fludarabine IV / Generic mfg. - LARVOL DELTA

FT576 in Subjects With Multiple Myeloma (clinicaltrials.gov) - P1 | N=31 | Completed | Sponsor: Fate Therapeutics | Active, not recruiting ➔ Completed

Monotherapy • Trial completion • Hematological Malignancies • Multiple Myeloma • Oncology

A Study of CNCT19 in the Treatment of Relapsed or Refractory Neurological Autoimmune Diseases (clinicaltrials.gov) - P1 | N=18 | Not yet recruiting | Sponsor: Juventas Cell Therapy Ltd.

New P1 trial • CNS Disorders • Immunology

Deeper remission achievement and longer leukemia-free survival with inati-cel as consolidation therapy in allogeneic transplant-ineligible adolescent and adult patients with B-ALL (ASH 2025) - P1/2 | "All patients receivedlymphodepletion with fludarabine (30 mg/m2/d ×3 days) and cyclophosphamide (500 mg/m2/d×2 days) followed by Ina-cel infusion (0.6×108 live CAR-T cells)...Specially, only one patient had grade 3 CRS and grade 3 ICANS, characterized by high fever andhypotension, and was managed with tocilizumab in combination with dexamethasone (80 mg).Additionally, 7 patients (63.6%) suffered from cytopenia... Our preliminary results of the intervention study demonstrate inati-cel, a CD19 CAR-T celltherapy leads to high MRD-negativity conversion and survival rates with manageable toxicity intransplant-ineligible B-ALL patients. More patients are being enrolled and followed up. Long-termsurvival data are likely to support CD19 CAR-T cell therapy as a new paradigm for consolidation therapyfor B-ALL patients."

Clinical • Acute Lymphocytic Leukemia • B Acute Lymphoblastic Leukemia • Bone Marrow Transplantation • Hematological Disorders • Hematological Malignancies • Infectious Disease • Leukemia • Transplantation

Update AML: Updated disease monitoring and treatment to enhance outcomes for pediatric AML (NCT07059975) (ASH 2025) - P1 | "These patients will receive the common salvage regimen Idarubicin-fludarabine-cytarabine (IdaFLA) as the second cycle (Induction 2) in lieu of standard DA8 (Daunorubicin-Cytarabine) chemotherapy...In UPDATE AML, IR patients will receive VIA in place of MA (mitoxantrone-cytarabine) to eliminate exposure to the cardiotoxic agent mitoxantrone. HR patients will receive VIA as Intensification 1 prior to SCT, in place of the genotoxic agent etoposide and to provide venetoclax exposure prior to SCT...All newly diagnosed patients at TXCH >1 month to <30 years old with non-FLT3-ITD+ AML will be eligible for enrollment after completing standard Induction 1 chemotherapy consisting of combination of daunorubicin and cytarabine +/- gemtuzumab...Over 3 years, the study is anticipated to accrue 36-40 patients and generate important feasibility data for our treatment and molecular MRD innovations. Our results will inform future cooperative group trials with respect to..."

Clinical • IO biomarker • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Pediatrics • FLT3

Venetoclax-based salvage therapy achieves high remission rates after 7+3 or low-dose cytarabine (ASH 2025) - "Introduction: In the Brazilian public health system (SUS), salvage chemotherapy for fit patients with relapsed/refractory (R/R) acute myeloid leukemia (AML) after standard 7+3 induction (7 days of cytarabine plus 3 days of an anthracycline) is usually FLAG-IDA (fludarabine, cytarabine, granulocyte colony-stimulating factor [G-CSF], and idarubicin) or MEC (mitoxantrone, etoposide, and cytarabine)...Although the venetoclax–cytarabine (VEN-ARAC) or venetoclax–azacitidine (VEN-AZA) combination is approved as first-line therapy for unfit patients, it is generally not available in SUS... In this real-world study, VEN-based salvage therapy induced rapid and high CR rates after 7+3 or LDAC, with a favorable safety profile, and may serve as a less-toxic bridge to allo-HSCT."

