A Dose-Escalation Phase 1 Clinical Trial of Autologous iPSC-Derived Platelets (iPLAT1)

Naoshi Sugimoto, M.D., Ph.D.
Center for iPS Cell Research and Application (CiRA), Kyoto University
Kyoto, Japan

Platelet transfusions can be complicated with alloimmune platelet transfusion refractoriness. It is difficult to find donors for patients with rare human leukocyte antigen-I or human platelet antigen (HPA). Naoshi Sugimoto, M.D., Ph.D., and team were part of a group that assisted in development of an ex vivo production system of induced pluripotent stem cells (iPSC)-derived platelet (PLT) products. This study aimed to assess the safety and efficacy of autologous iPSC-PLTs in a patient with rare HPA-1 mismatch. No significant adverse events were observed over one year. This marks a first benchmark in realizing iPSC-PLTs as a clinical measure.

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