Atgam (equine anti-thymocyte globulin) / Pfizer - LARVOL DELTA

A novel depleting anti-CD2 monoclonal antibody for induction of mixed chimerism and allograft tolerance in nonhuman primates (TTS 2026) - "However, substitution with rabbit anti-thymocyte globulin (Thymoglobulin) resulted in inferior outcomes, including donor-specific antibody (DSA) development and chronic rejection...Animals then underwent combined kidney and bone marrow transplantation (CKBMT) and received anti-CD154 (2–4 doses of 20 mg/kg during the first two post-transplant weeks) and a 28-day course of cyclosporine, after which immunosuppression was withdrawn... This novel anti-CD2 mAb induces higher and more durable myeloid and lymphoid chimerism than Thymoglobulin and achieves chimerism comparable to ATGAM. These effects appear to be associated with prolonged depletion of CD8 TEMs. While long-term graft outcomes are still under evaluation, the superior chimerism induction observed with anti-CD2 suggests the potential for improved transplant outcomes compared with Thymoglobulin."

Bone Marrow Transplantation • Immune Modulation • Immunology • Transplant Rejection • CD4 • CD40LG • CD8

TRIUMPH: TReatment for ImmUne Mediated PathopHysiology (clinicaltrials.gov) - P2 | N=44 | Active, not recruiting | Sponsor: National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) | N=163 ➔ 44

Enrollment change • CNS Disorders • Hepatic Encephalopathy • Hepatology • Liver Failure • Pediatrics

TRIUMPH: TReatment for ImmUne Mediated PathopHysiology (clinicaltrials.gov) - P2 | N=163 | Active, not recruiting | Sponsor: National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) | Recruiting ➔ Active, not recruiting

Enrollment closed • CNS Disorders • Hepatic Encephalopathy • Hepatology • Liver Failure • Pediatrics

Managing Immunological High Risk Kidney Transplant Patients In Nigeria, A Microcosm Of Resource-Constrained Setting; Evidence And Practice. (PubMed, Niger Med J) - "There is no change in the choice of immunosuppressive agent for kidney transplant in Nigeria and other centers and guidelines reviewed. With ongoing efforts to reduce the widening gap in transplant care by having an individualized approach to care, this narrative explores current practices in a resource-constrained setting as compared to guideline recommendations and provides a basis for future studies."

Journal • Review • Transplantation

Methylprednisolone, Horse Anti-Thymocyte Globulin, Cyclosporine, Filgrastim, and/or Pegfilgrastim or Pegfilgrastim Biosimilar in Treating Patients With Aplastic Anemia or Low or Intermediate-Risk Myelodysplastic Syndrome (clinicaltrials.gov) - P2 | N=140 | Recruiting | Sponsor: M.D. Anderson Cancer Center | Trial completion date: Jun 2026 ➔ Jun 2029 | Trial primary completion date: Jun 2026 ➔ Jun 2029

Trial completion date • Trial primary completion date • Anemia • Aplastic Anemia • Hematological Disorders • Hematological Malignancies • Myelodysplastic Syndrome • Neutropenia • Oncology

Differential Expression in Kidney Allograft Biopsies Between Non-Human Primates Conditioned with Horse versus Rabbit Anti-Thymocyte Globulin in a Mixed Chimerism-Based Tolerance Induction Protocol (ATC 2026) - "*Purpose: We have previously shown than the use of rabbit anti-thymocyte globulin (rATG; Thymoglobulin) has resulted in inferior mixed chimerism induction and kidney allograft tolerance in non-human primates versus horse anti-thymocyte globulin (hATG; ATGAM). Differential expression analysis using the B-HOT panel revealed differences in the immune microenvironment within kidney allograft biopsies in non-human primates conditioned with ATGAM or Thymoglobulin at 3 months post-transplant. These differences suggest variation in the immunomodulation achieved by the two ATG formulations that may impact long-term allograft outcomes."

Biopsy • Clinical • Preclinical • Bone Marrow Transplantation • Fibrosis • Immunology • Solid Organ Transplantation • ARG2 • CD163 • CXCL16 • STAT6

SHORT- AND LONG-TERM OUTCOMES OF EQUINE ANTITHYMOCYTE GLOBULIN-BASED DOUBLE AND TRIPLE IMMUNOSUPPRESSIVE THERAPY IN APLASTIC ANEMIA: A SYSTEMATIC REVIEW (EHA 2026) - "Patients ineligible for HSCT are treated with IST, equine antithymocyte globulin (eATG) and cyclosporine. Eltrombopag was initially introduced as salvage therapy in refractory AA and was evaluated in combination with eATG ‑ based IST, leading to its incorporation in upfront triple therapy (3T) in recent studies...Inconsistent long ‑ term outcome reporting, with many studies providing only one timepoint, limits longitudinal assessment and adds heterogeneity. More complete, longitudinal reporting by therapy and clinically relevant subgroups is needed to enable robust comparative assessment and inform optimal treatment strategies in AA."

