Campath (alemtuzumab)
/ Bayer, Sanofi
- LARVOL DELTA
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March 25, 2026
Nonmyeloablative pentostatin-cyclophosphamide preconditioning improves rates of engraftment in adults undergoing haploidentical HCT for sickle cell disease.
(PubMed, PLoS One)
- P1/2 | "Further study to determine an optimal nonmyeloablative haploidentical regimen for SCD patients with compromised organ function is imperative."
Journal • Acute Graft versus Host Disease • Chronic Graft versus Host Disease • Chronic Kidney Disease • Fibrosis • Genetic Disorders • Graft versus Host Disease • Hematological Disorders • Hematological Malignancies • Immunology • Nephrology • Oncology • Sickle Cell Disease • Transplant Rejection • Transplantation
December 09, 2025
Universal Base-Edited CAR7 T Cells for T-Cell Acute Lymphoblastic Leukemia.
(PubMed, N Engl J Med)
- "Universal BE-CAR7 T cells induced leukemic remission in patients with relapsed or refractory T-cell ALL, thus allowing successful allogeneic hematopoietic stem-cell transplantation in most of the patients. (Funded by the Medical Research Council and others; ISRCTN Registry number, ISRCTN15323014.)."
Journal • Acute Lymphocytic Leukemia • Bone Marrow Transplantation • Hematological Disorders • Hematological Malignancies • Infectious Disease • Leukemia • Oncology • Palliative care • T Acute Lymphoblastic Leukemia • T-cell Acute Lymphoblastic Lymphoma • Transplantation • CD52
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
March 18, 2026
Preclinical immunotoxicity assessment of bispecific antibodies using an ex vivo circulating human whole blood assay
(AACR 2026)
- "IL-2 was, as expected, not produced in response to either obinutuzumab or alemtuzumab, but was induced to high levels (similar to an agonistic anti-CD28 antibody) in the presence of glofitamab. Neither obinutuzumab nor glofitamab had any effect on complement activation in ID.Flow, whereas alemtuzumab induced increased levels of C3a and C5a.In conclusion, ID.Flow can be used to assess the immunotoxicity and potentially the efficacy of bispecific antibodies with glofitamab as a relevant clinical control."
Bispecific • Preclinical • B Cell Lymphoma • Diffuse Large B Cell Lymphoma • Hematological Malignancies • Lymphoma • Non-Hodgkin’s Lymphoma • Oncology • CD22 • CXCL8 • IFNG • IL2 • IL6 • TNFA
February 07, 2026
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
November 04, 2025
Incorporation of 8Gy total body irradiation into reduced intensity conditioning does not improve allogeneic transplant outcomes for high-risk adult acute lymphoblastic leukemia: Results from the randomised prospective phase 2 UK multicentre ALL-RIC impact study
(ASH 2025)
- "We therefore performed a prospective,randomised comparison of the UK FMA RIC regimen with a cyclophosphamide plus 8Gy TBI RIC protocol(Cy/8TBI), with the goal of improving OS in high-risk adult ALL.MethodsThe FMA RIC regimen (fludarabine 30mg/m2 IV for 5d; melphalan 140mg/m2 IV single dose; alemtuzumab 30mg IV D-1 for sibling donor/20mg IV D-2 & -1 for unrelated donor) was compared tocyclophosphamide 50mg/kg for 2d plus 8Gy TBI (4# over 2d) and alemtuzumab (dosed as per FMA).Intrathecal prophylaxis was given for 2y post-SCT. However, incorporation of 8Gy TBI into a RIC protocol designed for older adults was welltolerated, with no increases in acute or chronic GvHD, and no additional infectious or extramedullarytoxicity.These data highlight the importance of performing randomised trials of innovative conditioningregimens, if outcomes for older patients receiving allo-SCT for ALL are to be improved. Importantly theALL-RIC trial showcases feasibility and patient..."
Clinical • P2 data • Acute Graft versus Host Disease • Acute Lymphocytic Leukemia • Chronic Graft versus Host Disease • Graft versus Host Disease • Hematological Malignancies • Immunology • Infectious Disease • Leukemia • T Acute Lymphoblastic Leukemia • Transplantation • KMT2A
March 16, 2026
Evaluation of Pneumocystis jirovecii (PJP) and Cytomegalovirus (CMV) Prophylaxis Duration in Kidney Transplant Recipients
(UKKW 2026)
- "Valganciclovir is also administered to patients receiving alemtuzumab (Campath) induction immunosuppression, unless both donor and recipient are CMV- negative...57 patients were identified as having been started on co-trimoxazole/atovaquone, and 17 patients on valganciclovir, using a data collection tool developed by the Leicester Kidney Pharmacy Team... Of the 57 patients on PJP prophylaxis, 13 (23%) continued treatment beyond the 6 month period. Similarly, 6 out of 17 patients (35%) prescribed valganciclovir extended beyond the recommended duration. No documented clinical reasons justified these prolonged courses."
