Campath (alemtuzumab)
/ Bayer, Sanofi
- LARVOL DELTA
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December 05, 2025
Long-term update on safety and efficacy of low-dose post-transplant cyclophosphamide in the HLA-mismatched unrelated transplant setting
(ASH 2025)
- "Materials and Methods A prospective, single-centre study was conducted to assess the safety and efficacy of reduced-dose PTCy (15 or 25mg/kg), combined with low-dose alemtuzumab (5mg/day for 2 days) and cyclosporin, as GvHD prophylaxis in 9/10 mismatched unrelated (MMUD) peripheral blood stem cell transplant (PBSCT) setting (study group). Moreover, the ability to tailor PTCy dosing based on disease and relapse risk could allow for a personalized approach to GvHD prevention. These results warrant validation in larger prospective, randomized studies and exploration of novel PTCy-based combinations, potentially paving the way for new standards in GvHD prophylaxis."
Clinical • Post-transplantation • Acute Graft versus Host Disease • Chronic Graft versus Host Disease • Graft versus Host Disease • Hematological Disorders • Immunology • Transplantation
December 05, 2025
Weight based dosing of alemtuzumab as part of an enhanced lymphodepletion regimen may mitigate the risks of over and underexposure and improve response rates : Analysis of the nathali-01 trial
(ASH 2025)
- "LD regimens with alemtuzumab are currently being investigated in multiple clinical studies including the NatHaLi-01 study, a first-in-human, open-label, dose-finding and dose-expansion study of the dual CAR-T therapy UCART20x22 administered intravenously in subjects with relapsed or refractory B-Cell Non-Hodgkin Lymphoma (B-NHL). Adequate alemtuzumab exposure is important in achieving adequate lymphodepletion and disease response due to the positive impact on CAR-T cell expansion and the delay in host immune reconstitution. Increased weight with resultant lower alemtuzumab exposure may be associated with early host immune reconstitution and limited CAR T-cell expansion and suboptimal response. However, while adequate exposure is important, overexposure may be also associated with unnecessary toxicities which alters the risk benefit assessment."
B Cell Non-Hodgkin Lymphoma • Hematological Malignancies • Lymphoma • Non-Hodgkin’s Lymphoma
December 05, 2025
A single center review of secondary hemophagocytic lymphohistiocytosis treatment and outcomes.
(ASH 2025)
- "Initial therapy was malignancy-specific chemotherapy (39%), HLH-94 protocol based therapy (30%), steroids alone (23%), and anakinra (2%) while others were not treated (6%)...Overall, 46% of patients required several lines of treatment and were subsequently treated with several additional agents in the second line setting including malignancy directed therapy, ruxolitinib, alemtuzumab, eculizumab, IVIG, cyclosporine, tacrolimus and rituximab...To improve timely diagnosis and treatment decisions, we, like other centers, are implementing electronic medical record order sets for HLH diagnosis and management and organizing an HLH expert panel to provide timely case reviews and treatment recommendations. In memory of our esteemed colleague and co-author Stephen Njau, MD"
Clinical • Review • Dyslipidemia • Hematological Malignancies • Hemophagocytic lymphohistiocytosis • Hypertriglyceridemia • Immunology • Infectious Disease • Rare Diseases • Solid Tumor • IL2RA • ISG20
November 04, 2025
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
November 04, 2025
Impact of fludarabine exposure on overall survival in patients receiving fludarabine/cyclophosphamide reduced intensity conditioning hematopoietic cell transplantation for hematologic malignancies
(ASH 2025)
- P1/2, P2 | "Graft-versus-host disease (GVHD) prophylaxis consisted of either alemtuzumab andcyclosporine (AC) or tacrolimus, methotrexate, and sirolimus (TMS). While our findings suggest that the current dosing approach is effective in preventingFlu overexposure, larger studies are needed to adequately assess the impact of Flu exposure on survivaloutcomes. Future prospective research is warranted to refine and validate optimal Flu exposure in theRIC alloHCT setting."