Review • Anemia • Aplastic Anemia • Bone Marrow Transplantation • Hematological Disorders

Case Report: Successful treatment of steroid-refractory severe immunotherapy-induced pneumonitis with equine antithymocyte globulin. (PubMed, Front Oncol) - "Current management of IIP involves corticosteroids, mycophenolate mofetil (MMF), intravenous immunoglobulin, or infliximab for severe cases...A 50-year-old woman with recurrent unresectable melanoma in the right ankle developed grade 4 IIP after receiving two cycles of ipilimumab and nivolumab...At 12 months, the patient remained well from a respiratory standpoint, with no recurrence of melanoma. This case highlights the potential for eATG, with therapeutic CD2+/CD3+ T lymphocyte count monitoring, to address an unmet therapeutic need in patients with refractory ICI-induced IIP."

Journal • Cardiovascular • Immunology • Inflammation • Melanoma • Oncology • Pneumonia • Solid Tumor

Long-Term Clinical and Molecular Dynamics in Hypoplastic Myelodysplastic Neoplasia Treated With Immunosuppressive Therapy. (PubMed, Eur J Haematol) - "These findings suggest that in MDS-h, disease activity is largely driven by immune dysregulation rather than early molecular changes, and that repeated IST can yield sustained remissions. However, accumulating mutations may eventually predict malignant transformation, underscoring the importance of long-term molecular monitoring."

Journal • Acute Myelogenous Leukemia • Aplastic Anemia • Hematological Disorders • Hematological Malignancies • Leukemia • Myelodysplastic Syndrome • Oncology

OUTCOME OF UNRELATED AND HAPLOIDENTICAL HSCT AMONG CHILDREN WITH MYELODYSPLASTIC SYNDROME (EBMT 2026) - "16 patients were treated using the αβ T-cell depletion platform, 17 patients were treated using post-transplant cyclophosphamide (PTCy), 3 – with conventional CNI-based GVHD prophylaxis (group characteristics, table 1).All patients had cytopenia: transfusion dependence, leukopenia with neutropenia moderate to severe, 5 patients had progression to acute myeloid leukemia (AML).17 cases had germline condition predisposing to MDS: 2 - Shwachman-Diamond syndrome (SDS), 2 - Severe Congenital Neutropenia, 2 unspecified primary immune deficiency, 1 patient due to Diamond-Blackfan anemia, 2 patients had Down syndrome, 1 patient had Noonan syndrome...5 patients with AML received various courses of hypomethylating agents.Preparative regimen included treosulfan 42 gr/m2, fludarabine 150 mg/kg, thiotepa 10 mg/kg or melphalan 140 mg/m2. Thymoglobulin 5mg/kg or ATGAM 100 mg/kg and rituximab 200mg/m2 were used in a proportion of cases.Post-transplant GVHD prophylaxis included for αβ..."

Clinical • Acute Myelogenous Leukemia • Bone Marrow Transplantation • Developmental Disorders • Genetic Disorders • Graft versus Host Disease • Hematological Disorders • Hematological Malignancies • Immunology • Infectious Disease • Leukemia • Leukopenia • Myelodysplastic Syndrome • Neutropenia • Primary Immunodeficiency • GATA1 • GATA2

OUTCOMES OF ALLOGENEIC HSCT IN NIJMEGEN BREAKAGE SYNDROME WITH TREOSULFAN-BASED CONDITIONING REGIMEN - A MULTICENTER STUDY (EBMT 2026) - "We recently demonstrated experience of treosulfan 30g/m2 versus low doses of busulfan...All received fludarabine 150mg/m2, 24 cyclophosphamide (22–40mg/kg, 1–30mg/kg, 1–20mg/kg). In 37 patients, rabbit ATG (thymoglobulin, Genzyme) 3-7,5mg/kg, in 1 patient horse ATG (ATGAM) 100mg/kg, and in 2 patients no serotherapy was used.In 25 patients matched unrelated (19–10/10, 6–9/10 HLA-matched), in 9 mismatched related, in 6 matched sibling donor was used... Both doses of treosulfan 21g/m2 and 30g/m2 were well tolerated by NBS patients. However, lower dose of 21g/m2 was associated with higher risk of mixed chimerism and leukemia relapse versus increased incidence of secondary malignancy after higher dose of 30g/m2."

