Grafalon (rabbit anti-T-lymphocyte globulin) / Mundipharma, NeoPharm, Zydus Lifesci - LARVOL DELTA

PARVOVIRUS B19 WITH FEVER/RASH POST ALLO-HSCT: AML CASE REPORT (EHA 2025) - "A 66-year-old male patient, Acute myeloid leukemia in complete remission underwent allogeneic hematopoietic stem cell transplantation.The preconditioning regimen consisted of:TBI(6Gy)/Ara-C(0.5g/m²/d*4d)/DEC(20 mg/m²/d*3d)/FLU (30 mg/m²/d*5d)/Me-CCNU (250 mg/m²*1d)/ATG-F (5 mg/kg/d*4d), The process went smoothly overall.Successful engraftment of leukocytes and platelets was achieved...Dermatology considers pemphigus, Initial screening for herpes viruses in plasma and vesicular fluid secretions was negative(HHV1-8), After 1 week of treatment with application of acyclovir 0.5g q8h,methylprednisolone 8mg q12h,ruxolitinib 5mg q12h,there was no clinical improvement... For patients who have undergone allo-HSCT or are immunocompromised and present with fever accompanied by a rash and a progressive decline in hemoglobin levels, HPV B19 infection should be considered as a potential diagnosis. Plasma HPV B19 monitoring, histopathological examination (likely..."

Case report • Clinical • Acute Myelogenous Leukemia • Anemia • Bone Marrow Transplantation • Chronic Graft versus Host Disease • Dental Disorders • Dermatology • Dermatopathology • Graft versus Host Disease • Hematological Disorders • Hematological Malignancies • Immunology • Infectious Disease • Leukemia • Oncology • Xerostomia

A PROSPECTIVE COMPARATIVE STUDY OF ATLG VERSUS ANTI-T LYMPHOCYTE GLOBULIN AS GRAFT-VERSUS-HOST DISEASE PROPHYLAXIS IN UNRELATED ALLOGENEIC HEMATOPOIETIC CELL TRANSPLANTATION (EHA 2025) - "Data regarding the direct comparison of anti-T lymphocyte globulin (ATG) and Grafalon (ATLG) as GvHD prophylaxis in unrelated allo-HCT are limited...Six patients (12.5%) in the ATLG arm and 10 (22.2%) in ATG underwent post-infusion cyclophosphamide administration.The median follow-up period was 8 (4.5-15.5) months... In our analysis, the OS and DFS of these patients were higher in comparison to the rest of allo-HCT recipients. Thus, ATLG is shown as a safe and efficient agent for GvHD prophylaxis."

Clinical • Acute Myelogenous Leukemia • Graft versus Host Disease • Hematological Malignancies • Immunology • Leukemia • Oncology • Transplantation

ALLOGENEIC HEMATOPOIETIC STEM CELL TRANSPLANTATION FOR DIAMOND-BLACKFAN ANEMIA: FAVORABLE OUTCOMES FROM A SINGLE CENTER (EHA 2025) - "Myeloablative conditioning regimens included cyclophosphamide, fludarabine, thiotepa, busulfan, and ATG-F/ATG. Allo-HSCT is an effective curative therapy for DBA. In the absence of HLA-matched donors, haploidentical transplantation from non-carrier relatives is feasible. TCRαβ+ T-cell-depleted protocols significantly shorten engraftment time, reduce risks, and demonstrate excellent outcomes."

Clinical • Acute Graft versus Host Disease • Anemia • Aplastic Anemia • Bone Marrow Transplantation • Chronic Graft versus Host Disease • Genetic Disorders • Graft versus Host Disease • Hematological Disorders • Immunology • Infectious Disease • Oncology • Pediatrics • Rheumatology • Transplantation • RPL11 • RPL5 • RPS26