Clinical • Acute Myelogenous Leukemia • Chronic Lymphocytic Leukemia • Graft versus Host Disease • Hematological Malignancies • Immunology • Oncology • Transplantation
November 04, 2025
Increased alemtuzumab exposure correlates with improved responses in heavily pretreated R/R ALL patients: Analysis of the balli-01 trial
(ASH 2025)
- "Fludarabine,cyclophosphamide and alemtuzumab (FCA) was investigated as an LD regimen. Alemtuzumab is a critical component of the LD regimen in the BALLI-01 trial. For thisallogeneic CAR-T therapy, it is important to ensure adequate exposure to alemtuzumab in order tooptimize cell expansion and mitigate the risk of early host T cell reconstitution which may compromiseresponse in these heavily pretreated ALL patients with few, if any, alternative treatment options."
Clinical • Acute Lymphocytic Leukemia • B Acute Lymphoblastic Leukemia • Hematological Malignancies • Leukemia • CD52
November 04, 2025
MO-TRANS update: A randomized, double-blind, placebo-controlled, multi-center phase III study of mocravimod (MOC) as maintenance treatment in AML patients undergoing allogeneic hematopoietic cell transplantation
(ASH 2025)
- P1, P3 | "Use of ATG,alemtuzumab, cyclosporine A (CsA), and abatacept is excluded; other GvHD prophylaxis agents such aspost-transplant cyclophosphamide (PTCy) are permitted. There is an unmet medical need to maintain CR after allo-HCT, especially in AMLpatients with high risk factors or previous relapse. The MO-TRANS study aims to validate the therapeuticpotential of MOC in reducing relapse and GvHD in patients allografted for AML."
Clinical • P3 data • Acute Graft versus Host Disease • Acute Myelogenous Leukemia • Chronic Graft versus Host Disease • Diabetes • Graft versus Host Disease • Hematological Malignancies • Immunology • Leukemia • Macular Edema • Metabolic Disorders • Ocular Inflammation • Ophthalmology • Transplantation • Uveitis
November 04, 2025
Improved outcomes following allogeneic hematopoietic stem cell transplantation in patients with DDX41-mutated myeloid neoplasms: A prospective analysis of survival, GVHD, and transplant-related mortality
(ASH 2025)
- "The median interval between diagnosis andtransplant was 10.3 months ( 5.3–58.5).In keeping with the advanced age and comorbidities in this cohort, reduced-intensity conditioningregimens with fludarabine, busulphan and Alemtuzumab (57.8%) were predominantly used. Allogeneic HSCT in patients with DDX41-mutated myeloid neoplasms yielded favourable one-year overalland relapse-free survival rates, despite the cohort's older age and mixed disease spectrum. Thepredominance of reduced-intensity conditioning facilitated transplantation in this higher-risk groupwithout excess early toxicity or graft failure. Rates of acute and chronic GVHD were manageable andconsistent with patients with wild-type DDX41."
Clinical • 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 • DDX41 • TP53
November 04, 2025
Secondary malignancies after hematopoietic cell transplantation for sickle cell disease: A single-center experience
(ASH 2025)
- "This study aims to describe our single-center experience and evaluate theoccurrence and clinical characteristics of secondary malignancies following HCT in patients with SCD.We conducted a retrospective review of all patients with sickle cell disease (SCD) who underwent non-myeloablative conditioning withalemtuzumab and 300 cGy total body irradiation (TBI) for allogeneic hematopoietic cell transplantation (HCT) at our center...Diagnoses included MDS (n=2), MDS transformed to AML (n=1), CML (n=1), Philadelphia-positive ALL (n=1), and EBV-related PTLDwith stage IV DLBCL (n=1).Summary of Patients with Secondary MalignancyPatientAgeGenderTime post-transplantDiagnosisChimerism atDiagnosisGeneticAbnormalitiesTreatmentCurrentStatusLatestChimerismHbS (%)138Male4.8 yearsGraft failure MDS0%del(7q31),del(13q14),ASXL1, PTPN11,RUNX12nd transplant(FluBu4),AzacitidineMDSrecurrencepost 2nd HCT11%4.1229Female3.5 yearsGraft failurepost 2ndand 3rd..."