Clinical • Cytomegalovirus Infection • Immunology • Infectious Disease • Pneumonia • Respiratory Diseases • Transplant Rejection • Transplantation
March 16, 2026
Alemtuzumab-associated accommodative spasm in a kidney transplant recipient: a rare neuro-ophthalmic complication
(UKKW 2026)
- "Implication Transplant clinicians should consider accommodative spasm in patients with unexplained blurred vision and request cycloplegic refraction before attributing symptoms to metabolic derangements or drug toxicity. Next step Systematic incorporation of ophthalmic surveillance into transplant pharmacovigilance, registry-based adverse event reporting, and cross-specialty awareness in both nephrology and ophthalmology guidelines are needed to clarify incidence, mechanisms, and long-term outcomes of this rare complication."
Clinical • CNS Disorders • Diabetes • Fibrosis • Genetic Disorders • Hepatology • Immunology • Liver Cirrhosis • Metabolic Disorders • Multiple Sclerosis • Nephrology • Ocular Inflammation • Ophthalmology • Optic Neuritis • Retinal Disorders • Transplantation
March 16, 2026
Audit of prevalent BK viraemia and outcomes at Nottingham
(UKKW 2026)
- "Four patients had received Campath induction and two patients ATG for earlier rejection episodes, with four patients developing subsequent biopsy proven rejection after BK viraemia was detected, and immunosuppression had been reduced... BK viraemia was identified in a small number of our prevalent cohort and detected as a result of a rise in creatinine. In the majority of cases BK became undetectable but a small number of patients went on to lose their grafts due to rejection or BK nephropathy. There is data to suggest a link with bladder cancer and of the four cases in our cohort, half stained positive for BK."
Bladder Cancer • Genito-urinary Cancer • Infectious Disease • Respiratory Diseases • Solid Tumor • Urethral Cancer
March 16, 2026
Efficacy of avacopan therapy beyond 12 months in ANCA-Associated Vasculitis: Insights From Real-World Practice
(UKKW 2026)
- "All 13 had had extensive prior exposure to immunosuppression (median 3 agents; range 1–8); 6/13 had received ≥8 g rituximab and 5/13 ≥8 g cyclophosphamide, 5/13 had been steroid-dependent for more than 5 years. Additional prior therapies included abatacept, adalimumab, alemtuzumab, infliximab and IVIG...Concomitant maintenance therapy during avacopan treatment was rituximab in 12/15, methotrexate in 1/15, IVIG in 1/15, prednisolone alone in 1/15... Extended use of avacopan beyond 52 weeks was associated with further glucocorticoid reduction, improved disease control, and renal benefit in this complex set of ANCA vasculitis patients."
Clinical • Real-world • Real-world evidence • ANCA Vasculitis • Cardiovascular • Glomerulonephritis • Inflammation • Lupus Nephritis • Nephrology • Vasculitis
March 14, 2026
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
March 14, 2026
THE ROLE OF ALLOGENEIC HEMATOPOIETIC STEM CELL TRANSPLANTATION IN ADVANCED PRIMARY CUTANEOUS T-CELL LYMPHOMA - A MULTICENTER RETROSPECTIVE ANALYSIS
(EBMT 2026)
- "18 patients (64%) underwent myeloablative conditioning and in-vivo T-cell depletion was performed in 16 patients (57%; antithymocyte globulin: n=6; post-transplant cyclophosphamide: n=9; alemtuzumab: n=1). Although allo-HSCT can prolong RFS compared to conventional therapies, the durability of response is limited. Standardized transplant and post-transplant strategies to maintain disease remission are currently lacking and may be essential to fully exploit the curative potential of allo-HSCT in advanced CTCL."