COMPARISON OF DIFFERENT DOSES OF RABBIT ANTI-THYMOCYTE GLOBULIN (ATG-T) AND ANTI-T-LYMPHOCYTE GLOBULIN (ATLG) IN TBI-BASED MYELOABLATIVE CONDITIONING FOR HAPLOIDENTICAL TRANSPLANTATION IN ACUTE LYMPHOBLASTIC LEUKEMIA PATIENTS (EHA 2025) - "Background: Rabbit anti-thymocyte globulin (ATG-T, Sanofi) and anti-T-lymphocyte globulin (ATLG, Grafalon) are widely used for graft-versus-host disease (GVHD) prophylaxis in haploidentical hematopoietic stem cell transplantation (HID-HSCT). In the context of TBI-based MAC for ALL patients undergoing HID-HSCT,Although the higher ATG-T dose was linked to increased CMV/EBV viremia, PTLD, and GVHD compared to ATLG, its overall post-transplant efficacy appears optimal—likely due to the elevated NRM observed with the lower ATG-T dose and the higher CIR associated with ATLG. These findings offer valuable guidance for optimizing ATG formulation and dosing for GVHD prophylaxis in HID-HSCT, supporting more personalized treatment strategies for ALL patients."

Clinical • Preclinical • Acute Graft versus Host Disease • Acute Lymphocytic Leukemia • Bone Marrow Transplantation • Chronic Graft versus Host Disease • Graft versus Host Disease • Hematological Malignancies • Immunology • Infectious Disease • Leukemia • Oncology • Transplantation

COMPARISON OF THYMOGLOBULIN® VS GRAFALON® AS INDUCTION TREATMENT IN KIDNEY TRANSPLANTATION WITH MAASTRICHT TYPE III DONOR (ERA 2025) - "The two most commonly used drugs are Thymoglobulin® and Grafalon®, both rabbit antithymocyte globulins, although they have different antigenic profiles and antibody concentrations. These results suggest that Grafalon® and Thymoglobulin® are comparable in terms of efficacy and safety, with a more favourable haematological profile for Grafalon® in low- immunological-risk KT recipients. However, further studies are needed to confirm these findings."

Cardiovascular • Cytomegalovirus Infection • Infectious Disease • Transplant Rejection • Transplantation

Can ALC be used to optimise ATLG dose inkidney transplant patients ? (ERA 2025) - " The present is a prospective observational study conducted in kidney transplant department of tertiary care centre .As per protocol,40 KTR of intermediate risk group were given dynamic ATLG-Grafalon dosing as induction agent along with standard triple immunosuppressive therapy.Patients with high and low immunological risk were excluded.As per dynamic dosing,the KTR received first dose (3 mg/kg) on the day of transplant .Subsequent doses were determined according to ALC.The ATLG was targeted to keep the mean ALC< 500.The patients were followed up for infections and incidences of acute rejection till 3 months.A comparison of graft outcome, rejections and infections between 40 KTR receiving dynamic Grafalon dosing was done with historical data of 27 KTR who received standard Grafalon dosing (8 mg/kg)... The present study suggests that ALC can be used as a simple bedside tool in optimising the dosage of induction agent in KTR. Reduced dosage of ATLG decreases the risk of..."

Clinical • Infectious Disease • Transplant Rejection • Transplantation

Total Body Irradiation and Astatine-211-Labeled BC8-B10 Monoclonal Antibody for the Treatment of Nonmalignant Diseases (clinicaltrials.gov) - P1/2 | N=40 | Recruiting | Sponsor: Fred Hutchinson Cancer Center | Suspended ➔ Recruiting

Enrollment open • Oncology • HLA-A • HLA-B • HLA-C • HLA-DQB1 • HLA-DRB1

Mismatched Related Donor Versus Matched Unrelated Donor Stem Cell Transplantation for Children, Adolescents, and Young Adults With Acute Leukemia or Myelodysplastic Syndrome (clinicaltrials.gov) - P3 | N=435 | Active, not recruiting | Sponsor: Children's Oncology Group | Recruiting ➔ Active, not recruiting | Trial completion date: Dec 2027 ➔ Jun 2026 | Trial primary completion date: Dec 2027 ➔ Jun 2026

Enrollment closed • Trial completion date • Trial primary completion date • Acute Lymphocytic Leukemia • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Myelodysplastic Syndrome • Oncology • Transplantation • ABL1 • BCR • CEBPA • FLT3 • HLA-B • HLA-C • HLA-DQB1 • HLA-DRB1 • NPM1

Hematopoietic Stem Cell Transplantation in Severe Pediatric Sickle Cell Disease: Outcome and long-term complications, Saudi experience at King Faisal Specialist Hospital, Riyadh, Saudi Arabia. (PubMed, Mediterr J Hematol Infect Dis) - "All patients received myeloablative conditioning with Busulfan, Cyclophosphamide, and Anti T-Lymphocyte Globulin (Grafalon®): BU/CY/ATG in 114 (88.4%), BU/CY in 13 (10%) and other in 2 (2%). HSCT for children with sickle cell disease from fully matched siblings offers the best outcome using myeloablative conditioning. However, significant toxicities were observed secondary to myeloablative regimens, in particular long-term complications, which demands exploring the use of less toxic regimens."

