Efficacy and safety of equine anti-thymocyte immunoglobulin (eATG) in three Japanese patients with moderate to very severe aplastic anemia: a case series.

Autor: Kanda Y; Division of Hematology, Department of Medicine, Jichi Medical University, 3311-1 Yakushiji, Shimotsuke-Shi, Tochigi-Ken, 329-0498, Japan. ycanda-tky@umin.ac.jp., Mori T; Division of Hematology, Department of Medicine, Keio University School of Medicine, Tokyo, Japan.; Department of Hematology, Tokyo Medical and Dental University, Tokyo, Japan., Narita A; Department of Pediatrics, Nagoya University Graduate School of Medicine, Nagoya, Japan., Wolter KD; Pfizer Inc, New York, NY, USA., Yoshimatsu H; Pfizer R&D Japan, Tokyo, Japan., Nishimura K; Pfizer R&D Japan, Tokyo, Japan.
Jazyk: angličtina
Zdroj: International journal of hematology [Int J Hematol] 2023 Jan; Vol. 117 (1), pp. 37-43. Date of Electronic Publication: 2022 Nov 28.
DOI: 10.1007/s12185-022-03496-5
Abstrakt: Aplastic anemia results from lymphocyte-mediated destruction of hematopoietic stem cells. Immunosuppressive therapy with anti-thymocyte globulin (ATG) and cyclosporine is the standard front-line treatment for patients with severe aplastic anemia who are not suitable candidates for stem cell transplants. PF-06462700 is a potent equine ATG that targets T-lymphocytes and has been approved as a treatment for aplastic anemia outside of Japan for over 30 years. Due to the high medical need for PF-06462700, the Ministry of Health, Labor and Welfare requested its development for Japanese patients with aplastic anemia. In this case series, the efficacy and safety of PF-06462700, administered intravenously at 40 mg/kg/day for 4 days, were assessed over a 24-week period. This was as an open-label, single-arm, multicenter clinical study designed to enroll a minimum of three Japanese participants with aplastic anemia. Two participants met the primary outcome of hematologic response at week 12 and improvements in disease severity were observed. No deaths or serious adverse events were reported. The efficacy results from this case series suggest that administration of PF-06462700 is generally well-tolerated and produces a hematologic response in Japanese patients with aplastic anemia, which should be further evaluated in real-world studies.ClinicalTrials.gov identifier: NCT04350606.
(© 2022. Japanese Society of Hematology.)
Databáze: MEDLINE
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