibrosis • Pneumonia • Respiratory Diseases • Transplantation • ASXL1 • CALR • JAK2 • SF3B1 • U2AF1

The efficacy and safety of using low-dose cyclophosphamide, fludarabine and thymoglobulin in allogeneic hematopoietic stem cell transplantation for transfusion-dependent non-severe aplastic anemia:a retrospective study. (ASH 2025) - "Low dose cyclophosphamide did not cause fatal adverse cardiac and liver toxicity, and did not affect theeffectiveness of hematopoietic engraftment .These data highlight allo-HSCT using low-dose Cy(100-120mg/kg),fludarabine and thymoglobulin(ATG)regimen is safe and effective for TD-NSAA. KeywordsTransfusion-dependent non-severe aplastic anemia; Hematopoietic stem cell transplantationCyclophosphamide."

Retrospective data • Acute Graft versus Host Disease • Anemia • Aplastic Anemia • Bone Marrow Transplantation • Graft versus Host Disease • Hematological Disorders • Immunology • Transplantation

A novel thiotepa-containing conditioning regimen for haploidentical hematopoietic cell transplantation in pediatric aplastic anemia at high risk of cardiotoxicity (ASH 2025) - "The conditioning regimen included: Busulfan 3.2 g/kg/day ondays -8 and -7; thiotepa 10 mg/kg/day on day -6; cyclophosphamide 20 mg/kg/day on day -5 to -2; rabbitanti-thymocyte globulin (rATG) 2.5 mg/kg/day on day -5 to -2. Five patients had mild pericardial effusion, with amedian onset of 3 days prior to infusion; none required medical intervention. Among the 20 patients, twodeveloped grade III-IV aGVHD, one developed bronchiolitis obliterans syndrome (BOS), one developedEpstein-Barr virus (EBV)-related post-transplant lymphoproliferative disorder (PTLD), and oneexperienced cytomegalovirus (CMV) infection.ConclusionOur study demonstrates that a thiotepa-containing conditioning regimen with reducedcyclophosphamide is safe and effective in pediatric AA at risk of cadiotoxicity."

Clinical • Acute Graft versus Host Disease • Anemia • Aplastic Anemia • Bone Marrow Transplantation • Cardiovascular • Cytomegalovirus Infection • Graft versus Host Disease • Hematological Disorders • Immunology • Infectious Disease • Pediatrics • Pulmonary Disease • Respiratory Diseases • Transplantation

Thiotepa and busulfan combined with cyclophosphamide conditioning regimen plus maintenance therapy improved the disease-free survival of patients with relapsed/refractory haematologic malignancies after undergoing allogeneic transplantation (ASH 2025) - "Rabbit ATG was added in haploid-identical and unrelated-matched donor transplantation. This study suggests that the TBC conditioning regimen may be a promising option forpatients with R/R haematologic diseases undergoing allo-HSCT."

Clinical • Bone Marrow Transplantation • Central Nervous System Leukemia • Graft versus Host Disease • Hematological Malignancies • Immunology • Infectious Disease • Leukemia • Lymphoma • Oncology • Transplantation

A US real-world analysis of demographics, transplant patterns, and survival outcomes in posttransplant lymphoproliferative disorder (PTLD) (ASH 2025) - "Other agents includedprednisone (6.2%), cyclosporine (5.8%), sirolimus (5.8%), dexamethasone (4.8%), azathioprine (3.1%),methylprednisolone (1.9%), everolimus (0.7%), ibrutinib (0.6%), ruxolitinib (0.6%), mycophenolate(0.3%), basiliximab (0.2%), and thymoglobulin (0.1%).Extranodal, bone marrow, and CNS involvement were present in 29.5%, 1.9%, and 1.8% of cases,respectively...Median OSfor heart transplant recipients was 2730 days, and for those who underwent HSCT, 2643 days.Regarding treatment, only 10 patients received bispecific antibodies (glofitamab, epcoritamab, ormosunetuzumab) and only 23 patients received CAR-T therapy. This study represents one of the largest real-world analyses of PTLD in adult patients inthe US, utilizing the TriNetX dataset spanning over three decades. This study represents one of the largest real-world analyses of PTLD in adult patients inthe US, utilizing the TriNetX dataset spanning over three decades. Improvement in OS in more recentyears..."