Journal • Acute Graft versus Host Disease • Bone Marrow Transplantation • Chronic Graft versus Host Disease • Genetic Disorders • Graft versus Host Disease • Hematological Disorders • Immunology • Pediatrics • Sickle Cell Disease • Transplantation

THE SAFETY AND EFFICACY OF LIPOSOMAL MITOXANTRONE (LIPO-MIT) COMBINED WITH TBI-BASED CONDITIONING FOR ALLO-HSCT IN RELAPSED/REFRACTORY T-LBL AND T-ALL (EBMT 2025) - "The conditioning regimen consisted of Lipo-MIT 20 mg/m²/day from days -11 to -10, Ara-C 3 g/m²/day from days -11 to -9, TBI 2 Gy twice daily from days -8 to -6, cyclophosphamide (CY) 1.2–1.8 g/m²/day from days -5 to -4 (or fludarabine [FLU] 30 mg/m²/day from days -5 to -2), and MeCCNU 250 mg/m²/day on day -3. Antithymocyte globulin (ATG-F) and cyclosporine A (CSA)/mycophenolate mofetil (MMF)/methotrexate (MTX) were used for acute graft-versus-host disease (aGVHD) prophylaxis... Liposomal mitoxantrone (Lipo-MIT) combined with TBI-based conditioning regimen for allo-HSCT is a safe and effective approach that improves survival and reduces the risk of relapse in patients with R/R T-ALL and T-LBL."

Clinical • Acute Graft versus Host Disease • Bone Marrow Transplantation • Chronic Graft versus Host Disease • Graft versus Host Disease • Immunology • Infectious Disease • Lymphoma • T Acute Lymphoblastic Leukemia • T-cell Acute Lymphoblastic Lymphoma • CD7

IMMUNE DYSREGULATION AND AUTOIMMUNITY POST-HSCT IN RAG1-SCID: A CHALLENGING CASE REPORT (EBMT 2025) - "Genetic testing confirmed RAG1 deficiency, and viral screening identified asymptomatic CMV infection.The patient was initially treated with methylprednisolone and cyclosporine A for immune dysregulation and started infectious prophylaxis and CMV treatment. At 6 months of age, he underwent haploidentical HSCT from his father (Treosulfan, Fludarabine, Rituximab, and ATG Grafalon, with ex-vivo TCR αβ+/CD19+ cell depletion)...This improved following exclusive naso-jejunal feeding and he came off supplementary oxygen 15 month post HSCT.At 8 months, he developed hemorrhagic CMV retinitis, treated with intravitreal foscarnet and systemic foscarnet and ganciclovir, with resolution after one month...Given the difficulty in achieving adequate sirolimus levels, abatacept was introduced as a bridge therapy for 6 months... This case highlights the complex autoimmune complications that can arise after HSCT for RAG1 SCID, including AHIA, hypothyroidism, and glomerulosclerosis. The..."

Case report • Clinical • Alopecia • Anemia • Autoimmune Hemolytic Anemia • Bone Marrow Transplantation • Chronic Kidney Disease • Cytomegalovirus Infection • Dermatology • Endocrine Disorders • Focal Segmental Glomerulosclerosis • Gastroenterology • Gastroesophageal Reflux Disease • Genetic Disorders • Glomerulonephritis • Graft versus Host Disease • Hematological Disorders • Immunology • Infectious Disease • Neutropenia • Ocular Inflammation • Ophthalmology • Primary Immunodeficiency • Pulmonary Disease • Respiratory Diseases • Retinal Disorders • RAG1