Clinical • Acute Myelogenous Leukemia • Bone Marrow Transplantation • Diffuse Large B Cell Lymphoma • Genetic Disorders • Hematological Malignancies • Infectious Disease • Leukemia • Oncology • Sickle Cell Disease • Transplantation • ASXL1 • PTPN11 • TP53
November 04, 2025
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
November 04, 2025
Universal base-edited CAR7 T cells for T-cell acute lymphoblastic leukemia
(ASH 2025)
- "BE-CAR7 T cells were infused afterlymphodepletion (LD) with fludarabine (150 mg per square meter), cyclophosphamide (120 mg/kg) andalemtuzumab (1 mg/kg). As anticipated, viral reactivations were frequent, and three patientsexperienced significant virus-related morbidity post-transplant. Overall, 7/11 (63%) subjects dosed werein ongoing remission 3-36 months after transplant, and suspected CD7 negative leukemic escape hasbeen documented in 2 patients.Conclusions Universal BE-CAR7 T cells offer the prospect of leukemic remission for patients with CD7+ r/rT-ALL ahead of allo-SCT and will be further assessed in children and adults in extended cohorts."
Acute Lymphocytic Leukemia • Bone Marrow Transplantation • CNS Disorders • Dermatology • Graft versus Host Disease • Hematological Malignancies • Immunology • Leukemia • T Acute Lymphoblastic Leukemia • T-cell Acute Lymphoblastic Lymphoma • CD52 • CD7
November 04, 2025
Acute Myeloid Leukemia (AML) with TP53 alteration and outcomes after allogeneic hematopoietic stem cell transplantation: A CIBMTR registry study
(ASH 2025)
- "Post-transplantcyclophosphamide (PTCy) was used in 27%; and in vivo T-cell depletion (ATG or alemtuzumab) in 26%.After median follow-up of 66 months (3-130), 5-yr OS was 18% (Group 1), 18% (Group 2), 35% (Group 3) vs46% in controls (p<0.001)...This large, registry-based study with long term median follow-up showed that among patients withadverse-risk AML, TP53alt is a strong independent predictor of poor post-HCT outcomes in AML,particularly when TP53del is present. Patients with TP53del , with or without concurrent TP53mut,experienced the worst outcomes, followed by those with isolated TP53mut. Despite the limitations ofregistry data including lack of information on timing of TP53mut, variant allele frequency (VAF), number ofmutations, or accurate information on measurable residual disease (MRD), these findings reinforce theadverse prognostic impact of TP53alt and support its inclusion in risk stratification frameworks."
Acute Myelogenous Leukemia • Bone Marrow Transplantation • Graft versus Host Disease • Hematological Malignancies • Immunology • Leukemia • Transplantation • TP53
November 04, 2025
Reduced chronic graft versus host disease after allogeneic HSCT with cryopreserved peripheral blood stem cell grafts: A CIBMTR analysis
(ASH 2025)
- "Risk factors considered: patient age, donor age, donor type, ATG/Campath,Karnofsky score, conditioning regimen, GVHD prophylaxis, HCT-CI, refined disease risk index, donor-recipient CMV match, donor-recipient sex match, and year of HCT...The cohorts were similar in most keypatient and transplant characteristics except more use of post-transplantation cyclophosphamide (PTCy)with cryo (39.7%) than fresh (30.8%)...These findings suggest that cryopreservation has aclinically significant negative impact on graft quality in terms of engraftment, NRM and survival despite areduction in cGVHD. Further study is needed to understand the biological effect of cryopreservation on Tcell function leading to reduced cGVHD."
Acute Graft versus Host Disease • Bone Marrow Transplantation • Chronic Graft versus Host Disease • Graft versus Host Disease • Immunology • Infectious Disease • Novel Coronavirus Disease
November 04, 2025
Initial clinical data from the phase 1 study of DR-01, a non-fucosylated anti-CD94 antibody in patients with large granular lymphocytic leukemia
(ASH 2025)
- P1/2 | "Prior therapies includedmethotrexate in 29 (94%), cyclophosphamide in 15 (48%), cyclosporine in 13 (42%), and alemtuzumab in 4(13%), with 17 (55%) pts also receiving some other treatment including investigational agents. Preliminary safety and efficacy data show that DR-01 is well-tolerated with a promisingresponse rate and durable responses, supporting continued development of DR-01 as a therapy forLGLL."
