busulfan / Generic mfg. - LARVOL DELTA

In silico and in vitro investigations of the drug-drug interaction mechanisms between fludarabine and busulfan. (PubMed, Front Pharmacol) - "The present study underscores the importance of empirically validating in silico observations in pharmacological research by emphasizing the minimal effect of Flu on GST activity and its implications in clinical oncology. Moreover, the findings suggest that Flu is not likely to alter BU pharmacokinetics via the GSH-conjugation pathway."

Journal • Preclinical • Bone Marrow Transplantation • Oncology • Transplantation • GSTM1 • GSTP1

Superior outcomes with the FABT regimen in haploidentical transplantation for aplastic anemia in patients under 40 years old: a single-center retrospective study. (PubMed, Clin Exp Med) - "The FABT regimen featured reduced-dose busulfan, fludarabine, cyclophosphamide, thiotepa, and low-dose anti-thymocyte globulin (ATG), followed by post-transplant cyclophosphamide (PTCy), calcineurin inhibitors, and mycophenolate mofetil for Graft-versus-host disease (GVHD) prophylaxis. The FABT conditioning regimen demonstrates superior outcomes compared to the FCA regimen in young AA patients underwent haplo-HSCT, characterized by significantly improved GRFS and CR rates, and reduced CMV reactivation, without compromising engraftment or increasing GVHD. This regimen represents a promising therapeutic strategy for this patient population."

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

US Study of ECT-001-CB in Pediatric and Young Adult Patients With High-Risk Myeloid Malignancies (clinicaltrials.gov) - P1/2 | N=13 | Completed | Sponsor: ExCellThera inc. | Recruiting ➔ Completed | Trial completion date: Jun 2027 ➔ Dec 2025 | Trial primary completion date: Jun 2026 ➔ Dec 2025

Trial completion • Trial completion date • Trial primary completion date • Hematological Malignancies • Juvenile Myelomonocytic Leukemia • Myelodysplastic Syndrome • Oncology • Pediatrics • Transplantation • CD34 • HLA-DRB1

High-dose busulfan-melphalan vs melphalan and reinforced VRD for newly diagnosed multiple myeloma: a phase 3 GEM trial. (PubMed, Blood) - P3 | "We compared BUMEL vs. MEL200 outcomes in newly diagnosed multiple myeloma (NDMM) patients receiving intensified bortezomib, lenalidomide and dexamethasone (VRD) induction and consolidation therapy. After a median follow-up of 8.4 years, GEM2012 reported one of the longest PFS durations in NDMM patients, with BUMEL significantly favoring advanced ISS stages. The trial is registered at ClinicalTrials.gov (NCT01916252) and EudraCT (2012-005683-10)."

Journal • P3 data • Hematological Malignancies • Multiple Myeloma • Oncology • Transplantation

Matched unrelated vs. Haploidentical donor for allogeneic stem cell transplantation in patients with acute leukemia with identical GvHD prophylaxis – a randomized prospective european trial. (ASH 2025) - P2 | "Haplo-identical allogeneic cell transplantation (haploHCT) using post- transplant cyclophosphamide(PTCy) is increasingly used if no matched related or unrelated donor (MUD) is available...Disease and patient characteristics were well balanced in both armsregarding disease, AML CR1 (57%) or CR2 (10%) ALL CR1 (21%) and CR2 4% and HR- MDS in CR (6%),median donor age ( 29 vs 31 years) and conditioning intensity: Busulfan (for AML) and TBI (for ALL)based RIC (62%) or MAC (38%)...According to the results, the Data Safety Monitoring Board recommend stopping recruitment because itis unlikely to show a 10% superiority of HaploHCT to MUD regarding relapse incidence with the plannedsample size. The planned interims analysis of our prospective randomized multicenter study comparing10/10 MUD with haploHCT using identical PTCy based GvHD prophylaxis showed comparable toxicity, lowGvHD incidence, low NRM and favourable outcome in both arms suggesting that further recruitment..."