Clinical • Acute Graft versus Host Disease • Bone Marrow Transplantation • Chronic Graft versus Host Disease • Graft versus Host Disease • Hematological Malignancies • Immunology • Infectious Disease • Leukemia • Lymphoma • Metabolic Disorders • Primary Immunodeficiency • Rhabdomyosarcoma • Sarcoma • Solid Tumor • Transplant Rejection

A SYSTEMATIC REVIEW OF REAL-WORLD EVIDENCE ON THERAPIES FOR MODERATE APLASTIC ANAEMIA (EBMT 2026) - "Treatment choices depend on blood counts, transfusion dependency or other significant symptoms and include supportive care, monotherapy (cyclosporine [CSA], tacrolimus, daclizumab, thrombopoietin receptor agonists [TPO-RA], androgens), double or triple therapy, (combining horse or rabbit anti-thymocyte globulin [hATG or rATG], CSA/tacrolimus, and eltrombopag [EPAG]/TPO-RA or androgens), and/or haematopoietic stem-cell transplantation (HSCT)... Across heterogenous studies, OS and ORR have been documented for monotherapies, double, and triple therapies; HSCT was only reported in small, heterogeneous cohorts. Due to varied timepoints, missing data, and cohort selection, there was insufficient homogenous data for quantitative analysis. This SLR demonstrates a clear need for uniform definition of mAA and for comparative, prospective studies to enable the comparison of therapeutic effectiveness and to develop sufficient treatment algorithms."

Clinical • HEOR • Real-world • Real-world evidence • Review • Anemia • Aplastic Anemia • Bone Marrow Transplantation • Hematological Disorders

AUTOLOGOUS UMBILICAL CORD BLOOD TRANSPLANTATION WITHOUT CHEMOTHERAPY-BASED CONDITIONING IN A CHILD WITH SEVERE APLASTIC ANEMIA: A CASE REPORT (EBMT 2026) - " Immunoablation was initiated with four doses of equine antithymocyte globulin (Atgam, 40 mg/kg), after which a substantial number of residual T lymphocytes was observed (818/µL). To intensify the immunoablation, the patient subsequently received a 3-day course of antithymocyte globulin (Grafalon, 5 mg/kg), followed by a 2-day course of antithymocyte globulin (Thymoglobulin, 3.75 mg/kg), administered with methylprednisolone and cyclosporine A. Subsequently, the child underwent auto-UCBT containing 0.13 × 10⁶ CD34+ cells/kg and continued treatment with cyclosporine A and prednisone... Early neutrophil recovery may indicate better underlying stem cell reserve, due to auto-UCBT infusion, though this interpretation remains speculative. Auto-UCBT after immunosuppression-based protocol appears to be a promising curative option for pediatric SAA patients with stored autologous cord blood and without available matched sibling. A key prerequisite for autoUCBT in SAA is..."

Case report • Clinical • Anemia • Aplastic Anemia • Bone Marrow Transplantation • Hematological Disorders • Transplantation • CD34

A SYSTEMATIC REVIEW OF REAL-WORLD EVIDENCE ON THERAPIES FOR MODERATE APLASTIC ANAEMIA (EBMT 2026) - "Treatment choices depend on blood counts, transfusion dependency or other significant symptoms and include supportive care, monotherapy (cyclosporine [CSA], tacrolimus, daclizumab, thrombopoietin receptor agonists [TPO-RA], androgens), double or triple therapy, (combining horse or rabbit anti-thymocyte globulin [hATG or rATG], CSA/tacrolimus, and eltrombopag [EPAG]/TPO-RA or androgens), and/or haematopoietic stem-cell transplantation (HSCT)... Across heterogenous studies, OS and ORR have been documented for monotherapies, double, and triple therapies; HSCT was only reported in small, heterogeneous cohorts. Due to varied timepoints, missing data, and cohort selection, there was insufficient homogenous data for quantitative analysis. This SLR demonstrates a clear need for uniform definition of mAA and for comparative, prospective studies to enable the comparison of therapeutic effectiveness and to develop sufficient treatment algorithms."