COMPARISON OF DIFFERENT ANTI-HUMAN T CELL GLOBULINS ON IMMUNE RECONSTITUTION AND EARLY INFECTIONS AFTER CYCLOPHOSPHAMIDE-BASED CONDITIONING FOLLOWED BY AUTOLOGOUS TRANSPLANT IN PATIENTS WITH MULTIPLE SCLEROSIS (EBMT 2025) - "It is usually augmented by in vivo T-cell depletion using polyclonal anti-thymocyte globulin (ATG: Thymoglobulin; Genzyme-Sanofi) or anti-T lymphocyte globulin (ATLG: Grafalon, Neovii). Both are derived by vaccination of rabbits with human thymocytes (ATG) or the Jurkat T cell line (ATLG)...Virus reactivations were solely observed in the ATG group: CMV reactivation was detected in 5 patients, of whom 4 received outpatient treatment with Valaciclovir... Patients receiving ATLG demonstrated significantly higher numbers of B and T cells on day +30 compared to patients receiving ATG. We observed significantly lower numbers of moderate to mild infectious complications in the ATLG group. No severe infectious complications or death was noted."

Clinical • Bone Marrow Transplantation • CNS Disorders • Infectious Disease • Inflammation • Multiple Sclerosis • Respiratory Diseases • Transplantation • CD27 • CD8 • IL2RA • IL7R • ISG20 • MME

SAFETY AND EFFICACY OF FLUDARABINE PLUS MYELOABLATIVE DOSE OF TREOSULFAN (FT14) CONDITIONING REGIMEN FOR AML – INTERIM ANALYSIS FROM THE FT14 STUDY GROUP (EBMT 2025) - "While busulfan-based regimens such as fludarabine-busulfan (FB4) are widely regarded as the gold standard for reduced-toxicity myeloablative conditioning, they are associated with risks such as Veno-Occlusive Disease (VOD), pulmonary toxicity, and high Transplant-Related Mortality (TRM)...Graft sources included peripheral blood stem cells or bone marrow, with Graft-versus-Host Disease (GvHD) prophylaxis using ATG (Thymoglobulin or Grafalon), cyclosporine and short course methotrexate...On viral point of view, 6% experienced CMV reactivation despite letermovir prophylaxis, 11% had detectable EBV viraemia (with no case of post-transplant lymphoproliferative disease), 4% had SARS-CoV-2 and 2% HHV6 reactivation... FT14 is a safe and effective myeloablative conditioning regimen for AML, demonstrating excellent disease control with minimal toxicity. Its reduced transplant-related mortality(TRM) and GvHD rates make it particularly beneficial for patients at high risk of..."

Clinical • Acute Graft versus Host Disease • Acute Myelogenous Leukemia • Bone Marrow Transplantation • Chronic Graft versus Host Disease • Graft versus Host Disease • Hepatology • Immunology • Infectious Disease • Novel Coronavirus Disease • Respiratory Diseases

A SINGLE CENTRE RETROSPECTIVE ANALYSIS OF TREATMENT OUTCOMES IN APLASTIC ANAEMIA AT APOLLO HOSPITAL, HYDERABAD, INDIA (EBMT 2025) - "Treatments evaluated were horse ATG, BMT, and oral therapy with cyclosporine and thrombopoietin agonists. Fully matched sibling transplants for AA show excellent outcomes. Sibling donor transplants and h-ATG therapy are crucial for favorable results. rATLG (Grafalon) has minimal side effects and low GVHD incidence."

Retrospective data • Anemia • Aplastic Anemia • Bone Marrow Transplantation • Chronic Graft versus Host Disease • Graft versus Host Disease • Hematological Disorders • Immunology • Infectious Disease • Pneumonia • Respiratory Diseases • Septic Shock

ENHANCED GVHD PROPHYLAXIS WITH LOW-DOSE GRAFALON® AND POST-TRANSPLANT CYCLOPHOSPHAMIDE IN HAPLOIDENTICAL AND MISMATCHED UNRELATED DONOR STEM CELL TRANSPLANTATION (EBMT 2025) - "The combination of low-dose Grafalon® and PTCy appears to be a safe and effective GvHD prophylaxis strategy, demonstrating excellent tolerability with no severe acute or chronic GvHD and without increasing the risks of infection or relapse. Larger prospective studies and longer follow-up are needed to confirm these findings and establish the long-term efficacy of this approach."