Clinical data • P1 data • Hematological Malignancies • Infectious Disease • Leukemia • Lymphoma • Neutropenia • CD8 • GZMB • KLRD1
November 04, 2025
MYC/GSPT1 protein degradation in refractory/relapsed T-cell prolymphocytic leukemias
(ASH 2025)
- "T-PLLs are one of the most challenging hematologic malignancies based onrefractoriness to conventional chemotherapy and targeted therapies, including alemtuzumab.Chromosome rearrangement and overexpression of TCL-1 are hallmarks of T-PLL, accounting forapproximately 95% of cases...We investigated the activity of GT19715 in T-PLL patient-derivedxenograft (PDX) models established from relapsed/refractory T-PLL patients in vitro and in vivo... We identified increased protein translation of c-Myc and GSPT1 in T-PLL cells compared tonormal PBMCs. Targeted protein degradation of c-Myc and GSPT1 is highly effective inrelapsed/refractory T-PLL in vitro and in vivo, with over 900% extension of survival in a clinically relevant,PDX T-PLL model, providing rationale for clinical investigations."
IO biomarker • Hematological Malignancies • Leukemia • Lymphoma • Prolymphocytic Leukemia • Targeted Protein Degradation • BCL2 • CD2 • CD69 • CD7 • DPP4 • GSPT1 • MCL1 • MYC • STAT5 • STAT5AWqe • ZAP70
November 04, 2025
Clonal T-large granular lymphocytes (LGL) in chronic relapsing-remitting and refractory immune thrombocytopenia
(ASH 2025)
- "Three highly refractory patients achieved a long term stableresponse with T cell directed therapy, 2 with sirolimus and 1 with cyclosporine. The patient with thelongest disease history, post splenectomy and highly refractory required alemtuzumab to respond after aprolonged hospitalization due to platelet counts <5,000 and ongoing bleeding..ConclusionIn this ongoing study of LGL in patients with chronic ITP, the population described in this preliminaryreport had a more refractory clinical course with a history of multiple relapses while being treated withmultiple therapies...This has not been recommended in guidelines. An alternateapproach would be to perform lymphocyte flow analysis incorporating antibodies for CD57on patientswho have become refractory to antibody and B-cell targeted therapies."
IO biomarker • Immune Thrombocytopenic Purpura • Immunology • Inflammatory Arthritis • Lupus • Neutropenia • Thrombocytopenia • Thrombocytopenic Purpura • B3GAT1 • CD8 • STAT3
November 04, 2025
Trial in progress: Open-label dose-finding and dose-expansion study to evaluate the safety, expansion, persistence, and clinical activity of UCART20x22 in subjects with relapsed or refractory B-cell non-Hodgkin lymphoma (B-NHL) nathali-01
(ASH 2025)
- P1/2 | "After Lymphodepletion with FCA3 (fludarabine 30 mg/m2 × 3d,cyclophosphamide 0.5g/m2 × 3d, CLLS52 in a weight-based dose x 3 or 4d), a single infusion ofUCART20x22 is administered at a flat dose level ([DL1] 50 x 106 cells; [DL2] and 150 x 106 cells). Primary endpoints are incidence of dose-limiting toxicities andthe incidence and severity of treatment-emergent adverse events. Secondary endpoints includealemtuzumab PK parameters, objective response rate, duration of response, progression-free survival,and overall survival."
Clinical • IO biomarker • B Cell Non-Hodgkin Lymphoma • CNS Lymphoma • Graft versus Host Disease • Hematological Malignancies • Immunology • Lymphoma • Non-Hodgkin’s Lymphoma • CD22
November 04, 2025
Autologous, allogeneic haemopoietic stem cell transplantation and CART cell therapy for tranformed Waldenström's macroglobulinemia to high grade non-Hodgkin lymphoma. real world outcomes report from the lymphoma working party of the european society for blood and marrow transplantation (LWP EBMT)
(ASH 2025)
- "29 (63%) patients had in vivo T cell depletion (ofwhom 21 (46%) with ATG, 8 (17%) with Campath) and 5 had post-transplant cyclophosphamide (PTCY).With a median follow up of 7.8 years (3.1-8.2) the estimated OS was 43%, PFS was 29%, RI was 55%, NRMwas 17% and graft versus host disease (GVHD)-free/relapse-free survival (GRFS) 21% at 2 yearsrespectively. The limited available data reported poor outcomes of transformed WM to HGNHL. This EBMTretrospective analysis, the largest reported cohort of transformed WM patients to date, showed thatthese patients could be rescued with cellular therapies."