Metastases • Retrospective data • Acute Graft versus Host Disease • Bone Marrow Transplantation • Chronic Graft versus Host Disease • Cutaneous T-cell Lymphoma • Graft versus Host Disease • Hematological Malignancies • Immunology • Infectious Disease • Lymphoma • Mycosis Fungoides • Non-Hodgkin’s Lymphoma • Sezary Syndrome • T Cell Non-Hodgkin Lymphoma • Transplantation
March 14, 2026
SUCCESSFUL CORD BLOOD TRANSPLANTATION IN AN INFANT WITH LEUKOCYTE ADHESION DEFICIENCY TYPE III: A CASE REPORT AND 1 YEAR FOLLOW-UP
(EBMT 2026)
- "The patient underwent a stem cell transplant from an unrelated CBU at 4 months old, following Treosulfan, Fludarabine, Thiotepa, and Alemtuzumab conditioning...Immunosuppression with cyclosporine was discontinued in September 2025.Donor chimerism progressively improved: 85% in T cells and 100% in B cells and PMN 4 months post transplant, rising to 98.3% T-cell donor chimerism by 6 months post HSCT... This case highlights the effectiveness of cord blood transplantation as a curative option for LAD-III in the absence of matched donors. The patient achieved timely engraftment, full donor chimerism, resolution of bleeding manifestations, and robust immune recovery with an excellent clinical outcome. We believe CBUs represent a viable and potentially lifesaving strategy for patients with ultra-rare immunodeficiencies such as LAD-III."
Case report • Clinical • Acute Graft versus Host Disease • Bone Marrow Transplantation • Genetic Disorders • Graft versus Host Disease • Hematological Disorders • Immunology • Infectious Disease • Primary Immunodeficiency • Thrombocytopenia • Transplantation • CD4 • FERMT3
March 14, 2026
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
March 14, 2026
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
March 14, 2026
HAEMATOPOIETIC STEM CELL TRANSPLANT (HSCT) IN NEUROPATHIC MUCOPOLYSACCHARIDOSIS TYPE-II (MPS-II) PRESERVES NEURODEVELOPMENT AND SHOULD BE ROUTINE PRACTICE
(EBMT 2026)
- "He was conditioned with Busulfan and Fludarabine following the standard European Guidelines...The same donor donated PBSC and conditioning was with Fludarabine, Thiotepa and Treosulfan. Alemtuzumab serotherapy, Cyclosporin-A and mycophenolate mofetil used as GVHD prophylaxis. Defibrotide was used for VOD prophylaxis in second transplant due to experiencing mild VOD during first transplant.Multisystem evaluation including neurodevelopmental assessments were recorded over 6–7 follow-up-years... In neuropathic MPS-II, definitive therapy with HSCT in early infancy preserves neurodevelopment and significantly reduces the multi-system disease burden compared with initiating ERT in later childhood. This sibling case-comparison supports the rationale for the need of newborn screening and prompt referral for HSCT. We have separately developed a gene therapy programme to further improve outcomes of cellular therapy in MPS-II and are reporting those outcomes at this meeting."
Bone Marrow Transplantation • CNS Disorders • Gene Therapies • Graft versus Host Disease • Hunter Syndrome • Hurler Syndrome • Immunology • Lysosomal Storage Diseases • Metabolic Disorders • Rare Diseases • Transplantation
March 14, 2026
RELIABLE ENGRAFTMENT AND COMPARABLE OUTCOMES ACROSS DONOR SOURCES WITH BUSULFAN65–FLUDARABINE–THIOTEPA CONDITIONING IN PAEDIATRIC SICKLE CELL DISEASE
(EBMT 2026)
- " We report on 35 symptomatic SCD patients aged 1-17 years (median 6) transplanted between 2014 and 2025 on an institutional protocol (including an established psychosocial support framework) with a uniform conditioning: busulfan tAUC 65ng*h/ml, fludarabine 150mg/m² and thiotepa 10mg/kg (plus cyclophosphamide 29mg/kg in MMFD)...All patients received serotherapy depending on donor source and HLA match: alemtuzumab 0.4mg/kg for MMFD, 0.8mg/kg for (M)MUD and ATG Grafalon® 45mg/kg for MSD/MFD... These results, obtained in a relatively young and otherwise healthy pediatric cohort treated with a Bu65–Flu–TT conditioning regimen, demonstrate reliable engraftment and similarly favorable outcomes across MSD/MFD, (M)MUD, and MMFD donors. These findings may pave the way for broader consideration of very early HSCT from alternative donors in children with SCD."