Clinical • Acute Myelogenous Leukemia • Bone Marrow Transplantation • Febrile Neutropenia • Hematological Disorders • Hematological Malignancies • Infectious Disease • Leukemia • Neutropenia

Real-world AML survival paradox: Early escalation vs. sustained remission (ASH 2025) - "Patients were stratified into two cohorts: Cohort A (early escalation, n=1,460), comprising patients who received either second-line chemotherapy (including fludarabine, cladribine, gilteritinib, enasidinib, revumenib, olutasidenib, etoposide, or clofarabine) or hematopoietic stem cell transplant (HSCT) as salvage therapy within 6 months of completing initial therapy; and Cohort B (no escalation, n=10,540), consisting of patients who received no additional therapy during this same period. Our findings highlight the complex interplay between disease biology and treatment interventions: achieving MRD-negativity remains critical, but when MRD-positivity occurs, subsequent treatment decisions based on disease genetics prove critical. Prospective studies incorporating molecular MRD monitoring are needed to confirm these findings and further optimize post-remission therapy approaches."

Clinical • Real-world • Real-world evidence • Acute Myelogenous Leukemia • Bone Marrow Transplantation • Hematological Malignancies • Leukemia

Efficacy and safety of CAR-t cell therapy in richter transformation: A systematic review (ASH 2025) - "Lymphodepletion regimens included fludarabine/cyclophosphamide (FC) or bendamustine. CAR-T products administered were axi-cel (n=148), tisa-cel (n=131), liso-cel (n=57), brexu-cel (n=1), and investigational agents (n=29)... CAR-T therapy shows promising initial responses in RT, with ORR and CR rates comparable to de novo DLBCL. However, responses are often short-lived, with inferior PFS and OS compared to outcomes in aggressive lymphoma trials. RT patients face higher toxicity—particularly ICANS, cytopenias, and infections—reflecting disease biology and prior treatments."

CAR T-Cell Therapy • Clinical • IO biomarker • Review • B Cell Lymphoma • Chronic Lymphocytic Leukemia • CNS Disorders • Diffuse Large B Cell Lymphoma • Hematological Disorders • Hematological Malignancies • Infectious Disease • Leukemia • Lymphoma • Neutropenia • Non-Hodgkin’s Lymphoma • Richter's Syndrome • Thrombocytopenia • IGH • TP53

A genomic analysis of patients with chronic lymphocytic leukemia treated with fixed duration therapy in a first-line setting in alberta, Canada (ASH 2025) - "For younger, fit patients with favourable-risk genetic markers such as the absence of del(17p)/ TP53 mutations and mutated IGHV, first-line (1L) chemoimmunotherapy (CIT) with fludarabine, cyclophosphamide, and rituximab (FCR) remains a reasonable option. With the emergence of targeted fixed-duration (FD) therapies, such as venetoclax plus obinutuzumab (V+O) and ibrutinib plus venetoclax (I+V), which have demonstrated superior efficacy and reduced toxicity, CIT use has declined in the 1L setting (Al-Sawaf et al., 2024; Schnaiter et al., 2024; George et al., 2025)...Among patients receiving CIT, 61 (30.5%) received FCR, 76 (38%) received bendamustine plus rituximab (BR), 26 (13%) received chlorambucil plus obinutuzumab (Clb+O), and 6 (3%) received chlorambucil plus rituximab (Clb+R)... In this chart-reviewed cohort of 200 patients, the average age at diagnosis was 65 years and 69.8% were male. 22% and 22.5% presented with Rai stage 3 or 4, respectively, at 1L therapy..."

Clinical • Genomic analysis • IO biomarker • Omic analysis • Chronic Lymphocytic Leukemia • Hematological Malignancies • Leukemia • IGH • TP53

FLT3 testing and guideline concordance in Acute Myeloid Leukemia across an Indiana health system (ASH 2025) - "Current guidelines recommend FLT3 mutation testing at diagnosis to inform risk stratification and guide the use of FLT3 inhibitors such as midostaurin, quizartinib, and gilteritinib...The most frequently used induction therapies included venetoclax in combination with a hypomethylating agent (30%), 7+3 (15.7%), and a combination regimen consisting of fludarabine, cytarabine, idarubicin, and granulocyte colony-stimulating factor (FLAG IDA; 13.8%)... FLT3 testing was commonly performed in this cohort, yet notable variability in guideline adherence was observed. Guideline-concordant induction regimen selection was marginally higher for patients with FLT3 mutations. These findings underscore the need for institutional quality improvement initiatives aimed at enhancing the documentation of FLT3 status and optimizing the integration of guideline-directed therapies in AML management."

