busulfan / Generic mfg. - LARVOL DELTA

Treatment patterns and outcomes of primary central nervous system lymphoma treated with high-dose methotrexate with or without autologous stem cell transplantation or whole brain radiation in the rea-world setting (ASH 2025) - "During induction, 59% received HDMTX+rituximab (R), 39% received HDMTX+R+additional (A) chemotherapy such as temozolomide (MTR) (15%) and cytarabine/thiotepa (MATRix) (15%)...Thiotepa (TT)/BCNU was used for conditioning in 8 patients, and TT/Busulfan/Cyclophosphamide in 1... HDMTX-based induction chemotherapy is effective in patients with PCNSL, even in those with delayed diagnosis or initiation of therapy. Although a minority of patients received consolidation with ASCT, it was associated with 100% progression-free survival."

B Cell Lymphoma • CNS Lymphoma • Diffuse Large B Cell Lymphoma • Hematological Malignancies • Lymphoma • Non-Hodgkin’s Lymphoma • Primary Central Nervous System Lymphoma • Pulmonary Disease • Respiratory Diseases • Transplantation

Clinical characteristics and transplant outcomes in pediatric patients with transfusion-dependent alpha-thalassemia undergoing HSCT (ASH 2025) - "All patients received a standardized myeloablative conditioning regimen (GX-07-TM: busulfan, cyclophosphamide, fludarabine, ATG). This study characterizes the clinical features and transplant outcomes of TDT-α pediatric patients undergoing HSCT. The presence of early severe manifestations —such as non-deletional genotypes, transfusion dependency, growth restriction, and extramedullary hematopoiesis— may support early HSCT consideration. HSCT provides excellent long-term outcomes and should be considered a frontline curative option in this population."

Clinical • Bone Marrow Transplantation • Genetic Disorders • Graft versus Host Disease • Hematological Disorders • Immunology • Pediatrics • Transplantation

Outcomes of haploidentical hematopoietic stem cell transplantation in low- and middle-income countries: A systematic review and meta-analysis. (ASH 2025) - "Myeloablative conditioning (MAC) was used in 42% while reduced intensity/non-myeloablative (RIC/NMA) in 58% of, with RIC/NMA favoring Fludarabine-based regimens and MAC using full-dose Busulfan or high-dose TBI...The use of post-transplant cyclophosphamide (PTCy) was not associated with differences in OS at either time point compared to non-PTCy regimens (p = 0.4694)... Haploidentical hematopoietic cell transplantation is widely utilized in low- and middle-income countries, primarily for the treatment of acute leukemia, using peripheral blood stem cell grafts and a preference for reduced-intensity or non-myeloablative conditioning regimens over myeloablative ones. Survival outcomes remain modest and heterogeneous, emphasizing the need for standardized protocols and further research to optimize results in resource-constrained settings."

Retrospective data • Review • Acute Graft versus Host Disease • Aplastic Anemia • Bone Marrow Transplantation • Graft versus Host Disease • Hematological Malignancies • Immunology • Leukemia • Lymphoma • Metabolic Disorders • Myelodysplastic Syndrome • Transplantation

Analysis of growth, development and endocrine function in children with primary immunodeficiency disease after hematopoietic stem cell transplantation (ASH 2025) - "Preconditioning plan before transplantation and patient age at the time of transplantation are crucial. Busulfan, high-dose cyclophosphamide and radiotherapy significantly affect the occurrence of spontaneous puberty and spontaneous menstruation. Keywords :growth and development; endocrine function; hematopoietic stem cell transplantation; primary immunodeficiency disease;"

Clinical • Bone Marrow Transplantation • Immunology • Primary Immunodeficiency • 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

Late recurrence of transfusion dependent thalassemia 30 years after transplantation (ASH 2025) - "Case#1: A 16-month-old male was transplanted in 1982 (Lancet, 1982), conditioned with dimethyl busulfan and cyclophosphamide. These observations raise the possibility of long-term exhaustion of allogeneic engraftment and, simultaneously, support the concept of the persistence or "immortality" of autologous thalassemic hematopoietic stem cells, which may remain quiescent for decades following myeloablative conditioning. Systemic observation in the unique cohort of thalassemia patients is warranted"

Chronic Graft versus Host Disease • Genetic Disorders • Graft versus Host Disease • Hematological Disorders • Immunology • Sickle Cell Disease • Transplantation

Prognostic impact of mixed chimerism with undetectable measurable residual disease in pediatric and adolescent young adult patients undergoing allogeneic hematopoietic stem cell transplantation for hematological malignancies (ASH 2025) - "Conditioning regimens included TBI-based (n=4), busulfan-based (n=2), and treosulfan-based (n=2). Lineage-specific chimerism analysis offers superior prognostic value over global assessments. Standardized monitoring protocols are essential to guide post-HSCT management and avoid unnecessary and harmful interventions."