Clinical • Post-transplantation • Real-world • Real-world evidence • Bone Marrow Transplantation • Epstein-Barr Virus Infections • Hematological Malignancies • Hodgkin Lymphoma • Lymphoma • Solid Organ Transplantation • Transplantation

Real-world data on cardiovascular disease incidence in adults with acquired immune aplastic anemia. (ASH 2025) - "Patients had a median of 1 CVD risk factor (former/current smoker, BMI ≥30, hypertension,chronic kidney disease, hyperlipidemia, diabetes, hypo/hyperthyroidism) pre-AA.Treatment exposure included 81% receiving horse (n=60) or rabbit (n=2) anti-thymocyte globulin (ATG),83% cyclosporine (CSA), 58% eltrombopag (Epag), and 25% hematopoietic stem cell transplant (HSCT).Median times from AA to first ATG, CSA, and Epag were 6, 4, and 11 weeks, respectively. Arrhythmias appeared temporally linked to ATG and CSA exposure. The impactof CH, CE, and individual somatic mutations on IS, CAD, and PAD incidence remains unclear and warrantsfurther studies in larger cohorts."

Clinical • Real-world • Real-world evidence • Anemia • Aplastic Anemia • Bone Marrow Transplantation • Cardiovascular • Chronic Kidney Disease • Complement-mediated Rare Disorders • Congestive Heart Failure • Coronary Artery Disease • Diabetes • Dyslipidemia • Endocrine Disorders • Heart Failure • Hematological Disorders • Hematological Malignancies • Hypertension • Ischemic stroke • Metabolic Disorders • Myelodysplastic Syndrome • Myocardial Infarction • Nephrology • Paroxysmal Nocturnal Hemoglobinuria • Peripheral Arterial Disease • Rare Diseases • Renal Disease • BCOR • BCORL1 • CHEK2 • U2AF1

Prognostic significance of PNH clones in aplastic anemia treated with immunosuppression or allogeneic HSCT (ASH 2025) - "104(50.3%) patients received IST and 103 (49.7%) patients underwent HSCT as frontline therapy.In the IST cohort, the treatment included cyclosporine plus ATG (horse or rabbit) ± Eltombopag for 77patients (74%) and 27(26%) received other types of IST (Mycophenolate mofetil, danazol, steroid). AA patients with a PNH clone have improved survival with with IST compared to the AA patients without aPNH clone. Post-HSCT both cohorts have good survival, however patients with a PNH clone had lowerchronic GVHD incidence. Further studies to delineate the effect of PNH clone on GVHD incidence areneeded."

Acute Graft versus Host Disease • Anemia • Aplastic Anemia • Bone Marrow Transplantation • Chronic Graft versus Host Disease • Complement-mediated Rare Disorders • Graft versus Host Disease • Hematological Disorders • Immunology • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases

Excellent long-term outcomes after alemtuzumab-based hematopoietic cell transplantation (HCT) for severe aplastic anemia (SAA) and inherited bone marrow failure syndromes (IBMF): A 25-year British society of blood and marrow transplantation and cellular therapy (BSBMTCT) retrospective analysis of 1754 patients from the United Kingdom (UK) (ASH 2025) - "The FCC regimen (fludarabine, cyclophosphamideand alemtuzumab) with cyclosporin (without irradiation and methotrexate) for Graft-versus-Host Disease(GvHD) prophylaxis is UK standard of care due to excellent survival and low GvHD/rejection incidence... This is the largest report of BMF patients transplanted using a uniform alemtuzumab-basedregimen, with excellent 10-year survival, low rates of graft failure and GVHD in both SAA and IBMF. Theadded benefit of BM as a stem cell source was evident in children; future efforts in the adult cohortshould be directed toward improving outcomes in those >60yrs. Excellent OS and GFRS indicatealemtuzumab should be recommended in conditioning for all BMF syndromes."