Clinical • Acute Graft versus Host Disease • Bone Marrow Transplantation • Graft versus Host Disease • Hematological Malignancies • Immunology • Leukemia • Myelodysplastic Syndrome • Transplantation

Restoration of Spermatogenesis is Dependent on Activation of a SPRY4-ERK Checkpoint Following Germline Stem Cell Damage. (PubMed, Biol Reprod) - "Here, we demonstrate that following alkylating agent busulfan (BU)-induced injury in adult mice, germline-specific Spry4 gene deletion (Spry4G-KO) reduces stem cell regeneration with an enhanced genotoxic stress response and differentiation with rapidly enhanced nuclear ERK1/2 activity in undifferentiated (Aundiff) spermatogonia (including SSCs)...Furthermore, the MEK1/2 inhibitor PD0325901, but not mTORC1 inhibitor rapamycin, was sufficient to promote spermatogonial proliferation in Spry4G-KO testis 10 days post-BU treatment...In summary, germline-specific deletion of Spry4 results in hyper-activation of the MAPK/ERK pathway in Aundiff spermatogonia, reducing spermatogonial genome integrity, unleashing excessive spermatogenesis after germline damage, and ultimately impairing germline regeneration in adult males. Our study indicates an essential role for SPRY4-ERK signaling as a molecular checkpoint in securing SSC recovery upon chemotherapy drug-induced germline..."

Journal • Infertility • Sexual Disorders • CXCL12

SAFETY AND EFFICACY OF LIPOSOMAL MITOXANTRONE-INTENSIFIED BUSULFAN-BASED CONDITIONING REGIMEN FOR ALLOGENEIC HEMATOPOIETIC STEM CELL TRANSPLANTATION IN PATIENTS WITH HEMATOLOGICAL MALIGNANCIES (EBMT 2026) - "We conducted this study to evaluate the preliminary efficacy and safety of Lipo-MIT intensified busulfan (Bu)-based conditioning regimens (combined with cyclophosphamide or fludarabine) for allo-HSCT in patients with hematological malignancies. It is concluded that Lipo-MIT-enhanced Bu-based conditioning regimens were feasible and effective for allo-HSCT in AML, ALL, or MDS patients. However, the sample size needs to be expanded for further observation and evaluation of long-term survival and recurrence rates."

Clinical • 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 • Infectious Disease • Leukemia • Myelodysplastic Syndrome • Oncology • T Acute Lymphoblastic Leukemia • Transplantation

Results of the primary end-point of the LOC-R01: A randomized phase II Study of lenalidomide and ibrutinib in association with rituximab-methotrexate procarbazine vincristin (R-MPV) as a targeted induction treatment for patients aged 18 to 65 with a newly diagnosed primary central nervous system lymphoma (PCNSL) (ASH 2025) - P1/2 | "Background : With standard high-dose methotrexate (HDMTX) and cytarabine (HDAraC)-basedinductions, half of the patients achieved complete response (CR)...Responders received two cycles of HDAraCfollowed by HD thiotepa-busulfan and ASCT... Both arms met the predetermined threshold of efficacy with 86% and 82% of CR/CRurates in the lenalidomide and ibrutinib arm, respectively. R-MPV plus a BTK-inhibitor or animmunomodulatory drug constitutes an interesting first-line induction for PCNSL patients up to 65 years.Correlation of patient, disease and lymphoid subpopulations characteristics with response in eachtreatment arm will be explored to guide the choice of the targeted therapy. Ancillary studies regardingthe prognostic impact of baseline and end of induction levels of cytokines in the CSF, ctDNA inplasma/CSF and radiomic features, as biomarkers of response, are planned."

Clinical • IO biomarker • P2 data • CNS Disorders • CNS Lymphoma • Hematological Malignancies • Hepatology • Lymphoma • Mental Retardation • Non-Hodgkin’s Lymphoma • Primary Central Nervous System Lymphoma • Steven-Johnson Syndrome • CD4 • ICOS • PD-1

Myeloablative fractionated busulfan, fludarabine, cladribine, thiotepa, and venetoclax (Cladillac) conditioning for high-risk AML: A phase 2 trial (ASH 2025) - P2/3 | "To reduce GVHD-associated morbidity and mortality, weincorporated post-transplant cyclophosphamide (PTCy)...GVHD prophylaxis consisted of PTCy 50mg/kg on days +3 and +4, tacrolimus ± mycophenolate mofetil... This study met its primary endpoint, demonstrating a promising 3-year PFS of 58% in acohort of patients with very high-risk AML. Outcomes were particularly favorable in TP53 wild-typepatients, with a low relapse rate of 13% and 3-year OS of 74%. These findings support furtherinvestigation of this novel conditioning regimen."