Clinical • Residual disease • Acute Graft versus Host Disease • Bone Marrow Transplantation • Chronic Graft versus Host Disease • Graft versus Host Disease • Hematological Malignancies • Immunology • Infectious Disease • Lymphoma • Oncology • Pediatrics • Pneumonia • Respiratory Diseases • Transplantation

Results of abatacept addition to post transplant cyclophosphamide-based GVHD prophylaxys in allogeneic hematopoyetic stem cell transplantation (ASH 2025) - "All patients received peripheral blood as graft source and all patients received GVHD prophylaxis with ABA in combination with PTCY, tacrolimus, and mycophenolate mofetil...Reduced-intensity conditioning regimens were used in the majority of patients in both cohorts (88.2% vs 93.4%, p=0.5), most commonly conditioning included busulfan-fludarabine or busulfan-cyclophosphamide-fludarabine. In the +56 cohort, 2 patients received clofarabine-melphalan, according to internal protocol, due to disease persistence prior to transplant.With a median follow-up of 15.7 months (range 7.4–31.1), estimated 1-year PFS and OS were 66% (IC95%= 0.5-0.7) and 72% (IC95%= 0.5-0.8)... Our experience suggests, compared with previous analysis ( P Fernandez-Caldas. EBMT 2024 ), that addition of ABA to PTCY-based prophylaxis, in peripheral blood stem cell setting, may reduce GVHD and allow early immunosuppression withdrawal without increasing GVHD rates, though sample size is limited."

Post-transplantation • Acute Graft versus Host Disease • Acute Myelogenous Leukemia • Bone Marrow Transplantation • Chronic Graft versus Host Disease • Graft versus Host Disease • Hematological Malignancies • Immunology • Leukemia • Myelodysplastic Syndrome • Transplantation • CTLA4

FluMel100 with post-transplant cyclophosphamide: A safe and effective regimen for patients aged ≥75 years with MDS and AML (ASH 2025) - "Many published studies in older adults have often utilized non-myeloablative and reduced intensity conditioning regimens, including fludarabine/total body irradiation, fludarabine/melphalan (FluMel) and fludarabine/busulfan combined with varied graft versus host disease (GVHD) prophylaxis approaches...They underwent re-induction therapy with cladribine, low dose cytarabine and venetoclax, achieving a complete remission, followed by donor leukocyte infusion (DLI)... Eight patients were included: seven with AML (n=7) and one had MDS (n=1), Kanofsky Performance Status range 80-90%, HCT-CI (Comorbidity Index) range 0-7. All patients received peripheral blood stem cell (PBSC) grafts from human leukocyte antigen (HLA) matched unrelated donors (MUD). PT-Cy dose was 80 mg/kg (n= 7) and 50mg/kg (n= 1)."

Clinical • Post-transplantation • Acute Graft versus Host Disease • Acute Myelogenous Leukemia • Bone Marrow Transplantation • Chronic Graft versus Host Disease • Geriatric Disorders • Graft versus Host Disease • Hematological Malignancies • Immunology • Leukemia • Myelodysplastic Syndrome • Transplantation • CD33 • HLA-DPB1

Comparable survival outcomes after myeloablative allogeneic stem cell transplantation in secondary and de novo Acute Myeloid Leukemia in first complete remission: A propensity Score–Matched study (ASH 2025) - "Eligible patients were adults (≥18 years) diagnosed with de novo or secondary AML who received a first allogeneic hematopoietic stem cell transplantation (HSCT) with a myeloablative conditioning (MAC) regimen between 2013 and 2024, no ex vivo T-cell depletion, and no posttransplant cyclophosphamide (PTCy) as graft-versus-host disease (GVHD) prophylaxis...All patients received myeloablative conditioning (MAC), defined as regimens containing either total body irradiation (TBI) >6 Gy or a cumulative busulfan dose >8 mg/kg orally or >6.4 mg/kg intravenously...The two-year LFS, OS, and GRFS were 65.8% (95%CI,56.5-76.6) versus 54.4% (95%CI,39.6,74.7)( p =0.84), 67.9% (95%CI,58.7-78.5) versus 66.6% (95%CI,52.1-85.1)( p =0.84), and 56.6%(95%CI,47.1-68.2) versus 36.0%(95%CI,22.6-57.5) ( p =0.35), respectively (Table 3A). Similarly, no outcome differences were found when comparing sAML post-MDS or sAML post other malignant hematological disorders (OMHD)/solid tumors..."