Post-transplantation • Acute Graft versus Host Disease • Acute Myelogenous Leukemia • Bone Marrow Transplantation • Chronic Graft versus Host Disease • Chronic Myeloid Leukemia • Graft versus Host Disease • Hematological Malignancies • Immunology • Infectious Disease • Leukemia • Lymphoma • Non-Hodgkin’s Lymphoma • Oncology • Transplantation • CD4

SALVAGE TREATMENT OF PROGRESSIVE STEROID-REFRACTORY GRAFT-VERSUS-HOST DISEASE BY IMMUNOABLATIVE THERAPY WITHOUT STEM CELL SUPPORT IN PEDIATRIC PATIENTS (EBMT 2025) - " IAT comprises the administration of antithymocyte globulin Grafalon® (4 x 15 mg/kg), cyclophosphamide (2 x 60 mg/kg) and fludarabine (4 x 40 mg/m²) and leads to a reset of the immune network...Nevertheless, the patient achieved stable reconstitution.Table 1: Patient characteristicsADV = adenovirus, ALL = acute lymphoblastic leukemia, ATG = antithymocyte immunoglobulin, BKV = BK virus, CMV = cytomegalovirus, CNS = central nervous system, CSA = cyclosporine A, EBV = Epstein-Barr virus, ECP = extracorporeal photopheresis, Flu = fludarabine, HHV6 = human herpesvirus 6, JCV = JC virus, MMF = mycophenolate mofetil, MPS I = mucopolysaccharidosis type I, MSC = mesenchymal stromal cells, MSD = matched sibling donor, MTX = methotrexate, MUD = matched unrelated donor, TBI = total body irradiation, Thio = thiotepa, Treo = treosulfan, VP-16 = etoposide The patients in this case series with life-threatening SR-GvHD showed CR after IAT while ≥4 lines of immunosuppressive..."

Clinical • Acute Lymphocytic Leukemia • Chronic Graft versus Host Disease • Cytomegalovirus Infection • Epstein-Barr Virus Infections • Graft versus Host Disease • Hematological Disorders • Hematological Malignancies • Hurler Syndrome • Immunology • Leukemia • Oncology • Pediatrics • Scleroderma • Systemic Sclerosis • CD34

GRAFT VERSUS HOST DISEASE PROPHYLAXIS WITH ATG OR PTCY AFTER ALLOGENEIC TRANSPLANTATION FROM VOLUNTEER UNRELATED DONORS. A SINGLE CENTER RETROSPECTIVE PSEUDO-RANDOMIZED ANALYSIS (EBMT 2025) - "In our center we routinely used low dose alemtuzumab (Campath), however, since this drug is not provided any more by the manufacturer, we changed our GVHD prevention strategy to Anti T-lymphocyte globulin (Grafalon). As current data suggest that post-transplantation cyclophosphamide (PTCY) could be an efficient GVHD prophylactic strategy also in 10/10 well matched UD (WMUD) and 9/10 mismatched UD (MMUD) allo-HCT, after we treated 7 patients with ATG we decided to start to pseudo-randomize consecutive patients in receiving ATG or PTCY according to their unique patient number...Cyclosporine was administered at a dose of 5 mg/kg/day from day -3, in both groups, with doses adjusted to maintain a through level of 200-400 ng/ml. Mycophenolate mofetil (MMF) at a dosage of 1000 mg bd was added, in both groups, from day +1 until d+60, when allo-HCT was performed from a MMUD... We conclude that both ATG and PTCY are effective in preventing GVHD in UD allo-HCT. With the limited..."