Discordant • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • FLT3

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

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

Long-term HIV remission of a perinatally infected individual, following a hematopoietic cell transplantation from a CCR5Δ32 homozygous donor for multiple myeloma: The kyiv patient (ASH 2025) - "Complete remission (CR) was achieved after one cycle of melphalan/prednisone and local radiotherapy, but an abdominal relapse occurred six months later. Despite subsequent lines of therapy (bortezomib(bort)/lenalidomide(lena)/dexamethasone(dexa) and bendamustine/bort/dexa), the extramedullary multiple myeloma (MM) progressed. Remission was achieved after dexa/thalidomide/cisplatin/doxorubicin/cyclophosphamide/etoposide (DT-PACE) and an autologous HCT performed on 1/2021...Allogeneic HCT was performed in 8/2022 after fludarabine/melphalan conditioning and anti-thymocyte globulin from a CCR5-Δ32homozygous, unrelated HLA-matched donor; cyclosporine/methotrexate was utilized as graft-versus-host diseas e (GVHD) prophylaxis... To our knowledge, this represents the first report of ART-free HIV RNA suppression following allogeneic HCT with a CCR5Δ32homozygous donor in an individual perinatally infected with HIV. Despite the differences in the latent reservoir and the mechanism..."

Clinical • Graft versus Host Disease • Hematological Disorders • Hematological Malignancies • Human Immunodeficiency Virus • Immunology • Infectious Disease • Multiple Myeloma • Plasmacytoma • Transplantation • CCR5 • CD4 • CD8

Good report of secondary allogeneic hematopoietic stem cell transplantation in patients with refractory/relapsed Acute Myeloid Leukemia (ASH 2025) - "Thirty-five patients received a conditioning regimen primarily consisting of TBI plus fludarabine/cladribine, while 8 patients received a regimen primarily consisting of BU plus fludarabine/cladribine. Achieving CR prior to secondary HSCT is associated with higher post-transplantation OS rates. In cases where CR cannot be achieved, salvage secondary HSCT is also a viable option."

Clinical • Acute Graft versus Host Disease • Acute Myelogenous Leukemia • Bone Marrow Transplantation • Chronic Graft versus Host Disease • CNS Disorders • Cytomegalovirus Infection • Epstein-Barr Virus Infections • Graft versus Host Disease • Hematological Malignancies • Immunology • Infectious Disease • Leukemia • Novel Coronavirus Disease • Pneumonia • Respiratory Diseases • Transplantation

Risk of relapse and mortality after non-myeloablative allogeneic stem cell transplant for Acute Myeloid Leukemia and myelodysplastic syndrome after fludarabine, cyclophosphamide, and 200 centigray total body irradiation with gvhd prophylaxis using post-transplant cyclophosphamide, sirolimus, and mycophenolate mofetil: Impact of maintenance chemotherapy (ASH 2025) - "Oral HMA agents were decitabine/cedazuridine (dec-c) 3d/28-day cycle, and oral azacitidine (Oral-Aza) for 14 days per 28-day cycle...A total of 32 (31%) patients received post-HSCT maintenance chemotherapy: infusional HMA (n=2), recombinant-Granulocyte Colony Stimulating Factor (rhGCSF)/decitabine (n=2), gilteritinib (n=7), dec-c (n=9), oral-aza (2), and venetoclax/Azacitidine (n=8)... NMA conditioning with Flu/Cy/2Gy TBI yields low NRM. Post-transplant maintenance therapy, particularly with HMAs and targeted agents, is associated with reduced relapse in intermediate-risk and adverse-risk TP53 wt AML and MDS. However, outcomes remain poor for patients with TP53 mu t AML and MDS, indicating an urgent need for alternative or intensified maintenance strategies in these high-risk groups."