P2 data • Acute Graft versus Host Disease • Acute Myelogenous Leukemia • Chronic Graft versus Host Disease • Graft versus Host Disease • Hematological Malignancies • Immunology • Infectious Disease • Leukemia • FLT3 • TP53

BUSULFAN ASSOCIATED PULMONARY TOXICITY AND INFECTIOUS COMPLICATIONS FOLLOWING AUTOLOGOUS SCT IN A 3-YEAR-OLD CHILD WITH NEUROBLASTOMA (EBMT 2026) - "Background: High-dose busulfan/melphalan (Bu/Mel) remains a standard conditioning regimen for autologous stem cell transplantation in high-risk neuroblastoma but is associated with significant organ toxicity, particularly hepatic and pulmonary injury...A treatment with Defibrotide, Ganciclovir and Azithromycin was initiated...We started a treatment with Sildenafil, Macitentan, Epoprostenol in addition to Methylprednisolone pulse therapy and antifibrotic treatment with Nintedanib, Hydroxychloroquin and Azithromycin... This case illustrates the complex interplay between infectious complications, post-transplant inflammation and busulfan-induced pulmonary toxicity in a young neuroblastoma patient undergoing Bu/Mel conditioning and autologous SCT. Consideration of busulfan toxicities, especially when presenting with nonspecific early symptoms is important even when alternative explanations such as severe infection may appear equally plausible after autologous SCT. Early..."

Clinical • Acute Respiratory Distress Syndrome • Cardiovascular • Cystic Fibrosis • Cytomegalovirus Infection • Gastroenterology • Genetic Disorders • Hepatology • Immunology • Infectious Disease • Inflammation • Interstitial Lung Disease • Neuroblastoma • Pneumonia • Pulmonary Arterial Hypertension • Pulmonary Disease • Respiratory Diseases • Septic Shock • Solid Tumor

ACCESS: HLA-Mismatched Unrelated Donor Hematopoietic Cell Transplantation With Post-Transplantation Cyclophosphamide (clinicaltrials.gov) - P2 | N=300 | Active, not recruiting | Sponsor: Center for International Blood and Marrow Transplant Research | Trial completion date: Dec 2025 ➔ Dec 2026 | Trial primary completion date: Dec 2025 ➔ Aug 2026

Trial completion date • Trial primary completion date • Acute Lymphocytic Leukemia • Acute Myelogenous Leukemia • Chronic Lymphocytic Leukemia • Chronic Myeloid Leukemia • Hematological Malignancies • Leukemia • Lymphoma • Myelodysplastic Syndrome • Oncology • Transplantation • HLA-B • HLA-C • HLA-DPB1 • HLA-DQB1 • HLA-DRB1

Reduced MBF Regimen for Patients >=55 Years With Myeloid Malignancies (clinicaltrials.gov) - P2 | N=61 | Recruiting | Sponsor: Shanghai Jiao Tong University School of Medicine | Trial completion date: Dec 2025 ➔ Jun 2027 | Trial primary completion date: Jun 2025 ➔ Dec 2026

Trial completion date • Trial primary completion date • Oncology

Clinical Analysis of Allogeneic Hematopoietic Stem Cell Transplantation for Very Early-Onset Inflammatory Bowel Disease Caused by IL-10RA Mutation (PubMed, Zhongguo Shi Yan Xue Ye Xue Za Zhi) - "Allo-HSCT is an efficacious therapeutic approach for children with VEO-IBD caused by IL-10RA mutation. The implementation of early HSCT is crucial for improving the prognosis and quality of life of children with VEO-IBD."

Journal • Retrospective data • Acute Graft versus Host Disease • Bone Marrow Transplantation • Chronic Graft versus Host Disease • Gastroenterology • Gastrointestinal Disorder • Graft versus Host Disease • Hepatology • Immunology • Inflammation • Inflammatory Bowel Disease • Pediatrics • Transplantation

Evaluation of busulfan stability in infusion bags and biological matrix: Application to routine therapeutic drug monitoring with adaptive dosing in children (AACR 2026) - "Busulfan blood samples must be rapidly proceeded in refrigerated condition upon sampling to allow reliable TDM and adaptive dosing in paediatric oncology."

