Safety and efficacy of baricitinib in steroid-resistant or relapsed immune thrombocytopenia: An open-label pilot study.

Autor:	Zhao P; Peking University Institute of Hematology, Peking University People's Hospital, Beijing, China.; National Clinical Research Center for Hematologic Disease, Beijing, China.; Beijing Key Laboratory of Hematopoietic Stem Cell Transplantation, Beijing, China.; Collaborative Innovation Center of Hematology, Peking University, Beijing, China., An ZY; Peking University Institute of Hematology, Peking University People's Hospital, Beijing, China.; National Clinical Research Center for Hematologic Disease, Beijing, China.; Beijing Key Laboratory of Hematopoietic Stem Cell Transplantation, Beijing, China.; Collaborative Innovation Center of Hematology, Peking University, Beijing, China., Fu HX; Peking University Institute of Hematology, Peking University People's Hospital, Beijing, China.; National Clinical Research Center for Hematologic Disease, Beijing, China.; Beijing Key Laboratory of Hematopoietic Stem Cell Transplantation, Beijing, China.; Collaborative Innovation Center of Hematology, Peking University, Beijing, China., Liu HX; Department of Clinical Epidemiology, Peking University People's Hospital, Beijing, China., Feng CJ; Peking University Institute of Hematology, Peking University People's Hospital, Beijing, China.; National Clinical Research Center for Hematologic Disease, Beijing, China.; Beijing Key Laboratory of Hematopoietic Stem Cell Transplantation, Beijing, China.; Collaborative Innovation Center of Hematology, Peking University, Beijing, China., Huang QS; Peking University Institute of Hematology, Peking University People's Hospital, Beijing, China.; National Clinical Research Center for Hematologic Disease, Beijing, China.; Beijing Key Laboratory of Hematopoietic Stem Cell Transplantation, Beijing, China.; Collaborative Innovation Center of Hematology, Peking University, Beijing, China., Wu J; Peking University Institute of Hematology, Peking University People's Hospital, Beijing, China.; National Clinical Research Center for Hematologic Disease, Beijing, China.; Beijing Key Laboratory of Hematopoietic Stem Cell Transplantation, Beijing, China.; Collaborative Innovation Center of Hematology, Peking University, Beijing, China., Wu YJ; Peking University Institute of Hematology, Peking University People's Hospital, Beijing, China.; National Clinical Research Center for Hematologic Disease, Beijing, China.; Beijing Key Laboratory of Hematopoietic Stem Cell Transplantation, Beijing, China.; Collaborative Innovation Center of Hematology, Peking University, Beijing, China., Yang LP; Peking University Institute of Hematology, Peking University People's Hospital, Beijing, China.; National Clinical Research Center for Hematologic Disease, Beijing, China.; Beijing Key Laboratory of Hematopoietic Stem Cell Transplantation, Beijing, China.; Collaborative Innovation Center of Hematology, Peking University, Beijing, China., Qu QY; Peking University Institute of Hematology, Peking University People's Hospital, Beijing, China.; National Clinical Research Center for Hematologic Disease, Beijing, China.; Beijing Key Laboratory of Hematopoietic Stem Cell Transplantation, Beijing, China.; Collaborative Innovation Center of Hematology, Peking University, Beijing, China., Chen YX; Peking University Institute of Hematology, Peking University People's Hospital, Beijing, China.; National Clinical Research Center for Hematologic Disease, Beijing, China.; Beijing Key Laboratory of Hematopoietic Stem Cell Transplantation, Beijing, China.; Collaborative Innovation Center of Hematology, Peking University, Beijing, China., Li ML; Peking University Institute of Hematology, Peking University People's Hospital, Beijing, China.; National Clinical Research Center for Hematologic Disease, Beijing, China.; Beijing Key Laboratory of Hematopoietic Stem Cell Transplantation, Beijing, China.; Collaborative Innovation Center of Hematology, Peking University, Beijing, China., Wang CC; Peking University Institute of Hematology, Peking University People's Hospital, Beijing, China.; National Clinical Research Center for Hematologic Disease, Beijing, China.; Beijing Key Laboratory of Hematopoietic Stem Cell Transplantation, Beijing, China.; Collaborative Innovation Center of Hematology, Peking University, Beijing, China., Chen Q; Peking University Institute of Hematology, Peking University People's Hospital, Beijing, China.; National Clinical Research Center for Hematologic Disease, Beijing, China.; Beijing Key Laboratory of Hematopoietic Stem Cell Transplantation, Beijing, China.; Collaborative Innovation Center of Hematology, Peking University, Beijing, China., Zhu XL; Peking University Institute of Hematology, Peking University People's Hospital, Beijing, China.; National Clinical Research Center for Hematologic Disease, Beijing, China.; Beijing Key Laboratory of Hematopoietic Stem Cell Transplantation, Beijing, China.; Collaborative Innovation Center of Hematology, Peking University, Beijing, China., He Y; Peking University Institute of Hematology, Peking University People's Hospital, Beijing, China.; National Clinical Research Center for Hematologic Disease, Beijing, China.; Beijing Key