Clinical • Acute Graft versus Host Disease • Acute Myelogenous Leukemia • Aplastic Anemia • Bone Marrow Transplantation • Chronic Graft versus Host Disease • Graft versus Host Disease • Hematological Malignancies • Immunology • Leukemia • Myelodysplastic Syndrome • Myeloproliferative Neoplasm • Solid Tumor • Transplantation

Total marrow irradiation-based, intensified reduced-intensity conditioning regimen for haploidentical hematopoietic cell transplantation with posttransplant cyclophosphamide: A prospective study (ASH 2025) - P1 | "Patients received Haplo-HCT with a conditioning regimen consisting of intravenous busulfan (total area under the curve, AUC, of 9,600 µM*min), fludarabine (total dose of 150 mg/m²), and total marrow irradiation (6 Gy, administered in two days with two daily doses of 1.5 Gy). In one patient, melphalan 50 mg/m2 was substituted for busulfan...These results show a good balance between the effectiveness of the conditioning regimen and toxicity. Future studies should confirm these promising results in haplo-HCT with TMI-based RIC conditioning using a larger patient cohort.Acknowledgements The authors declare no conflict of interest."

Clinical • Post-transplantation • Acute Graft versus Host Disease • Acute Myelogenous Leukemia • Bone Marrow Transplantation • Chronic Graft versus Host Disease • Chronic Myelomonocytic Leukemia • Graft versus Host Disease • Hematological Malignancies • Immunology • Leukemia • Lymphoma • Myelodysplastic Syndrome • Non-Hodgkin’s Lymphoma • 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

Allogeneic hematopoietic cell transplantation for infantile osteopetrosis: High rates of sinusoidal obstruction syndrome/veno-occlusive disease and transplant-related morbidity (ASH 2025) - "Busulfan-based conditioning is widely used in myeloablative regimens in osteopetrosis alloHCT, increasing SOS/VOD risk in an already at-risk population...4/24 (17%) patients received prophylactic defibrotide...In this population, we demonstrated a high retrospective incidence of criteria fulfillment for diagnosis of VOD/SOS at 71% and 42% for Modified Seattle and Baltimore diagnostic criteria respectively, as well as a low 3-year overall survival of 36%. Our findings reinforce the need to optimize conditioning regimens as well as the approach to complications in the post-transplant setting."

Acute Graft versus Host Disease • Aplastic Anemia • Bone Marrow Transplantation • Chronic Graft versus Host Disease • Genetic Disorders • Graft versus Host Disease • Hematological Disorders • Hepatology • Immunology • Infectious Disease • Transplantation

Haploidentical hematopoietic stem cell transplantation with bussulfan, fludarabine and cyclophosphamide and total body irradiation 200cgy conditioning with post-transplant cyclophosphamide and peripheral blood stem cells as an alternative regimen to reduce graft rejection in sickle cell disease (ASH 2025) - "Graft-versus-host disease (GvHD) prophylaxis included PTCy (50 mg/kg on days +3 and +4), mycophenolate mofetil, and sirolimus. Our data support the feasibility and safety of haploidentical HSCT using busulfan, fludarabine, and cyclophosphamide RIC combined with PTCy and PBSC in adult SCD patients. This approach yielded sustained full donor chimerism with low incidence of severe GvHD and graft failure, offering a practical alternative in resource-limited settings where thiotepa is not easily available. Considering the ongoing challenges in donor availability and conditioning toxicity, further studies with larger cohorts and longer follow-up are warranted to confirm the durability of engraftment, late effects, and overall survival benefits."

