Safety, Pharmacokinetics, and Efficacy Study of Porcine and Rabbit Antithymocyte Globulin in the Treatment of Patients With Severe Aplastic Anemia: A Multicenter, Prospective, Randomized, Open-Label Clinical Trial
In brief
Porcine and rabbit ATG each achieved about 19% complete response at six months
In a randomized study of 93 patients with severe aplastic anemia, complete response rates at six months were about 19% with either porcine or rabbit antithymocyte globulin; overall response rates were 70% and 57%, respectively. Two-year response and survival were similar, though the treatments affected immune cells differently. Their similar outcomes support either option, but the study was small.
- Journal
- MedComm (Q1)
- Published
- 17 October 2026
- Study design
- Randomized controlled trial
- Evidence level
- Level 1, High (CEBM 1b)
- Authors
- Yawen Zhang, Yingying Zhang, Yuemin Gong, Yu Sun, Guangsheng He
- PMID
- 42893333
- DOI
- 10.1002/mco2.71084
Why clinicians should know about it
- Picked for Immunology and Allergy (top studies of the week, 1 November 2026): Randomized trial comparing porcine vs rabbit ATG in SAA
Abstract
Severe aplastic anemia (SAA) is a T lymphocyte-mediated bone marrow failure disorder. Only porcine ATG (p-ATG) and rabbit ATG (r-ATG) are available in mainland China, with the optimal first-line regimen undetermined. This study compared their pharmacokinetics, efficacy, and safety. Ninety-three SAA patients ineligible for transplantation or lacking a suitable donor (p-ATG, n = 55; r-ATG, n = 38) who were receiving immunosuppressive therapy were prospectively enrolled between 2020 and 2024. The primary endpoint was dynamic changes in lymphocyte subsets. Both ATGs peaked on treatment Day 5; r-ATG cleared significantly faster, with comparable peak active concentrations. The p-ATG group showed a stable early neutrophil increase, whereas the r-ATG group had an initial decline, with comparable recovery at 1 month. The r-ATG group exhibited prolonger lymphopenia, lower CD3+/CD4+ T cells, reduced Treg cells, and higher CD19+ B-cell proportions. IL-10 was significantly elevated on day 2 after ATG administration. At 6 months, the CRR was 18.87% with p-ATG and 18.92% with r-ATG; ORR was 69.81% with p-ATG and 56.76% with r-ATG. Two-year ORR and overall survival were similar, with no significant differences in infection or serum sickness. P-ATG and r-ATG have distinct pharmacokinetic and immunomodulatory but comparable efficacy and safety profiles, supporting interchangeable use in clinical practice.
Abstract as published, via PubMed.
For healthcare professionals. The summary is generated by AI from the published abstract, and the evidence level is assigned automatically from the study design on the Oxford CEBM hierarchy. Neither is medical advice. Read the full paper before changing practice.