Clinical • HEOR • Real-world • Real-world evidence • Review • Anemia • Aplastic Anemia • Bone Marrow Transplantation • Hematological Disorders

OUTCOME OF UNRELATED AND HAPLOIDENTICAL HSCT AMONG CHILDREN WITH MYELODYSPLASTIC SYNDROME (EBMT 2026) - "16 patients were treated using the αβ T-cell depletion platform, 17 patients were treated using post-transplant cyclophosphamide (PTCy), 3 – with conventional CNI-based GVHD prophylaxis (group characteristics, table 1).All patients had cytopenia: transfusion dependence, leukopenia with neutropenia moderate to severe, 5 patients had progression to acute myeloid leukemia (AML).17 cases had germline condition predisposing to MDS: 2 - Shwachman-Diamond syndrome (SDS), 2 - Severe Congenital Neutropenia, 2 unspecified primary immune deficiency, 1 patient due to Diamond-Blackfan anemia, 2 patients had Down syndrome, 1 patient had Noonan syndrome...5 patients with AML received various courses of hypomethylating agents.Preparative regimen included treosulfan 42 gr/m2, fludarabine 150 mg/kg, thiotepa 10 mg/kg or melphalan 140 mg/m2. Thymoglobulin 5mg/kg or ATGAM 100 mg/kg and rituximab 200mg/m2 were used in a proportion of cases.Post-transplant GVHD prophylaxis included for αβ..."

Clinical • Acute Myelogenous Leukemia • Bone Marrow Transplantation • Developmental Disorders • Genetic Disorders • Graft versus Host Disease • Hematological Disorders • Hematological Malignancies • Immunology • Infectious Disease • Leukemia • Leukopenia • Myelodysplastic Syndrome • Neutropenia • Primary Immunodeficiency • GATA1 • GATA2

OUTCOMES OF ALLOGENEIC HSCT IN NIJMEGEN BREAKAGE SYNDROME WITH TREOSULFAN-BASED CONDITIONING REGIMEN - A MULTICENTER STUDY (EBMT 2026) - "We recently demonstrated experience of treosulfan 30g/m2 versus low doses of busulfan...All received fludarabine 150mg/m2, 24 cyclophosphamide (22–40mg/kg, 1–30mg/kg, 1–20mg/kg). In 37 patients, rabbit ATG (thymoglobulin, Genzyme) 3-7,5mg/kg, in 1 patient horse ATG (ATGAM) 100mg/kg, and in 2 patients no serotherapy was used.In 25 patients matched unrelated (19–10/10, 6–9/10 HLA-matched), in 9 mismatched related, in 6 matched sibling donor was used... Both doses of treosulfan 21g/m2 and 30g/m2 were well tolerated by NBS patients. However, lower dose of 21g/m2 was associated with higher risk of mixed chimerism and leukemia relapse versus increased incidence of secondary malignancy after higher dose of 30g/m2."

Clinical • Acute Graft versus Host Disease • Bone Marrow Transplantation • Chronic Graft versus Host Disease • Graft versus Host Disease • Hematological Malignancies • Immunology • Infectious Disease • Leukemia • Lymphoma • Metabolic Disorders • Primary Immunodeficiency • Rhabdomyosarcoma • Sarcoma • Solid Tumor • Transplant Rejection

TRIUMPH: TReatment for ImmUne Mediated PathopHysiology (clinicaltrials.gov) - P2 | N=163 | Recruiting | Sponsor: National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) | Trial completion date: May 2027 ➔ Feb 2027 | Trial primary completion date: Apr 2027 ➔ Aug 2026

Trial completion date • Trial primary completion date • CNS Disorders • Hepatic Encephalopathy • Hepatology • Liver Failure • Pediatrics

Stem Cell Transplantation in Crohn's Disease (clinicaltrials.gov) - P1/2 | N=15 | Recruiting | Sponsor: Cedars-Sinai Medical Center | Trial completion date: Sep 2026 ➔ Sep 2027 | Trial primary completion date: Sep 2026 ➔ Sep 2027

Trial completion date • Trial primary completion date • Bone Marrow Transplantation • Crohn's disease • Gastroenterology • Genetic Disorders • Immunology • Inflammatory Bowel Disease • Transplantation

A meta-analysis of immunosuppressive and Pharmacological therapies in aplastic anaemia with and without Indigenous equine antithymocyte globulin (eATG). (PubMed, Ann Hematol) - "Therefore, we performed a meta-analysis to evaluate the effectiveness and safety of various immunosuppressive strategies-particularly equine antithymocyte globulin (eATG) combined with cyclosporine A (CSA), with or without eltrombopag (EPAG), as well as CSA and anabolic steroids-based monotherapies, for the treatment of AA in Indian patients. Regimens incorporating EPAG offer superior response rates, and THYMOGAM presents a cost-effective yet clinically viable alternative to ATGAM. These insights support more tailored, accessible treatment strategies in resource-constrained settings."