Post-transplantation • Acute Graft versus Host Disease • Bone Marrow Transplantation • Cytomegalovirus Infection • Genetic Disorders • Graft versus Host Disease • Hematological Disorders • Hepatology • Immunology • Infectious Disease • Inflammation • Mucositis • Sickle Cell Disease • Transplant Rejection • Transplantation

Outcomes of myeloablative vs reduced and non-myeloablative conditioning in MRD-negative AML and ALL with ptcy-based GVHD prophylaxis (ASH 2025) - "In the current post-transplant cyclophosphamide (PTCY) era, outcomes may differ given the widespread use of PTCY-based graft-versus-host disease (GVHD) prophylaxis...Reduced intensity conditioning included fludarabine 30mg/m 2 for 4 consecutive days plus 8 Gy TBI, or fludarabine plus either melphalan or busulfan, with 2 Gy TBI...All patients received PTCY in combination with a calcineurin inhibitor and mycophenolate mofetil as GVHD prophylaxis... In MRD-negative AML and ALL patients undergoing allo-HSCT with PTCY-based GVHD prophylaxis, NMA regimens appear to offer comparable survival and GVHD outcomes to MAC, with potentially lower toxicity. These findings suggest NMA conditioning may be a viable and safer alternative to MAC in select MRD-negative patients in the PTCY era."

Minimal residual disease • 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

First report of successful, lifesaving and timely graft rescue using an off-the-shelf cryopreserved, cadaveric marrow in myeloablative transplant with an unexpected unavailability of a living matched unrelated donor. (ASH 2025) - " A 34-year-old male with AML (NPM1, FLT3 TKD, KRAS, PTPN11, WT1, and RAD21 mutations) in first complete remission (CR1) began myeloablative fludarabine/busulfan conditioning in preparation for a 10/10 HLA-matched unrelated donor peripheral blood stem cell transplant...GVHD prophylaxis consisted of tacrolimus, mycophenolate mofetil, and post-transplant cyclophosphamide (PTCy, 50 mg/kg/d on Days +3 and +4). Patient received filgrastim post-transplant... This case report illustrates the feasibility and life-saving potential of using cryopreserved bone marrow from deceased donors in urgent allo-HCT scenarios. Day 0 was delayed by only 48 hours and the product was identified within 24 hours of request. Timely access to an off-the-shelf marrow product prevented catastrophic graft failure or debilitating opportunistic infections."

Acute Myelogenous Leukemia • Graft versus Host Disease • Hematological Malignancies • Immunology • Infectious Disease • Leukemia • Transplantation • CD33 • CD34 • FLT3 • KRAS • NPM1 • PTPN11 • RAD21 • WT1

Hematopoietic stem cell transplantation in T-cell lymphomas: A promising strategy challenging poor prognosis (ASH 2025) - "Four (10%) patients received brentuximab...Myeloablative conditioning was used in 92.5%, primarily PEAM in autologous, and fludarabine/busulfan-based regimens in allogenic settings... HSCT proved to be a viable therapeutic option, particularly in advanced-stage disease, challenging the historically poor outcomes associated with TCL. HSCT was successfully performed as consolidation therapy and in R/R cases, resulting in improved outcomes. The increased risk of progression in R/R group reinforces the value of HSCT as consolidation in CR1."

Bone Marrow Transplantation • Graft versus Host Disease • Hematological Malignancies • Immunology • Infectious Disease • Lymphoblastic Lymphoma • Lymphoma • Natural Killer/T-cell Lymphoma • Non-Hodgkin’s Lymphoma • Peripheral T-cell Lymphoma • T Cell Non-Hodgkin Lymphoma • Transplantation • ALK • TNFRSF8

efficacy and safety of treosulfan- vs. busulfan-based conditioning regimens in adult allogeneic hematopoietic stem cell transplantation for myelodysplastic syndromes and acute myeloid leukemia: A systematic review and meta-analysis. (ASH 2025) - "Treosulfan-based conditioning regimens offer favorable efficacy and safety compared to busulfan, with reduced NRM and improved donor chimerism without compromising engraftment or increasing GVHD or relapse risk. These findings support Treosulfan as a clinically effective and better-tolerated alternative, particularly for older or medically vulnerable transplant recipients."