Retrospective data • Acute Graft versus Host Disease • Anemia • Aplastic Anemia • Chronic Graft versus Host Disease • Genetic Disorders • Graft versus Host Disease • Hematological Disorders • Immunology • Transplantation

Aplastic anemia treatment in older adults in resource-limited settings: Can rabbit ATG bridge the gap? (ASH 2025) - "Background The treatment of choice for aplastic anemia (AA) is triple immunosuppressive therapy with horseanti-thymocyte globulin (h-ATG), cyclosporine (CsA), and eltrombopag (ETB), as it has shown toimprove overall response rates (ORR) and relapse-free survival, even in the small subgroup ofpatients ≥65 years old with ECOG ≤2. Surprisingly, CsA/ETB consistently showed higher ORR and longer OScompared to r-ATG. These findings call for a reassessment of the optimal treatment strategy forAA in patients ≥60 years in countries without access to h-ATG."

Clinical • Preclinical • Anemia • Aplastic Anemia • Cardiovascular • Hematological Disorders • Hypertension • Infectious Disease

A novel reduce toxity myeloablative conditioning regimen f-BMT improves efficacy and safety of allogeneic hematopoietic stem cell transplantation in patients with transfusion-dependent thalassemia: A multicenter clinical trial (ASH 2025) - P=N/A | "The F-BMT regimenincluded fludarabine 30 mg/m² (days –7 to –2), busulfan 130 mg/m² (day –7), thiotepa 250 mg/m² (day –5), melphalan 100 mg/m² (day –3). For graft versus host disease (GVHD) prentation, porcine anti-humanlymphocyte immunoglobulin (p-ALG) (25 mg/kg) or antithymocyte globulin (r-ATG) (2.5 mg/kg ) on days –1, cyclophosphamide 25 mg/kg (days +3 and +4)methotrexate 5 mg/m² (day +5), cyclosporine (initiatedon day +5, continued for 12 months). For HID donors, additional agents were administered: p-ALG (25mg/kg ) or r-ATG (2 mg/kg) on day day +5, ruxolitinib (days +5 to +28)...Post-transplant lymphoproliferative disorder(PTLD) was diagnosed in one patient and was cured after Rituximab injection.. Chronic graft-versus-hostdisease (cGVHD) occurred in 8.9% (6 cases) of evaluable patients, involving the pulmonary and hepaticsystems, with all cases rated as moderate; all cases were cured with appropriate therapy. No cases ofthrombotic..."

Clinical • Acute Graft versus Host Disease • Bone Marrow Transplantation • Chronic Graft versus Host Disease • CNS Disorders • Cytomegalovirus Infection • Epstein-Barr Virus Infections • Genetic Disorders • Graft versus Host Disease • Hematological Disorders • Hepatology • Immunology • Infectious Disease • Pneumonia • Respiratory Diseases • Septic Shock • Transplant Rejection • Transplantation

Long-term follow-up of patients with aplastic anemia enrolled in two phase 2/3 trials of immunosuppressive therapy plus romiplostim as a first-line treatment: Final report for up to 5 years (ASH 2025) - P, P2/3 | "Background Results from two phase 2/3 clinical trials evaluating the safety and efficacy of immunosuppressivetherapy (IST) plus romiplostim (ROMI) for IST-naïve patients with aplastic anemia (AA) have beenpreviously reported: the 531-003 trial [rabbit anti-thymocyte globulin (ATG) plus cyclosporin (CsA) plusROMI; NCT03957694] and the 531-004 trial [CsA plus ROMI; NCT04095936]).Although information on the long-term treatment outcomes is essential due to the chronic life-threatening nature of the disease, the long-term follow-up data on effectiveness and safety of ROMItreatment have been lacking after completion of 27 or 53 weeks in the two studies. In addition, a subset ofpatients achieved treatment-free remission with successful discontinuing of both ROMI and CsA. IST plusROMI can be recommended as a first-line treatment option for IST-naïve patients with AA who are noteligible for transplantation"

Clinical • P2/3 data • Acute Myelogenous Leukemia • Anemia • Aplastic Anemia • Hematological Malignancies • Infectious Disease • Leukemia • Myelodysplastic Syndrome • Pneumonia • Respiratory Diseases