CAR T-Cell Therapy • Clinical • Real-world • Real-world evidence • Acute Graft versus Host Disease • Bone Marrow Transplantation • Graft versus Host Disease • Hematological Disorders • Hematological Malignancies • Immunology • Lymphoma • Lymphoplasmacytic Lymphoma • Non-Hodgkin’s Lymphoma • Transplantation • Waldenstrom Macroglobulinemia
November 04, 2025
Letermovir cytomegalovirus prophylaxis in hematopoietic-cell transplantation; Real world evidence from high endemic regions.
(ASH 2025)
- "Patients were categorized intotwo groups:Study Group: Patients at ultra-high risk for CMV reactivation—defined as those who receivedhaploidentical or mismatched transplants, had R+/D− CMV serostatus, or received T-cell depletionwith anti-thymocyte globulin (ATG) or alemtuzumab (Campath)—who received letermovirprophylaxis and completed at least one year of post-transplant follow-up.Control Group: Patients with similar risk profiles who did not receive letermovir prophylaxis butcompleted at least one year of follow-up.The primary objective was to assess the proportion of patients with clinically significant CMV infection(csCMVi) through Week 24 post-transplant among those without detectable CMV DNA at baseline.Secondary objectives included:Proportion of patients with csCMVi through Week 14Time to csCMVi through Week 24Transplant-related mortality (TRM)Graft failureIncidence and severity of graft-versus-host disease (GVHD)Clinical and laboratory data were collected from..."
Clinical • HEOR • Real-world • Real-world evidence • Bone Marrow Transplantation • Cytomegalovirus Infection • Genetic Disorders • Graft versus Host Disease • Hematological Disorders • Hematological Malignancies • Immunology • Infectious Disease • Leukemia • Sickle Cell Disease • Transplantation
November 04, 2025
GVHD intel 1.0: A scalable, HLA-enhanced machine learning model for predicting acute and chronic GVHD
(ASH 2025)
- "Two distinct test cohorts were used: UAE46 (46 allogeneic HSCT recipients from UAE) andKHCC150 (150 adult allogeneic HSCT recipients from KHCC), collected later and independently of trainingdata.Variables included donor type, sex mismatch, disease status, time from diagnosis to transplant, andagents used in conditioning and GVHD prophylaxis (e.g., TBI, ATG, alemtuzumab use etc.)...GVHD-Intel 1.0 is a robust, interpretable machine learning framework developed from real-world,multicenter data. By integrating widely available clinical variables and HLA allele data, it outperformsconventional clinical risk scores in predicting both acute and chronic GVHD in allo-HSCT recipients.Slightly reduced performance in aGVHD prediction is likely due to the lack of relevant variables. Nextsteps include prospective validation across multiple transplant centers globally and continued refinementthrough expanded data integration."
Machine learning • Acute Graft versus Host Disease • Bone Marrow Transplantation • Chronic Graft versus Host Disease • Graft versus Host Disease • Immunology
November 04, 2025
Venetoclax-based therapies in patients with Relapsed/Refractory T-cell prolymphocytic leukemia
(ASH 2025)
- "The median number of prior lines of therapy before VEN initiation was 1(range, 1–5); 19 pts (54%) were treated with VEN-based therapy in 1st salvage while 16 pts (46%) weretreated in 2nd or later salvage.VEN was combined with cladribine (+/- additional agents) in 17 pts (49%), including 8 (23%) who alsoreceived ruxolitinib. Nine pts (26%) were treated with VEN and pentostatin (+/- alemtuzumab), 3 (9%) withFCM (fludarabine, cyclophosphamide, mitoxantrone), 2 (6%) with alemtuzumab alone, 2 (6%) withruxolitinib alone, and 1 (3%) each with bendamustine or as monotherapy... Pts with R/R T-PLL continue to face poor prognosis. VEN-based therapies demonstrateencouraging response rates in this refractory, proliferative, heavily pretreated population, with improvedsurvival observed among responders and those with longer duration of 1st remission. However, OSremains short, highlighting the need for prospective trials to optimize VEN-based treatment and postremission strategies in..."