Retrospective data • Review • Acute Graft versus Host Disease • Acute Myelogenous Leukemia • Bone Marrow Transplantation • Chronic Graft versus Host Disease • Graft versus Host Disease • Hematological Malignancies • Hepatology • Immunology • Infectious Disease • Leukemia • Myelodysplastic Syndrome • Transplantation

A rare case of high-risk M4 Acute Myeloid Leukemia with ETV6::ABL1 rearrangement in a young adult with preceding chronic idiopathic thrombocytopenia: Insights and implications (ASH 2025) - "Treatment Course: The patient was treated per the AAML1831 protocol for high-risk disease, with the addition of Dasatinib due to potential Imatinib resistance in ETV6::ABL1-positive disease...The patient received a 10/10 HLA-matched unrelated donor allogeneic HSCT in May of 2024 with a Busulfan and Cyclophosphamide conditioning regimen... This report highlights an extremely rare case of AML with ETV6::ABL1 rearrangement in a young adult with prior idiopathic thrombocytopenia, successfully treated with a combination of AAML1831-based chemotherapy, Dasatinib, and allogeneic HSCT. Her disease course emphasizes the vital importance of advanced cytogenetic and molecular diagnostics in cytopenic patients, the tailored use of targeted therapies, and HSCT for rare high-risk rearrangements. Broader study of ETV6::ABL1 driven AML in children and adults is needed to establish the optimal management strategies and to better understand the significance of preceding cytopenias in..."

Clinical • Acute Graft versus Host Disease • Acute Myelogenous Leukemia • Bone Marrow Transplantation • Chronic Graft versus Host Disease • Graft versus Host Disease • Hematological Malignancies • Hepatology • Immunology • Leukemia • Myelodysplastic Syndrome • Orthopedics • Thrombocytopenia • Thrombocytopenic Purpura • ABL1 • ETV6 • PHF6

Allogeneic hematopoietic stem cell transplantation in treatment of Shwachman- diamond syndrome: A Report of 25 Pediatric cases from a Single center (ASH 2025) - "All patients used myeloablating conditioning.The regimen of the case with AML consisted of busulfan, fludarabine, cytarabine, cyclophosphamide and anti-thymocyte globulin(ATG)...All were given cyclosporine and mycophenolate mofetil to prevent graft-versus- host disease(GVHD). Balliximab was given at +1 day to the patients whose donor was MMRD and methotrexate was given at +1,+4,+7,+11 days to the patients whose donor was MMUD or UCB...Melphalan was administered before donor peripheral blood stem cell infusion, resulting in negative MRD... Allo-HSCT is an effective treatment for SDS complicated with severe hematological abnormalities.Compared with MMRD and MMUD HSCT, the incidence of GVHD in CBT is lower. MDS complicated with both complex karyotype and Tp53 mutation was associated with an extremely poor prognosis."

Clinical • Acute Graft versus Host Disease • Acute Myelogenous Leukemia • Bone Marrow Transplantation • Chronic Graft versus Host Disease • Genetic Disorders • Graft versus Host Disease • Hematological Disorders • Hematological Malignancies • Immunology • Leukemia • Myelodysplastic Syndrome • Neutropenia • Palliative care • Pediatrics • Thrombocytopenia • Transplantation • TP53

iPSC-derived mesenchymal stromal cells (iMSC) provide superior support for hematopoietic reconstitution compared to BM-MSCs (ASH 2025) - "Notably, iMSCs were resistant to conditioning agents includingcyclophosphamide, fludarabine, and busulfan, as well as to high-dose irradiation—mirroring thechemoresistance of BM-MSCs and enabling their use in post-chemotherapy support. iMSC-treated animals displayed significantlyaccelerated white blood cell, platelet count and hemoglobin recovery following sublethal irradiationcompared to BM-MSC-treated or control groups. Conclusion, Data suggest the use of iMSCs as a scalable, genetically stable, and homogenous alternativeto BM-MSCs, with superior expansion capacity for hematopoietic engraftment after myeloablationtherapy."

Stroma • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Sarcoma • Solid Tumor • CD34 • CXCL12 • MMP1 • THY1 • TNFSF12

Transplantation in combination with CAR T-cell therapy for refractory/relapsed aggressive B-cell lymphoma after failure of CD19 CAR-T cell therapy (ASH 2025) - "Twelve patients (50%) were selected for auto-HSCT+CAR-Tand the other 12 (50%) for allo-HSCT+donor CAR-T, at a median of 8 months (range: 2-48) post-CD19 CAR-T infusion.Prior to the study, CD20/CD22 antigen expression in tumor tissue was confirmed by pathology,with the target selected based on antigen expression.The conditioning regimen for the auto-HSCT+CAR-T group was BEAM, while that for the allo-HSCT+donorCAR-T group consisted of a busulfan-fludarabine-based regimen. Transplantation combined with CAR-T therapy presents an effective option for R/R B-celllymphoma following the failure of CD19 CAR-T. Effective BT prior to transplantation significantlyenhances survival rates."