Retrospective data • Acute Graft versus Host Disease • Chronic Graft versus Host Disease • Graft versus Host Disease • Hematological Disorders • Hematological Malignancies • Immunology • Leukemia • Oncology • Transplantation

SINGLE CENTRE OBSERVATIONAL ANALYSIS OF GVHD PROPHYLAXIS WITH ANTI HUMAN T LYMPHOCYTE IMMUNOGLOBULIN (RATLG) IN THALASSEMIA MAJOR PATIENTS UNDERGOING STEM CELL TRANSPLANTATION (EBMT 2025) - "The use of Grafalon (rATLG) in the conditioning regimen for HSCT at KD Ambani Hospital showed a low incidence of acute GVHD (3 out of 34 patients) and no chronic GVHD. The majority of patients survived, indicating a favourable outcome with this treatment protocol. Clinical Trial Registry: NA"

Clinical • Acute Graft versus Host Disease • Beta-Thalassemia • Bone Marrow Transplantation • Chronic Graft versus Host Disease • Genetic Disorders • Graft versus Host Disease • Immunology • Transplantation

SINGLE CENTRE OBSERVATIONAL ANALYSIS OF GVHD PROPHYLAXIS WITH ANTI HUMAN T LYMPHOCYTE IMMUNOGLOBULIN (RATLG) IN THALASSEMIA MAJOR PATIENTS UNDERGOING STEM CELL TRANSPLANTATION (EBMT 2025) - "The use of Grafalon (rATLG) in the conditioning regimen for HSCT at KD Ambani Hospital showed a low incidence of acute GVHD (3 out of 34 patients) and no chronic GVHD. The majority of patients survived, indicating a favourable outcome with this treatment protocol. Clinical Trial Registry: NA"

Clinical • Acute Graft versus Host Disease • Beta-Thalassemia • Bone Marrow Transplantation • Chronic Graft versus Host Disease • Genetic Disorders • Graft versus Host Disease • Immunology • Transplantation

COMPARATIVE SINGLE CENTRE OBSERVATIONAL ANALYSIS OF GVHD PROPHYLAXIS WITH GRAFALON (RATLG) AND THYMOGLOBULIN (RATG) IN THALASSEMIA MAJOR PATIENTS UNDERGOING STEM CELL TRANSPLANTATION (EBMT 2025) - "The use of Grafalon (rATLG) in the conditioning regimen for HSCT showed a lower incidence of acute and chronic GVHD compared to Thymoglobulin. The majority of patients in the Grafalon group survived with satisfactory immune reconstitution, indicating a more favourable outcome with this treatment protocol. Clinical Trial Registry: NA"

Clinical • Acute Graft versus Host Disease • Beta-Thalassemia • Bone Marrow Transplantation • Chronic Graft versus Host Disease • Genetic Disorders • Graft versus Host Disease • Immunology • Infectious Disease • Transplantation

COMPARATIVE SINGLE CENTRE OBSERVATIONAL ANALYSIS OF GVHD PROPHYLAXIS WITH GRAFALON (RATLG) AND THYMOGLOBULIN (RATG) IN THALASSEMIA MAJOR PATIENTS UNDERGOING STEM CELL TRANSPLANTATION (EBMT 2025) - "The use of Grafalon (rATLG) in the conditioning regimen for HSCT showed a lower incidence of acute and chronic GVHD compared to Thymoglobulin. The majority of patients in the Grafalon group survived with satisfactory immune reconstitution, indicating a more favourable outcome with this treatment protocol. Clinical Trial Registry: NA"

Clinical • Acute Graft versus Host Disease • Beta-Thalassemia • Bone Marrow Transplantation • Chronic Graft versus Host Disease • Genetic Disorders • Graft versus Host Disease • Immunology • Infectious Disease • Transplantation

EARLY LOW-DOSE ATG CONDITIONING FOR SINGLE UNIT CORD BLOOD TRANSPLANT IN HIGH-RISK MALIGNANCIES: GOOD ENGRAFTMENT, REDUCED GVHD, AND LOW NRM (EBMT 2025) - "In 88% of patients the conditioning regimen was Thiotepa, Busulfan, and Fludarabine-based (TBF), with the remaining receiving total body irradiation (TBI). A total low dose (15 mg/kg) of ATG (Grafalon, Neovii) was given from day -6 to -2. GvHD prophylaxis included cyclosporine (CSA) and Mycophenolate Mofetil (MMF), which was given until day +35... This study shows that single-unit CBT with an early, low-dose ATG-based conditioning regimen achieves high engraftment rates and rapid leukocyte recovery. Despite the very high-risk clinical profiles of these patients, the incidence of GvHD was low and NRM remained acceptable. These findings show that CBT still represents an effective option for high-risk patients lacking suitable donors."