Clinical • IO biomarker • Hematological Disorders • Hematological Malignancies • Infectious Disease • Leukemia • Neutropenia • Prolymphocytic Leukemia • Thrombocytopenia • JAK1 • JAK3 • STAT5B
November 04, 2025
Heterogeneity of circulating CD57+ cytotoxic cell subsets across disease stage and aggressiveness in cutaneous T cell lymphoma
(ASH 2025)
- "Aggressivecourse was defined as death from disease within two years or progression requiring systemic HDACinhibitors, chemotherapy, or alemtuzumab after failure of mogamulizumab and extracorporealphotopheresis. Circulating CD57⁺ subsets demonstrate significant variation across CTCL disease states and SSsubgroups. CD57⁺CD4⁺ T cells were most elevated at SS diagnosis, particularly in aggressive cases,suggesting a potential role in disease onset or progression. CD57⁺ NK cells were increased in bothaggressive SS at diagnosis and less aggressive treated SS, indicating a possible immune response fromtreatment or disease burden rather than aggressiveness alone."
Heterogeneity • Cutaneous T-cell Lymphoma • Dermatology • Dermatopathology • Hematological Malignancies • Human Immunodeficiency Virus • Immunology • Infectious Disease • Lymphoma • Mycosis Fungoides • Sezary Syndrome • Solid Tumor • T Cell Non-Hodgkin Lymphoma • B3GAT1 • CD4 • CD8
November 04, 2025
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
November 04, 2025
Impact of pre-transplant bone marrow cellularity and fibrosis on post-transplant outcomes in sickle cell disease
(ASH 2025)
- P1/2 | "All patients received alemtuzumab and TBI with post-HCT cyclophosphamide; some received additional pre-conditioning with pentostatin andcyclophosphamide. Our initial data suggest that neither baseline cellularity nor fibrosis significantly impacted HCT outcomes.Interestingly, all patients with fibrosis ratings > 2 demonstrated graft failure. Further, we observe thatbaseline cellularity is associated with the presence of DDR mutations before HCT. We will expand to ournon-myeloablative matched-sibling HCT cohort, increasing our overall study sample size and moredefinitively assessing the impact of baseline cellularity and fibrosis values on HCT outcomes and CH.Further studies are indicated to evaluate whether increased erythropoietic stress is associated with anincreased prevalence of baseline DDR-CH."
Post-transplantation • Pre-transplantation • Fibrosis • Genetic Disorders • Hematological Disorders • Immunology • Sickle Cell Disease • Transplant Rejection • Transplantation
November 04, 2025
A decade of progress: A comparative analysis of real-world survival in chronic lymphocytic leukemia across therapeutic eras
(ASH 2025)
- "The ECOG E1912 randomized study demonstratedsuperior overall survival of ibrutinib-based therapy compared to chemoimmunotherapy with fludarabine,cyclophosphamide, and rituximab (FCR)...For these cohorts, 5-year all-cause mortality was comparedbetween the pre-2015 and 2015-2020 groups, and 2-year all-cause mortality was compared between thepre-2015 and post-2020 groups.Second, to directly compare treatment effectiveness, patients who received systemic therapy werestratified into two cohorts based on the class of agent received, irrespective of the treatment date:Chemoimmunotherapy (CIT): Regimens including FCR, BR, Fludarabine with Rituximab,Chlorambucil ± Obinutuzumab, and Alemtuzumab-based treatments.Novel Targeted Therapy: Regimens including covalent BTK inhibitors (Ibrutinib, Acalabrutinib,Zanubrutinib), BCL-2 inhibitor Venetoclax (± Rituximab or Obinutuzumab), and PI3K inhibitors(Idelalisib, Duvelisib).For these treatment-regimen cohorts, 10-year outcomes..."
Clinical • Real-world • Real-world evidence • Chronic Lymphocytic Leukemia • Hematological Malignancies • Leukemia
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