CAR T-Cell Therapy • Combination therapy • IO biomarker • Acute Graft versus Host Disease • B Cell Lymphoma • Bone Marrow Transplantation • Diffuse Large B Cell Lymphoma • Graft versus Host Disease • Hematological Malignancies • Infectious Disease • Lymphoma • Non-Hodgkin’s Lymphoma • Transplantation • CD20 • CD22

Trial in progress: Safety and feasibility of a venetoclax-augmented treosulfan-based reduced intensity conditioning before allogeneic stem cell transplantation in AML, MDS/AML and higher risk MDS (VeStAL) (ASH 2025) - "While Fludarabine plus Busulfan(FluBu) is a very popular RIC regimen, data from a randomized controlled trial have demonstratedsuperior overall survival (OS) with Treosulfan (FluTreo) instead of Busulfan as alkylating agent (3 year OS67 vs. 56%; Beelen et al., Am J Hematol, 2022). A scientific companion programincludes spatial assessment of the bone marrow microenvironment under therapy and monitoring ofchimerism via single nucleotide polymorphism using digital PCR. The study was submitted via CTIS on30.07.2025 and will start enrollment early in 2026."

Clinical • IO biomarker • Acute Myelogenous Leukemia • Bone Marrow Transplantation • Graft versus Host Disease • Hematological Malignancies • Immunology • Leukemia • Myelodysplastic Syndrome • Sarcoma • Solid Tumor • Transplantation

Treosulfan-based vs. busulfan-based conditioning for hematopoietic stem cell transplantation: A systematic review and meta-analysis. (ASH 2025) - "No substantial or significance differenceswere observed between regimens for toxicity (RR = 1.11, p = 0.72), TRM (RR = 0.84, p = 0.69), NRM (RR =0.93, p = 0.84), or relapse incidence (RR = 1.47, p = 0.11).ConclusionAlthough most outcomes between Treosulfan- and Busulfan-based regimens were statisticallycomparable, Treosulfan demonstrated non-inferiority in toxicity, TRM, NRM, and relapse incidence, alongwith consistently favorable results in OS, EFS, GVHD, and engraftment. These findings, reinforced bysensitivity analysis, emphasize the robustness of the results and highlight Treosulfan's potential as areliable conditioning agent for broader clinical application."

Retrospective data • Review • Bone Marrow Transplantation • Graft versus Host Disease • Hematological Malignancies • Immunology • Transplantation

Haploidentical hematopoietic stem cell transplantation using low-dose alemtuzumab is effective for standard-risk hematological malignancies (ASH 2025) - "[Background ] We have been performing allogeneic hematopoietic stem cell transplantation fromhaploidentical related donor using anti-CD52 antibody, alemtuzumab, at a total dose of 1.2 mg/kg or 0.96mg/kg (Am J Hematol 2013; 88:294-300) and 0.5 mg/kg (Eur J Haematol 2019; 102: 256-264) in pre-transplant regimens, combined with cyclosporine (CSA) at the target concentration of 500 ng/ml withrapid tapering and methotrexate (MTX) at a dose of 10-7-7-7 mg/m2 as graft-versus-host disease (GVHD)prophylaxis...Total body irradiation(TBI) of 12 Gy and cyclophosphamide (CY) or fludarabine (FLU), intravenous busulfan (ivBU) andmelphalan (MEL) were mainly used as myeloablative regimen, and FLU, MEL, and TBI of 4 Gy was themainly used reduced-intensity regimen...On the other hand, outcomes of patients with standard-riskdisease are promising, and combination with low-dose MTX may improve OS. Further studies arewarranted to establish the optimal role of haploidentical HSCT using low-dose..."

Acute Graft versus Host Disease • Bone Marrow Transplantation • Chronic Graft versus Host Disease • Chronic Myeloid Leukemia • Graft versus Host Disease • Hematological Malignancies • Immunology • Leukemia • Myelodysplastic Syndrome • Myeloproliferative Neoplasm • Oncology • Transplantation