Acute Graft versus Host Disease • Bone Marrow Transplantation • Chronic Graft versus Host Disease • Graft versus Host Disease • Hematological Disorders • Hematological Malignancies • Immunology • Infectious Disease • Leukemia • Oncology • Transplantation

EARLY LOW-DOSE ATG CONDITIONING FOR SINGLE UNIT CORD BLOOD TRANSPLANT IN HIGH-RISK MALIGNANCIES: GOOD ENGRAFTMENT, REDUCED GVHD, AND LOW NRM (EBMT 2025) - "In 88% of patients the conditioning regimen was Thiotepa, Busulfan, and Fludarabine-based (TBF), with the remaining receiving total body irradiation (TBI). A total low dose (15 mg/kg) of ATG (Grafalon, Neovii) was given from day -6 to -2. GvHD prophylaxis included cyclosporine (CSA) and Mycophenolate Mofetil (MMF), which was given until day +35... This study shows that single-unit CBT with an early, low-dose ATG-based conditioning regimen achieves high engraftment rates and rapid leukocyte recovery. Despite the very high-risk clinical profiles of these patients, the incidence of GvHD was low and NRM remained acceptable. These findings show that CBT still represents an effective option for high-risk patients lacking suitable donors."

Acute Graft versus Host Disease • Bone Marrow Transplantation • Chronic Graft versus Host Disease • Graft versus Host Disease • Hematological Disorders • Hematological Malignancies • Immunology • Infectious Disease • Leukemia • Oncology • Transplantation

DONOR CD45 RA DEPLETED CELLS FOR THE TREATMENT OF SEVERE CRYPTOSPORIDIOSIS FOLLOWING HAPLOIDENTICAL HSCT (EBMT 2025) - "Both were homozygous for c.5134 C>A mutations in DOCK8, reported to be a founder mutation in the Bedouin population of southern Israel.Pre-transplant conditioning included Fludarabine (160 mg/m2), Treosulfan (36 g/m2), Thiotepa (10mg/kg), anti-thymocyte globulin (ATG-Grafalon) (12mg/kg), and Rituximab (200mg/m2)...Both patients had profound weight loss and required parenteral nutrition; Neither responded to conventional anti-cryptosporidial therapy including Nitazoxanide, Paromomycin and Azithromycin... Donor CD45RA-depleted cells represent a promising therapeutic strategy for severe cryptosporidiosis in patients following haploidentical HSCT prior to T cell recovery. Use of CD45RA depletion obviates the need for exposure of donor cells to specific stimulatory peptides, and demonstrates promise in the treatment if unusual infections in this at-risk population."

Atopic Dermatitis • Bone Marrow Transplantation • Dermatitis • Dermatology • Human Immunodeficiency Virus • Immunology • Infectious Disease • Primary Immunodeficiency • DOCK8 • PTPRC

TREOSULFAN, THIOTEPA AND FLUDARABIN REPRESENTS A SAFE AND EFFICIENT ALTERNATIVE CONDITIONING IN T CELL DEPLETED HAPLOIDENTICAL AND MSD HSCT FOR PATIENTS WITH TDT (EBMT 2025) - "All patients received treosulfan, thiotepa, fludarabine (FTT) and ATG-Grafalon®, administered before start of conditioning in T-haplo-HSCT and prior to day 0 in MSD-BMT. Immunosuppression (IST) consisted in all but two cases of tacrolimus (or cyclosporine) and mycophenolate mofetil...Most importantly, only one case of suspected veno-occlusive disease (VOD) in a MSD-BMT patient resolved completely after treatment with defibrotide and a single case of IST-associated PRES was observed in the T-haplo group. These first, retrospective data on the safety and efficacy of T-haplo-HSCT support early transplantation in TDT patients without MD. Furthermore, with only one case of VOD (4%), treosulfan has proven to be an excellent alternative to busulfan in this high-risk patient population, where the incidence of VOD can be as high as 21%, even with pharmacokinetic-adjusted busulfan dosing."

Clinical • Acute Graft versus Host Disease • Bone Marrow Transplantation • Chronic Graft versus Host Disease • Genetic Disorders • Graft versus Host Disease • Hematological Disorders • Hepatology • Immunology • Neutropenia • Transplant Rejection • CD34