Clinical • Oncology

LETERMOVIR USE IN PEDIATRIC HSCT PATIENTS UNDER 12 YEARS OLD: UPDATED DATA FROM A RETROSPECTIVE MULTICENTER STUDY OF THE PDWP AND IEWP OF THE EBMT (EBMT 2026) - "Conditioning regimens included treosulfan-based (44.5%), TBI-based (32.2%), busulfan-based (15.1%), and other (8.2%). In-vivo T-cell depletion (TCD) was used in 34.2% of patients (ATG 27.4%, Campath 6.8%), and ex-vivo TCD in 11.6%.Table 1: Patient and transplant characteristics With a median follow-up of 1 year (95% CI: 0.9–1.1), the 1-year cumulative incidence of CMV reactivation was 23.8% (95% CI: 16–30) overall, 21.4% (95% CI: 13.6–29.2) during primary prophylaxis, and 32.6% (95% CI: 13.1–45) during secondary prophylaxis (Figure 1)...Among patients with CMV reactivation, one death was attributed to relapsed disease.Letermovir was well tolerated with only two patients experiencing mild gastrointestinal toxicity, and no additional significant adverse events reported.CMV reactivations were managed using standard antiviral strategies, including valganciclovir (n=10), ganciclovir (n=8), foscarnet (n=7), and cidofovir (n=1) or continued letermovir alone (n=8)... In this..."

Retrospective data • Bone Marrow Transplantation • Cytomegalovirus Infection • Graft versus Host Disease • Hepatology • Immunology • Infectious Disease • Pediatrics

High event-free (EFS) and overall survival (OS) after non-total body irradiation (TBI) conditioning and allogeneic hematopoietic cell transplantation (HCT) in next-generation-sequencing minimal residual disease (NGS-MRD) negative B-acute lymphoblastic leukemia (B-ALL): Results from the EndRAD trial (PTCTC ONC1701) (ASH 2025) - P2 | "Based upon retrospectivedata showing low rates of relapse, we hypothesized that patients with negative pre-HCT MRD by next-generation-sequencing of IgH B-cell receptor rearrangements (NGS-MRD) could achieve 2-year EFSexceeding 75% with a non-TBI regimen, an outcome comparable to those receiving TBI-based regimens. The Pediatric Transplantation and Cellular Therapy Consortium (PTCTC) conducted a phase IIprospective trial at 45 Centers in North America (ONC1701 EndRAD: NCT03509961) between 2018 and2025 to evaluate outcomes of myeloablative non-TBI conditioning regimens for allogeneic HCT in B-ALLpatients at lower risk for relapse defined by absence of NGS-MRD (Clonoseq) of B-cell receptorrearrangements (BCR) just prior to HCT...Mismatched related/haploidentical grafts received post-transplant cyclophosphamide orTCRαβ/CD19 depletion according to institutional preference...Of patientsenrolled, 33% were White/Non-Hispanic, 37% Hispanic, 12% Black or African American, and..."

Biomarker • Clinical • IO biomarker • Minimal residual disease • Next-generation sequencing • Residual disease • Acute Graft versus Host Disease • Acute Lymphocytic Leukemia • B Acute Lymphoblastic Leukemia • CNS Disorders • Graft versus Host Disease • Hematological Malignancies • Immunology • Leukemia • Transplantation

The Efficacy and Safety of Bevacizumab/Irinotecan/Temozolomide (BIT) for Relapsed/Refractory Neuroblastoma: The UK Children's Cancer and Leukaemia Group Experience. (PubMed, Pediatr Blood Cancer) - "The promising efficacy and tolerability of BIT reported by the BEACON trial are reproduced in real-world data with a larger cohort. Further randomised studies are needed to separately identify optimal treatment strategies for relapsed and refractory disease."

Journal • Febrile Neutropenia • Hematological Disorders • Hematological Malignancies • Leukemia • Neuroblastoma • Oncology • Solid Tumor

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

ALLOGENEIC HEMATOPOIETIC STEM CELL TRANSPLANTATION FOR MYELOFIBROSIS: PATTERNS AND OUTCOME AFTER RELAPSE AND LONG-TERM FOLLOW UP (EBMT 2026) - "The conditioning regimen was fludarabine and reduced dose intravenous busulfan (FluBu, total 6.4-8 mg/kg, n=25); fludarabine, thiotepa, busulfan (TBF, n=61) or other regimens (n=16). GVHD prophylaxis included cyclosporine and methotrexate or mycophenolate and ATG (grafalon).With a median follow-up of 5.6 years (range, 0.1-22 years), 56 patients are alive and 46 have died, 30 of non-relapse causes and 16 of relapse... Allogeneic HSCT can allow long-term survival and possible cure in myelofibrosis. However, long-term survivors are at continuous risk for late relapse and late NRM. For those who are not cured, HSCT may reset the pattern of progression of the myeloproliferative process."