Laboratory of Hematopoietic Stem Cell Transplantation, Beijing, China.; Collaborative Innovation Center of Hematology, Peking University, Beijing, China., Zhang YY; Peking University Institute of Hematology, Peking University People's Hospital, Beijing, China.; National Clinical Research Center for Hematologic Disease, Beijing, China.; Beijing Key Laboratory of Hematopoietic Stem Cell Transplantation, Beijing, China.; Collaborative Innovation Center of Hematology, Peking University, Beijing, China., Jiang Q; Peking University Institute of Hematology, Peking University People's Hospital, Beijing, China.; National Clinical Research Center for Hematologic Disease, Beijing, China.; Beijing Key Laboratory of Hematopoietic Stem Cell Transplantation, Beijing, China.; Collaborative Innovation Center of Hematology, Peking University, Beijing, China., Jiang H; Peking University Institute of Hematology, Peking University People's Hospital, Beijing, China.; National Clinical Research Center for Hematologic Disease, Beijing, China.; Beijing Key Laboratory of Hematopoietic Stem Cell Transplantation, Beijing, China.; Collaborative Innovation Center of Hematology, Peking University, Beijing, China., Lu J; Peking University Institute of Hematology, Peking University People's Hospital, Beijing, China.; National Clinical Research Center for Hematologic Disease, Beijing, China.; Beijing Key Laboratory of Hematopoietic Stem Cell Transplantation, Beijing, China.; Collaborative Innovation Center of Hematology, Peking University, Beijing, China., Chang YJ; Peking University Institute of Hematology, Peking University People's Hospital, Beijing, China.; National Clinical Research Center for Hematologic Disease, Beijing, China.; Beijing Key Laboratory of Hematopoietic Stem Cell Transplantation, Beijing, China.; Collaborative Innovation Center of Hematology, Peking University, Beijing, China., Zhao XS; Peking University Institute of Hematology, Peking University People's Hospital, Beijing, China.; National Clinical Research Center for Hematologic Disease, Beijing, China.; Beijing Key Laboratory of Hematopoietic Stem Cell Transplantation, Beijing, China.; Collaborative Innovation Center of Hematology, Peking University, Beijing, China., Zhao XY; Peking University Institute of Hematology, Peking University People's Hospital, Beijing, China.; National Clinical Research Center for Hematologic Disease, Beijing, China.; Beijing Key Laboratory of Hematopoietic Stem Cell Transplantation, Beijing, China.; Collaborative Innovation Center of Hematology, Peking University, Beijing, China., Huang XJ; Peking University Institute of Hematology, Peking University People's Hospital, Beijing, China.; National Clinical Research Center for Hematologic Disease, Beijing, China.; Beijing Key Laboratory of Hematopoietic Stem Cell Transplantation, Beijing, China.; Collaborative Innovation Center of Hematology, Peking University, Beijing, China., Zhang XH; Peking University Institute of Hematology, Peking University People's Hospital, Beijing, China.; National Clinical Research Center for Hematologic Disease, Beijing, China.; Beijing Key Laboratory of Hematopoietic Stem Cell Transplantation, Beijing, China.; Collaborative Innovation Center of Hematology, Peking University, Beijing, China.
Jazyk:	angličtina
Zdroj:	American journal of hematology [Am J Hematol] 2024 Jul 09. Date of Electronic Publication: 2024 Jul 09.
DOI:	10.1002/ajh.27433
Abstrakt:	Patients with steroid-resistant or relapsed immune thrombocytopenia (ITP) suffer increased bleeding risk and impaired quality of life. Baricitinib, an oral Janus-associated kinases (JAK) inhibitor, could alleviate both innate and adaptive immune disorders without inducing thrombocytopenia in several autoimmune diseases. Accordingly, an open-label, single-arm, phase 2 trial (NCT05446831) was initiated to explore the safety and efficacy of baricitinib in ITP. Eligible patients were adults with primary ITP who were refractory to corticosteroids and at least one subsequent treatment, and had platelet counts below 30 × 10 9 /L at enrolment. Participants received baricitinib 4 mg daily for 6 months. The primary endpoint was durable response at the 6-month follow-up. A total of 35 patients were enrolled. Durable response was achieved in 20 patients (57.1%, 95% confidence interval, 39.9 to 74.4), and initial response in 23 (65.7%) patients. For patients responding to baricitinib, the median time to response was 12 (IQR 6-20) days, and the median peak platelet count was 94 (IQR 72-128) × 10 9 /L. Among the 27 patients undergoing extend observation, 12 (44.4%) remained responsive for a median duration of approximately 20 weeks after baricitinib discontinuation. Adverse events were reported in 11 (31.4%) patients, including infections in 6 (17.1%) patients during the treatment period. Treatment discontinuation due to an adverse event was reported in 2 (5.7%) patients. Evidence from this pilot study suggested that baricitinib might be a novel candidate for the armamentarium of ITP-modifying agents. Future studies are warranted to validate the safety, efficacy, and optimal dosing of baricitinib in patients with ITP. (© 2024 Wiley Periodicals LLC.)
Databáze:	MEDLINE
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