Clinical • Journal • Retrospective data • Review • Anemia • Aplastic Anemia • Bone Marrow Transplantation • Febrile Neutropenia • Hematological Disorders • Neutropenia • Transplantation

Selective lymphodepletion underlies the efficacy of horse anti-thymocyte globulin-based immunosuppressive therapy in aplastic anemia. (PubMed, Haematologica) - "This could explain the crucial contribution of long-term ciclosporin to successful IST. Collectively, our results identify ATGAM exposure as a factor influencing hematologic recovery, and indicate that the therapeutic effect of IST goes beyond total lymphodepletion but is rather the result of selective depletion and suppression of key lymphocyte subpopulations."

Journal • Anemia • Aplastic Anemia • Hematological Disorders • CCR6 • CD27 • CD4 • CD8 • KLRG1

Atgam Efficacy and Safety in Moderate-to-Very Severe Acquired Aplastic Anemia: Outcome of a Large Multicenter Cohort of 634 Children and Adults from the French Authorization for Temporary Use Surveillance Program (ASH 2023) - "Results In total, 634 patients with moderate-to-very severe AA were treated with ATGAM (n=537 first-line; n=68 refractory/relapse; n=29 not classified) in addition to cyclosporine (40 first-line patients also received eltrombopag) from January 2012 to August 2022. No new safety risks were identified in this large cohort of patients. Treatment with ATGAM remains of benefit in patients with moderate-to-very severe AA."

Clinical • Anemia • Aplastic Anemia • Hematological Disorders • Immunology

Long-term outcomes of αβ T-cell/CD19 B-cell-depleted peripheral blood stem cell transplantation from unrelated donors in pediatric and adolescent patients with severe aplastic anemia: a single-center study. (PubMed, Cytotherapy) - "TCR alpha/beta depletion is a robust platform for allogeneic HSCT from MUD in severe aplastic anemia. Results should be further improved by additional measures to control viral infections and prevent rejection."

Journal • Acute Graft versus Host Disease • Anemia • Aplastic Anemia • Bone Marrow Transplantation • Chronic Graft versus Host Disease • Complement-mediated Rare Disorders • Graft versus Host Disease • Hematological Disorders • Immunology • Infectious Disease • Paroxysmal Nocturnal Hemoglobinuria • Pediatrics • Rare Diseases • Transplantation • CD34

A Trial Comparing Unrelated Donor BMT With IST for Pediatric and Young Adult Patients With Severe Aplastic Anemia (TransIT, BMT CTN 2202) (clinicaltrials.gov) - P3 | N=53 | Active, not recruiting | Sponsor: Boston Children's Hospital | Recruiting ➔ Active, not recruiting | N=234 ➔ 53

Enrollment change • Enrollment closed • Anemia • Aplastic Anemia • Bone Marrow Transplantation • Hematological Disorders • Pediatrics • Transplantation • HLA-B • HLA-C • HLA-DQB1 • HLA-DRB1

Autologous Stem Cell Transplant for Neurologic Autoimmune Diseases (clinicaltrials.gov) - P2 | N=53 | Active, not recruiting | Sponsor: Fred Hutchinson Cancer Center | Trial primary completion date: Jun 2025 ➔ Dec 2025

Trial primary completion date • CNS Disorders • Immunology • Movement Disorders • Multiple Sclerosis • Myasthenia Gravis • Neuromyelitis Optica Spectrum Disorder • Pain • Rare Diseases • Transplantation • Vasculitis

A new era in the treatment of aplastic anemia (PubMed, Rinsho Ketsueki) - "Cyclosporine (CsA) monotherapy is recommended for patients who have mild disease or are moderately transfusion-independent, whereas the combination of anti-thymocyte globulin (ATG) and CsA is the standard treatment for severe disease. In 2023, equine ATG (ATGAM®) was approved in Japan, expanding the options for IST. TPO-RA options for combination therapy with IST now include romiplostim in addition to eltrombopag, and studies have demonstrated the efficacy of triple combination therapy with ATG, CsA, and a TPO-RA has been demonstrated. In the context of HSCT, HLA-haploidentical transplantation using post-transplant cyclophosphamide is increasingly being considered as an option for patients without an HLA-matched donor due to its improved safety and efficacy. This review provides a comprehensive overview of the latest advances in AA treatment, including novel therapeutic strategies, and discusses future therapeutic directions to further improve patient outcomes."

Journal • Review • Anemia • Aplastic Anemia • Bone Marrow Transplantation • Hematological Disorders • Transplantation