Acute Myelogenous Leukemia • Bone Marrow Transplantation • Cardiovascular • Chronic Graft versus Host Disease • Graft versus Host Disease • Immunology • Myelofibrosis • Polycythemia Vera • Transplantation

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

GLOBALISING HAEMATOPOIETIC STEM CELL TRANSPLANTATION FOR CEREBRAL X-LINKED ADRENOLEUKODYSTROPHY: A UK–INDIA MULTICENTRE EXPERIENCE (EBMT 2026) - "All patients received myeloablative conditioning with Fludarabine and Busulfan or Fludarabine/Treosulfan/Thiotepa plus ATG/Alemtuzumab. Allogeneic HSCT provided robust outcomes for CALD patients, demonstrating excellent engraftment, acceptable toxicity, and 100% survival. Neurological and radiological stability was maintained in all transplant recipients, contrasting sharply with the rapid decline and 50% mortality observed in the non-HSCT comparator cohort. Our multi-center experience reinforces HSCT as an effective, disease-modifying therapy."

Clinical • Bone Marrow Transplantation • Endocrine Disorders • Genetic Disorders • Graft versus Host Disease • Immunology • Nephrology • Transplantation

FACTORS ASSOCIATED WITH MIXED CHIMERISM IN CHILDREN WITH NON-MALIGNANT DISEASES TRANSPLANTED WITH A TREOSULFAN- BASED REGIMEN (EBMT 2026) - "Children with non-malignant diseases have been shown to have increasing incidence of mixed chimerism when undergoing HSCT at a younger age with melphalan-based (Fitch, et al, 2024) and with busulfan-based conditioning (Dvorak, et al, 2025)...All patients received treosulfan and fludarabine, some patients received additional serotherapy (either ATG or alemtuzumab) and/or thiotepa (10 mg/kg on day -2 before HSCT)... Treosulfan and fludarabine conditioning does not ensure durable engraftment in patients with non-malignant diseases especially in disorders of neutrophil dysfunction. Increased mixed chimerism is associated with age <6 years old, suggesting the previous observations of Fitch and Dvorak may be more universal. Still, the addition of thiotepa to conditioning was the most consistent predictor of full donor chimerism."

Clinical • Bone Marrow Transplantation • Transplant Rejection • Transplantation

LONG TERM FOLLOW-UP OF THALASSEMIA PATIENTS SHOWS REASONABLE OUTCOMES AFTER TRANSPLANTATION- A SINGLE CENTER EXPERIENCE FROM INDIA (EBMT 2026) - "Median pre-transplant ferritin was 1720 ng/ml (IQR:1260-2220), median liver size was 2 cm (IQR:2.0-3.5), median hydroxyurea exposure was 9 years (IQR:4.7-14.4 years), median number of blood transfusions before transplant was 94 (IQR: 58-142)...Five patients received treosulfan instead of busulfan as part of conditioning... Our real-world data shows excellent post-transplant outcomes in Indian children with thalassemia similar to the ones reported in western literature."

Clinical • Cardiovascular • Genetic Disorders • Graft versus Host Disease • Hepatology • Hypertension • Immunology • Infectious Disease • Transplant Rejection • Transplantation

TREOSULFAN-BASED CONDITIONING IS ASSOCIATED WITH BETTER OUTCOMES IN ADOLESCENTS AND YOUNG ADULTS UNDERGOING MATCHED RELATED DONOR TRANSPLANTATION FOR THALASSEMIA MAJOR (EBMT 2026) - "Conditioning consisted of either Busulfan–Cyclophosphamide (Bu/Cy ± ATG) or Treosulfan–Fludarabine–Thiotepa (FTT). Treosulfan-based conditioning significantly reduces early toxicity and improves long-term OS and TFS in AYA patients with TM undergoing MRD transplantation, including those in high-risk subgroups. These findings support Treosulfan-based conditioning as the preferred regimen for AYA patients in resource-constrained settings."

Clinical • Acute Graft versus Host Disease • Beta-Thalassemia • Chronic Graft versus Host Disease • Genetic Disorders • Graft versus Host Disease • Hematological Disorders • Hepatology • Immunology • Transplantation

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