Thymoglobuline and grafalon show comparable transplant outcomes in patients with aplastic anemia undergoing allogeneic stem cell transplantation: A multicenter EBMT saawp study of 1603 patients (ASH 2025) - "Introduction:Rabbit anti-thymocyte globulin (ATG) is a key component of conditioning regimens for patients withaplastic anemia (AA) undergoing allogeneic hematopoietic stem cell transplantation (HSCT).Thymoglobuline (Thymo) is widely regarded as the standard formulation due to its extensive clinicaladoption and inclusion in international guidelines, while Grafalon (Grafa) is also commonly used inclinical practice. In this large, registry-based cohort of AA patients undergoing HSCT, no significant differences wereobserved in OS, GF, GVHD incidence, or GRFS between Thymo and Grafa. However, Grafalon dosageappeared to influence outcomes, particularly with respect to chronic GVHD. These findings support theclinical equivalence of both rabbit ATG formulations in the setting of HSCT and suggest that ATG selectioncan be guided by institutional preference or availability without compromising patient outcomes."

Clinical • Acute Graft versus Host Disease • Anemia • Aplastic Anemia • Bone Marrow Transplantation • Chronic Graft versus Host Disease • Graft versus Host Disease • Immunology • Transplantation

Superior disease-free survival of allogeneic hematopoietic stem cell transplantation in elderly patients with hematological malignancies in complete remission by reduced-intensity or myeloablative conditioning (ASH 2025) - "For RIC cohort, the regimen with total bodyirradiation (TBI, 6-7Gy, fractionated), fludarabine (30mg/m2, 5 days) and cytarabine (1g/m2, 3 days) wasused. For MAC cohort, the regimen with TBI (8-10Gy, fractionated, n=15)/total marrow irradiation (10-12Gy, fractionated, n=2) or busulfan (0.8mg/kg q6h, 3 days, n=24), fludarabine (30mg/m2, 5 days) andcytarabine (1g/m2, 3 days) was used. Rabbit antithymocyte globulin was used in unrelated andhaploidentical transplants. Cyclosporine, mycophenolate mofetil and short-term methotrexate wereemployed for graft-versus-host disease prophylaxis. Out of 57 patients, 56 achieved full donor chimerism because one patients with AML (NR in pre-transplant) had active disease at their first disease evaluation post-HSCT... Under our protocol, OS and DFS have been improved remarkably by allo-HSCT in elderlypatients with hematological malignancies, especially, the patients in CR pre-transplant have achieved74.05% long-term DFS. Although RIC..."

Clinical • Acute Lymphocytic Leukemia • Acute Myelogenous Leukemia • Bone Marrow Transplantation • Chronic Myelomonocytic Leukemia • CNS Disorders • Graft versus Host Disease • Hematological Malignancies • Immunology • Infectious Disease • Leukemia • Lymphoma • Myelodysplastic Syndrome • Transplantation

Long-term outcomes of allogeneic stem cell transplantation in β-thalassemia major: A single centre experience from Algeria (ASH 2025) - "The preparative conditioningregimens consist on oral Busulfan 500 mg/m2 or adjusted-Busilvex, Ciclophosphamide 200 mg/kg andThymoglobulin 10 mg/kg (Pesaro class 1-2); Busulfan 14 mg/kg or adjusted-Busilvex, Ciclophosphamide120 mg/kg and Thymoglobulin 10 mg/kg (Pesaro class 3); adjusted-Busilvex, Thiotepa 10mg/kg andFludarabine 160 mg/m2 (Cord blood). GVHD prophylaxis consisted of Ciclosporin combined with shortMethotrexate, or Ciclosporin alone (for cord blood)... Despite a high proportion of pts advanced risk factors (older age, high Pesaro class), allo-HSCT provided sustained long-term survival in this cohort. Early transplantation remains critical toimproving outcomes in TD β-thalassemia major."