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 • Myelodysplastic Syndrome • Transplantation • FLT3 • TP53

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

Clinical efficacy and safety for pediatric patients with severe combined immunodeficiency disease treated with modified allo-haploidentical stem cell transplantation (ASH 2025) - "A modified transplant protocol with PTCy (25-35 mg/kg/day on days +3 and +4) was performed, including fludarabine 160 mg/m2 divided into 4 days on day -8 to -5, Melphalan 100 mg/m 2 divided into two days on day -3, -2, Cyclophosphamide 20mg/kg/day on day -5, -4 and porcrine anti-human lymphocyte immunoglobulin 25mg/kg/d on day -8 to -6. Our modified protocol provides a new treatment method for SCID patients without suitable donor, expanded donor pool. Using the reduced intensity conditioning regimen, reduced toxicity and heavy economic burden, lower rate of GVHD increased high quality of life. Due to the small sample size, further confirmation is needed with a larger number of cases."

Clinical • Bone Marrow Transplantation • Genetic Disorders • Graft versus Host Disease • Immunology • Infectious Disease • Novel Coronavirus Disease • Pediatrics • Primary Immunodeficiency • Pulmonary Disease • Respiratory Diseases • Transplantation

Effectiveness of belumosudil in acute graft-versus-host disease (aGVHD) after allogeneic hematopoietic stem cell transplantation (allo-HSCT): A real-world observational study (ASH 2025) - "All patients received myeloablative conditioning, with 75% of patients administered the fludarabine plus modified TBI regimen incorporating craniospinal, marrow, and nodal targets. Standard GVHD prophylaxis was cyclosporine/MMF/methotrexate + anti-CD25/ATG/ALG...Conclusion : This study provides the first disclosed real-world evidence of belumosudil in acute GVHD, demonstrating unprecedented 100% ORR (75% cases with a complete response) with rapid median time to response of 13.5 days and well tolerated. Despite the inherent limitations of this pilot investigation, these findings warrant validation in pivotal multicenter trials."

Clinical • Observational data • Real-world • Real-world evidence • Acute Graft versus Host Disease • Bone Marrow Transplantation • Chronic Graft versus Host Disease • Dermatology • Graft versus Host Disease • Hematological Malignancies • Immunology • Infectious Disease • Leukemia • Respiratory Diseases • Transplantation • CD80 • CD86 • IL17A • IL21 • STAT5

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

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

Paroxysmal nocturnal hemoglobinuria: Transplant experience using post-transplant cyclophosphamide in a resource-limited setting (ASH 2025) - "On the other hand, complement inhibitors (eculizumab, ravulizumab) have emerged as effective treatments with lower toxicity, though they are not curative...MRD transplant patients received Fludarabine/Melphalan (Flu/Mel) and haploidentical transplant received standard Baltimore protocol...Among them, two patients developed CMV reactivation, which was well controlled with valganciclovir... Allogeneic HSCT remains a potentially curative but high risk option for PNH patients, particularly in resource limited settings. Our experience highlights the significant transplant related morbidity and mortality, even with modern GVHD prophylaxis such as PTCy.Given the availability of safer, though non-curative, therapeutic alternatives like complement inhibitors, urgent advocacy is needed. Patient groups, healthcare providers, policy-makers, and international aid organizations must collaborate to improve access to life-saving treatments in low-income countries."

Post-transplantation • Aplastic Anemia • Bone Marrow Transplantation • Cardiovascular • Chronic Graft versus Host Disease • Complement-mediated Rare Disorders • Febrile Neutropenia • Gastroenterology • Gastrointestinal Disorder • Graft versus Host Disease • Hematological Disorders • Hematological Malignancies • Hepatology • Immunology • Infectious Disease • Inflammation • Mucositis • Myelodysplastic Syndrome • Paroxysmal Nocturnal Hemoglobinuria • Pneumonia • Rare Diseases • Respiratory Diseases • Thrombosis • 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

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

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