Acute Graft versus Host Disease • Bone Marrow Transplantation • Chronic Graft versus Host Disease • Chronic Kidney Disease • Genetic Disorders • Graft versus Host Disease • Hematological Disorders • Immunology • Infectious Disease • Nephrology • Novel Coronavirus Disease • Pediatrics • Pulmonary Disease • Respiratory Diseases • Sickle Cell Disease
March 14, 2026
FIRST REPORTED ALLOGENEIC HEMATOPOIETIC STEM CELL TRANSPLANTATION (ALLO-HSCT) FOR RAD50 DEFICIENCY: INITIAL CLINICAL EXPERIENCE IN TWO PEDIATRIC PATIENTS
(EBMT 2026)
- "Within one year, bone marrow dysplasia with a del(5q) clone and blasts up to 6–7% evolved, consistent with MDS on the background of inborn error of immunity.Unrelated-donor allo-HSCT (conditioning regimen: fludarabine 150 mg/m², busulfan 4 mg/kg, ATG 5 mg/kg and rituximab 375 mg/m², with PTCy-based GVHD prophylaxis) was complicated by HHV-6 infection, severe infusion-associated rash, and subsequent graft rejection by day +60. Six months later a second haploidentical HSCT from the mother (conditioning regimen: fludarabine 150 mg/m², treosulfan 21 g/m², rituximab 375 mg/m² and alemtuzumab 0.4 mg/kg with PTCy-based GVHD prophylaxis) resulted in timely engraftment and was complicated by megakaryocytic lineage hypofunction, viral reactivation, COVID-19 infection, and mild skin GVHD.Case #2...The course was marked by transfusion dependence, recurrent infections and refractory thrombocytopenia despite romiplostim and eltrombopag therapy.At the age of..."
Clinical • Acute Kidney Injury • Aplastic Anemia • Bone Marrow Transplantation • CNS Disorders • Graft versus Host Disease • Hematological Disorders • Herpes Simplex • Immunology • Infectious Disease • Metabolic Disorders • Nephrology • Novel Coronavirus Disease • Pediatrics • Thrombocytopenia • Transplant Rejection • Transplantation • RAD50
March 14, 2026
63 HLA-IDENTICAL RELATED HAEMOTOPOIETIC STEM CELL TRANSPLANTS IN SICKLE CELL DISEASE
(EBMT 2026)
- "Since 2015, hydroxyurea was initiated/intensified 1 month before to keep reticulocytes <100,000/mm³...Ovarian tissue is cryopreserved since 2015.Conditioning regimen:Until January 2015: Busulfan, cyclophosphamide, alemtuzumab. Graft versus host disease (GVHD) prophylaxis: cyclosporine A (CsA) and methotrexate.After January 2015: Thiotepa, Treosulfan, Fludarabine, anti-thymocyte globulin (myeloablative conditioning but reduced toxicity).GVHD prophylaxis: CsA, mycophenolate mofetil (MMF) (Jan 2015–Feb 2019); tacrolimus, MMF (Feb 2019–May 2024) or tacrolimus, MMF and post-transplant cyclophosphamide (PTCy) (January 2025–May 2025).Epidemiological, clinical, and analytical parameters were collected... The results of our study, the largest HSCT series in Spain, is similar to the international cohort and confirms the role of HSCT for children with SCD . Long-term follow-up remains essential to identify late complications."
Acute Graft versus Host Disease • Acute Kidney Injury • Bone Marrow Transplantation • Cardiovascular • Chronic Graft versus Host Disease • CNS Disorders • Epilepsy • Gene Therapies • Genetic Disorders • Graft versus Host Disease • Hematological Disorders • Hematological Malignancies • Hodgkin Lymphoma • Hypertension • Immunology • Lymphoma • Sickle Cell Disease • Subarachnoid Hemorrhage • Transplantation
March 14, 2026
A SINGLE CENTRE RETROSPECTIVE REVIEW OF DONOR LYMPHOCYTE INFUSION - INDICATIONS, OUTCOMES AND TOXICITY
(EBMT 2026)
- "83.1% of DLI was given in the context of Fludarabine/Melphalan/Alemtuzumab (FMC) conditioning with 45.7% of all FMC conditioned transplants requiring DLI.105 patients received pDLI. This study demonstrated high rates of DLI use particularly in the context of reduced intensity conditioning regimes. We provide further evidence of the of efficacy of DLI particularly in preventing disease relapse however GVHD remains a significant complication. Further strategies to reduce DLI use in future and to manage its toxicity are required."
Retrospective data • Review • Graft versus Host Disease • Immunology
March 14, 2026
NOVEL PRECISION ALEMTUZUMAB DOSING RESULTS IN BETTER DAY 0 TARGET RANGE ACHIEVEMENT AND IMPROVED HSCT OUTCOMES IN IEI PATIENTS
(EBMT 2026)
- P=N/A | "Our novel precision alemtuzumab dosing is feasible and results in target range achievement in >70% of patients (compared to historic experience of approximately 25% of patients treated with standard-of-care alemtuzumab dosing regimens). It appears to result in low rates of acute GVHD, clinically significant mixed chimerism, and secondary graft failure at day +100."