Clinical • Acute Graft versus Host Disease • Beta-Thalassemia • Bone Marrow Transplantation • Chronic Graft versus Host Disease • CNS Disorders • Fibrosis • Genetic Disorders • Graft versus Host Disease • Head and Neck Cancer • Hepatology • Immunology • Infectious Disease • Liver Cirrhosis • Oral Cancer • Transplantation • Ventriculomegaly • CD34

Incidence and characteristics of myeloid neoplasms in patients with multiple myeloma treated with novel myeloma-directed therapies (ASH 2025) - "Twenty-three patients had undergone ASCT, 9 of whom received a second ASCT.Maintenance therapy was administered in 20 patients: 17 received lenalidomide (median 17 cycles; range4–115), and 3 received thalidomide (median 13 cycles; range 11–48)...Among AML cases, 6 received intensive induction (FLAI+venetoclax in 4, CPX-351 in2); 2 received azacitidine+venetoclax...GVHD prophylaxis included methotrexate,cyclosporine A, and rATG for matched donors, and post-transplant cyclophosphamide, mycophenolatemofetil, and tacrolimus for haploidentical transplants...Early identification and molecular profiling are essential for diagnosis and risk stratification.Allo-SCT remains the only potentially curative approach. These findings underscore the need for long-term monitoring and individualized management strategies in MM survivors."

Clinical • IO biomarker • Acute Myelogenous Leukemia • Graft versus Host Disease • Hematological Disorders • Hematological Malignancies • Immunology • Leukemia • Multiple Myeloma • Myelodysplastic Syndrome • CSF3R • DNMT3A • KRAS • NRAS • RUNX1 • TP53

Immune tolerance induction after haploidentical stem cell transplant following organ transplantation in sickle cell disease (ASH 2025) - "A non-myeloablative conditioning regimen consisting of rabbit anti-thymocyte globulin, thiotepa, fludarabine,cyclophosphamide, and low-dose total body irradiation (200 cGy) was used...Graft-versus-host disease (GvHD) prophylaxis included PTCy, Sirolimus, and Mycophenolatemofetil...To the best of our knowledge, this is the first reported series of patients with severe SCD who successfullyunderwent sequential solid organ transplantation liver or kidney followed by haplo-HCT, utilizing thesame HLA-related donor for both procedures.Our experience demonstrates the feasibility, safety, and curative potential of dual transplantation usingthe same haploidentical related donor in patients with advanced SCD and related liver or kidneydysfunction. This strategy offers a curative platform durable donor chimerism, organ graft function, andsuccessful immunosuppression withdrawal through the induction of immune tolerance for advance SCDwith severe organ dysfunction. With improved..."

Acute Graft versus Host Disease • Bone Marrow Transplantation • Chronic Graft versus Host Disease • Genetic Disorders • Graft versus Host Disease • Hematological Disorders • Immunology • Liver Failure • Nephrology • Sickle Cell Disease • Solid Organ Transplantation • Transplantation • CD8

Comparison of alemtuzumab and ATG-based conditioning in adult recipients of allogeneic hematopoietic cell transplantation (HCT) for severe aplastic anemia: A retrospective study (ASH 2025) - "The ATG-based regimen consisted of intravenous (IV) fludarabine 30mg/m²/day from day −5 to −2, cyclophosphamide 60 mg/kg/day on days −5 and −4, and rabbit anti-thymocyte globulin (Thymoglobulin) at a total dose of 4.5 mg/kg, administered over days -3 to -1. Three-year event-free survival (EFS) was 64% (40–78) and 70% (53–83), and overall survival (OS) was 91% (68–97) and 85%(68–93) in the alemtuzumab and ATG groups, respectively (p=0.59 and p=0.43).On multivariable analysis, alemtuzumab use was associated with faster platelet engraftment, with nosignificant impact on rates of acute or chronic GvHD, primary graft failure, EFS, or OS.ConclusionAlemtuzumab-based conditioning was associated with faster platelet engraftment compared to ATG, withcomparable rates of graft failure, GvHD, EFS, and OS between the two groups. Our study suggests thateither agent is a reasonable option, with choice guided by availability and institutional experience."

Retrospective data • Acute Graft versus Host Disease • Anemia • Aplastic Anemia • Chronic Graft versus Host Disease • Graft versus Host Disease • Hematological Disorders • Immunology • Transplantation

The impact of HLA loss of heterozygosity on outcome after haploidentical hematopoietic stem cell transplantation in hematological malignancies (ASH 2025) - "Allpatients received haplo-HSCT; pre-transplant disease status was CR (80.0%, n=36) or NR (20.0%, n=9).Conditioning regimens were busulfan (Bu)-cyclophosphamide (Cy) (66.7%), total body Irradiation (TBI)-Cy(17.8%), Bu-fludarabine (flu) (13.3%), or TBI-flu (2.2%). ATG-fresenius (ATG-F) or ATG-thymoglobuline(ATG-T) were used for GVHD prophylaxis... Second transplantation improves survival in patients experiencing relapse following haplo-HSCT who demonstrate HLA loss. Survival outcomes were independent of HLA loss pattern (haplotype vs.locus-specific) or extent (number of loci lost). Poor prognostic factors were pre-transplant NR status andthe use of TBI-based conditioning."

