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

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

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

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

ATGAM versus Thymoglobulin in Kidney Transplant Tolerance Induction in Non-Human Primates (WTC 2025) - "Therefore, we aim to evaluate rabbit anti-thymocyte globulin (Thymoglobulin) as an alternative T-cell depleting agent for tolerance induction through the mixed chimerism approach.* MHC mismatched cynomolgus monkeys underwent a preconditioning regimen consisting of 1.5 Gy total body irradiation, a peri-transplant course of a Bcl-2 inhibitor (Venetoclax 10mg/kg from day -4 to day +6), 7 Gy local thymic irradiation and either ATGAM (50mg/kg on days -2, -1 and 0; n=8) or Thymoglobulin (5mg/kg on days -1 and 0; n=6)...All recipients then underwent combined kidney and bone marrow transplantation (CKBMT) and received anti-CD154 mAb (20mg/kg on days 0, 2, 5 and 12) along with a 28-day course of cyclosporine, after which no immunosuppression was administered... Compared to Thymoglobulin, using ATGAM as part of a chimerism based tolerance induction strategy promoted longer-lasting myeloid and lymphoid chimerism with a lower rate of rejection but similar overall graft survival...."

Bone Marrow Transplantation • Infectious Disease • Transplant Rejection • Transplantation • CD40LG

Emerging Trends in List Price Adjustment Strategies Following Implementation of the Inflation Reduction Act (ISPOR 2025) - "Among products analyzed, Pfizer’s Atgam experienced the largest price increase at +15%. In contrast, significant price decreases were observed for Boehringer Ingelheim’s Atrovent (-35%) and Merck’s Januvia and Janumet/Janumet XR (-42.4%)...Excluding Januvia, the year-over-year (YoY) percent change in price for the six other IRA negotiation products with 2025 data declined from +4.0% in 2024 to +2.4% in 2025; Eliquis had the largest shift YoY from +6% in 2024 to +2% in 2025... List price adjustments reflect both product-specific and broader industry dynamics. In anticipation of IRA negotiations, IRA products are experiencing lower than average price increases. As government regulations evolve and pricing pressure on pharmaceuticals increase, assessing price adjustments in the context of anticipated payer management and government penalties will be a key consideration for manufacturers."

Unrelated Donor Transplant Versus Immune Therapy in Pediatric Severe Aplastic Anemia (clinicaltrials.gov) - P=N/A | N=40 | Completed | Sponsor: Michael Pulsipher | Active, not recruiting ➔ Completed

Trial completion • Anemia • Aplastic Anemia • Hematological Disorders • Pediatrics • Transplantation • HLA-B • HLA-C • HLA-DQB1 • HLA-DRB1

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: Jan 2027 ➔ May 2027 | Trial primary completion date: Jan 2026 ➔ Apr 2027

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

COMPARISON OF OUTCOMES OF IMMUNOSUPPRESSIVE THERAPY WITH RABBIT VS. HORSE ANTI-THYMOCYTE GLOBULIN AND CYCLOSPORINE A IN PATIENTS WITH REFRACTORY CYTOPENIA OF CHILDHOOD ​ (EBMT 2025) - " We report the outcomes of IST in 133 children (80 males/53 females) with RCC and treated with either h-ATG (Atgam®, n=64) or r-ATG (Thymoglobulin®, n=69) and cyclosporine A. The median age at diagnosis was 9.0 (1.7-18.1) years...Eltrombopag was added on day 344 in one patient... The results suggest that IST with both h-ATG and r-ATG is an effective therapy, achieving a response rate of approximately 50% in RCC patients. Although no significant differences were observed in response rate or FFS between the two ATG groups, OS was higher in patients receiving h-ATG, indicating a potential advantage of h-ATG. Patients with persistent severe neutropenia face a high risk of mortality and should be considered for early second-line HSCT."

Clinical • Preclinical • Acute Myelogenous Leukemia • Anemia • Aplastic Anemia • Bone Marrow Transplantation • Hematological Disorders • Hematological Malignancies • Infectious Disease • Leukemia • Neutropenia • Oncology • Renal Cell Carcinoma • Septic Shock

COMPARISON OF OUTCOMES OF IMMUNOSUPPRESSIVE THERAPY WITH RABBIT VS. HORSE ANTI-THYMOCYTE GLOBULIN AND CYCLOSPORINE A IN PATIENTS WITH REFRACTORY CYTOPENIA OF CHILDHOOD ​ (EBMT 2025) - " We report the outcomes of IST in 133 children (80 males/53 females) with RCC and treated with either h-ATG (Atgam®, n=64) or r-ATG (Thymoglobulin®, n=69) and cyclosporine A. The median age at diagnosis was 9.0 (1.7-18.1) years...Eltrombopag was added on day 344 in one patient... The results suggest that IST with both h-ATG and r-ATG is an effective therapy, achieving a response rate of approximately 50% in RCC patients. Although no significant differences were observed in response rate or FFS between the two ATG groups, OS was higher in patients receiving h-ATG, indicating a potential advantage of h-ATG. Patients with persistent severe neutropenia face a high risk of mortality and should be considered for early second-line HSCT."

Clinical • Preclinical • Acute Myelogenous Leukemia • Anemia • Aplastic Anemia • Bone Marrow Transplantation • Hematological Disorders • Hematological Malignancies • Infectious Disease • Leukemia • Neutropenia • Oncology • Renal Cell Carcinoma • Septic Shock

IN VIVO EFFECTS AND PHARMACODYNAMICS OF ATGAM IN ACQUIRED APLASTIC ANEMIA PATIENTS (EBMT 2025) - "The first-line treatment for most AA patients is immunosuppressive therapy (IST), consisting of 4 consecutive days 40 mg/kg/day horse-derived anti-thymocyte globulin (ATGAM®) and long-term ciclosporin. This study provides unique insight into the pharmacokinetics and pharmacodynamics of ATGAM in AA patients. We found that patients with a higher body weight have higher ATGAM exposures, which tend to correlate with improved hematologic recovery. Additionally, our analyses demonstrate that the therapeutic effect of ATGAM may be linked to the targeted depletion of specific lymphoid populations."

PK/PD data • Preclinical • Anemia • Aplastic Anemia • Hematological Disorders • Immunology

IN VIVO EFFECTS AND PHARMACODYNAMICS OF ATGAM IN ACQUIRED APLASTIC ANEMIA PATIENTS (EBMT 2025) - "The first-line treatment for most AA patients is immunosuppressive therapy (IST), consisting of 4 consecutive days 40 mg/kg/day horse-derived anti-thymocyte globulin (ATGAM®) and long-term ciclosporin. This study provides unique insight into the pharmacokinetics and pharmacodynamics of ATGAM in AA patients. We found that patients with a higher body weight have higher ATGAM exposures, which tend to correlate with improved hematologic recovery. Additionally, our analyses demonstrate that the therapeutic effect of ATGAM may be linked to the targeted depletion of specific lymphoid populations."

PK/PD data • Preclinical • Anemia • Aplastic Anemia • Hematological Disorders • Immunology

Stem Cell Transplantation in Crohn's Disease (clinicaltrials.gov) - P1/2 | N=15 | Recruiting | Sponsor: Cedars-Sinai Medical Center | Trial completion date: Jan 2025 ➔ Sep 2026 | Trial primary completion date: Jan 2025 ➔ Sep 2026

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