Clinical • Acute Graft versus Host Disease • Bone Marrow Transplantation • Graft versus Host Disease • Immunology • CD34
March 14, 2026
A SINGLE CENTRE EXPERIENCE OF ALLOGENEIC STEM CELL TRANSPLANT IN PATIENTS OVER 70
(EBMT 2026)
- "All received reduced-intensity conditioning (fludarabine/melphalan/alemtuzumab). AlloSCT in patients ≥70 years showed acceptable outcomes with good survival outcomes and zero transplant-related mortality. High rates of GVHD and infections highlight the need for improved post-transplant care, particularly in optimising GVHD prophylaxis/treatment and infection prevention."
Clinical • Acute Graft versus Host Disease • Chronic Graft versus Host Disease • Graft versus Host Disease • Hematological Disorders • Immunology • Infectious Disease • Transplantation
March 14, 2026
AGE AT TRANSPLANT DETERMINES OUTCOME FOLLOWING HEMATOPOIETIC STEM CELL TRANSPLANTATION FOR MITOCHONDRIAL NEURO-GASTROINTESTINAL ENCEPHALOPATHY (MNGIE)
(EBMT 2026)
- "Two patients received fludarabine, treosulfan, and thiotepa (FTT) whilst two received busulfan and fludarabine conditioning. All had alemtuzumab as part of the institutional protocol for non-malignant disease... Our unique experience highlights the critical importance of timing of HSCT in MNGIE in determining outcome. Those without disease manifestations do not develop them, but those with them do not improve. This persistent morbidity underscores the importance of considering MNGIE for inclusion in newborn screening programs to enable the earliest possible therapeutic intervention."
Bone Marrow Transplantation • Cardiovascular • CNS Disorders • Graft versus Host Disease • Immunology • Transplantation • TYMP
March 14, 2026
ALEMTUZUMAB PROVIDES DURABLE DEPLETION OF CIRCULATING CAR-T CELLS IN A PATIENT WITH HYPOKINETIC MOVEMENT DISORDERS AFTER BCMA-TARGETING CAR-T CELL THERAPY
(EBMT 2026)
- "Apart from cytokine release syndrome (CRS °II), which was successfully managed with tocilizumab and anakinra, the patient experienced no other adverse events in the first days following cilta-cel infusion...Based on previous reports, treatment attempts were made with cyclophosphamide, venetoclax, and ruxolitinib, complemented by intrathecal triple therapy after detection of CAR-T cells in the cerebrospinal fluid (CSF)...However, peak infiltration of the CSF occurred significantly later, on day 39 at a concentration of 157 CAR-T cells/µl, despite multiple cycles of intrathecal chemotherapy with dexamethasone, methotrexate and cytarabine...Flow cytometric analysis of potential immunotherapy targets revealed low expression of CD194 (mogalizumab) and CD30 (brentuximab vedotin) on circulating CAR-T cells, while strong expression of CD52 (alemtuzumab) was observed... Non-ICANS neurotoxicities (NINTs), including MNTs are associated with uncontrolled expansion of CAR-T..."
CAR T-Cell Therapy • Clinical • IO biomarker • CNS Disorders • Cognitive Disorders • Developmental Disorders • Hematological Malignancies • Movement Disorders • Multiple Myeloma • Parkinson's Disease • CCR4 • CD4 • CD52 • CD8 • TNFRSF8
March 14, 2026
HAPLOIDENTICAL VS MATCHED UNRELATED DONOR TRANSPLANTATION IN SEVERE APLASTIC ANEMIA: A SINGLE-CENTER COHORT
(EBMT 2026)
- "Among MUD recipients, 17 received fludarabine (Flu), cyclophosphamide (Cy), and total body irradiation (TBI) 2 cGy with anti-thymocyte globulin (ATG; 4.5 mg/kg), methotrexate (MTX), and cyclosporine (CsA), while six did not receive TBI. Of these, five received Flu/Cy/alemtuzumab and one received Flu/Cy/ATG with MTX/CsA... Haploidentical HCT using a PTCy platform is an effective and feasible alternative to MUD HCT in SAA, with comparable engraftment, GVHD, and survival outcomes."
Clinical • Acute Graft versus Host Disease • Anemia • Aplastic Anemia • Chronic Graft versus Host Disease • Graft versus Host Disease • Hematological Disorders • Immunology • Infectious Disease • Transplantation
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