IO biomarker • Acute Lymphocytic Leukemia • Acute Myelogenous Leukemia • Bone Marrow Transplantation • Graft versus Host Disease • Hematological Malignancies • Hepatology • Immunology • Infectious Disease • Leukemia • Liver Failure • Lymphoma • Myelodysplastic Syndrome • Non-Hodgkin’s Lymphoma • Oncology • Transplantation • HLA-B • HLA-C • HLA-DQB1 • HLA-DRB1

Hematopoietic stem cell transplantation using briquilimab (Anti-CD117 Antibody-Conditioning), immunosuppression and TCRαβ+ T-cell/CD19+ B-cell depleted haploidentical grafts in patients with fanconi anemia: An approach without irradiation, busulfan and calcineurin inhibitors. (ASH 2025) - "FA patients have renal abnormalities and are prone to renal toxicities.Therefore, avoiding calcineurin inhibitors increases the renal safety during transplant.Objective: To reduce acute and long-term treatment-related toxicities, we have developed a first of itskind treatment intended to improve the safety of allo-HSCT using: 1) a TBI- and busulfan-freeconditioning regimen consisting of briquilimab, rabbit ATG (rATG - Thymoglobulin), fludarabine,cyclophosphamide and rituximab - briquilimab is a monoclonal antibody (mAb) that targets humanCD117 to clear host HSCs from their niches enabling blood and immune reconstitution with minimaltoxicity with other agents being used for immune suppression to prevent immunologic rejection; 2)transplantation of TCRαβ+ T-cell/CD19+ B-cell hematopoietic grafts - a stem cell therapy that enhancesdonor hematopoietic and immune reconstitution while decreasing GvHD; and 3) pharmacokineticanalysis for briquilimab, rATG and fludarabine to..."

Clinical • Acute Graft versus Host Disease • Anemia • Aplastic Anemia • Bone Marrow Transplantation • Chronic Graft versus Host Disease • Genetic Disorders • Graft versus Host Disease • Immunology • Transplant Rejection • Transplantation • CD34 • KIT • NCAM1

Enhanced engraftment and immune recovery in thalassemia via optimized conditioning for TCRαβ⁺-depleted haploidentical transplants (ASH 2025) - "Both groups shared the following conditioning regimen: Days -21 to -19: rATG (5mg/kg); Day -11: donor lymphocyte infusionDLI (2×10⁸/kg); Days -8 and -7: cyclophosphamide (50mg/kg/day); Days -6 to -4: busulfan (90-100 mg/m²/day; target CSS: 500-700; Days -6 to -2: fludarabine (40mg/m²/day); Day -3: thiotepa (10 mg/kg); and Day -1: rituximab (100 mg/m²) for all patients. GVHDprophylaxis comprised tacrolimus/sirolimus (target level: 5-10 ng/mL), with mycophenolate mofetil addedfor high-risk patients.Result1.The graft composited of 23.86×10⁶/kg (range 14.30-61.12) CD34 cells, with 11.90×10⁸/kg (6.15-21.5)mononuclear cell...CD34⁺ >30×10⁶/kg: Higher cGVHD risk (OR=2.8; 95% CI 1.2–6.5; P=0.040).4.IR and infections: Infected patients showed markedly lower γδ-T, NK, and CD4⁺T cells vs. non-infected(12-month γδ-T: 275/μL vs. 369/μL; P<0.01). Rapid γδ-T reconstitution (236/μL at 3 months) was critical forcontrolling severe..."

Acute Graft versus Host Disease • Beta-Thalassemia • Bone Marrow Transplantation • Chronic Graft versus Host Disease • Genetic Disorders • Graft versus Host Disease • Immunology • Infectious Disease • Pneumonia • Respiratory Diseases • Transplantation