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1207 Baricitinib in Combination with High-Dose Dexamethasone As First-Line Treatment for Newly Diagnosed Immune Thrombocytopenia: A Prospective, Multicenter, Randomized Trial

Program: Oral and Poster Abstracts
Session: 311. Disorders of Platelet Number or Function: Clinical and Epidemiological: Poster I
Hematology Disease Topics & Pathways:
Research, clinical trials, Bleeding and Clotting, adult, Clinical Research, Combination therapy, Diseases, thrombocytopenias, Therapies, Study Population, Human
Saturday, December 9, 2023, 5:30 PM-7:30 PM

Peng Zhao1*, Zhuoyu An2*, Haixia Fu, MD2*, Daihong Liu3*, Liping Dou3*, Yi Liu4*, Hui Liu5*, Zhao Wang6*, Yujun Dong7*, Hongmei Jing8*, Hebing Zhou9*, Zhen-Ling Li10*, Lihong Li11*, Qiu-Sha Huang1*, Jin Wu2*, Yejun Wu2*, Chen-Cong Wang2*, Xiaolu Zhu, MD12*, Qi Chen2*, Yun He2*, Kaiyan Liu, MD, PhD2 and Xiaohui Zhang, MD, PhD2*

1Peking University People's Hospital, Peking University Institute of Hematology, Beijing, CHN
2Peking University People's Hospital, Peking University Institute of Hematology, Beijing, China
3Chinese PLA General Hospital, Beijing, China
4The Sixth Medical Center of PLA General Hospital, Beijing, CHN
5Beijing Hospital, Beijing, China
6Beijing Friendship Hospital, Beijing, China
7Peking University First Hospital, Beijing, China
8Peking University Third Hospital, Beijing, China
9Beijing Luhe Hospital, Beijing, China
10China-Japan Friendship Hospital, Beijing,, Beijing, CHN
11Beijing Tsinghua Changgeng Hospital, Beijing, China
12Peking University Institute of Hematology, Peking University People’s Hospital, Peking University Institute of Hematology, National Clinical Research Center for Hematologic Disease, Beijing Key Laboratory of Hematopoietic Stem Cell Transplantation, Beijing, China


High-dose dexamethasone is among the standard initial therapeutic options for patients with newly diagnosed immune thrombocytopenia (ITP). Although most patients achieve initial remission, optimal first-line therapy remains a challenge due to the low rates of sustained response and the high incidence of corticosteroid-dependency. Antiplatelet autoantibodies and splenic macrophages play central roles in the pathogenesis of ITP. Baricitinib, a Janus-associated kinase 1/2 (JAK1/2) inhibitor, can inhibit antigen presentation by innate immune cells and subsequent T-cell activation while also alleviating B-cell abnormalities and antibody production. The results of our single-arm pilot study suggested that baricitinib might increase the rate of durable response in patients with steroid-resistant or relapsed ITP (NCT05446831, presented at EHA2023, P1594). Accordingly, we further conducted a randomized, controlled trial to evaluate the efficacy and safety of baricitinib plus high-dose dexamethasone as first-line therapy for adulthood ITP.


This multicenter, open-label, randomized, controlled, phase 2 trial was conducted in 10 different tertiary medical centers in China. Adult patients with newly diagnosed, treatment-naïve, confirmed primary ITP who had a platelet count <30×10⁹/L, or a platelet count <50×10⁹/L with clinically significant bleeding, were eligible for this study. Patients were randomly assigned at a 1:1 ratio to receive baricitinib (2 mg daily for 6 consecutive months) plus high-dose dexamethasone (40 mg daily for 4 consecutive days, repeated in the case of lack of response by Day 10) or high-dose dexamethasone alone. The primary endpoint was durable response at the 6-month follow-up. Efficacy endpoints were analyzed in the intention-to-treat population. Safety profiles were analyzed in all patients receiving at least one dose of study medications. The study protocol was approved by the ethics committee or institutional review board of each participating center and was registered with ClinicalTrials.gov (NCT 05932524).


A total of 172 patients were screened, of which 132 patients were eligible for this study. Patients were randomly assigned to receive baricitinib plus high-dose dexamethasone (n=66) or high-dose dexamethasone monotherapy (n=66). Four patients did not receive allocated treatment, leaving 128 patients in the safety analysis set. At the 6-month follow-up, a significantly higher proportion of participants in the baricitinib plus high-dose dexamethasone group (42/66, 63.6%) than in the high-dose dexamethasone monotherapy group (25/66, 37.9%) achieved durable response (OR 2.870, 95% CI 1.416-5.816; p=0.003). Initial response was achieved by 52 (78.8%) patients in the combination group and 47 (71.2%) patients in the monotherapy group. No significant difference was observed in the median time from initial treatment to response between the two groups. Complete response (at any time during the treatment) was achieved by 40 (60.6%) patients in the combination group and 35 (53.0%) patients in the monotherapy group. Patients responding to treatment in the combination group showed significantly longer durations of response than responders in the monotherapy group. Patients in both groups reported significantly improved health-related quality of life. The most common adverse events were infections (15/64, 23.4%) and insomnia (12/64, 18.8%) in the combination group, and insomnia (10/64, 15.6%) and anxiety or mood disorders (9/64, 14.1%) in the monotherapy group. No grade 4 or worse adverse events or treatment-related deaths were reported in either group. No thrombotic events, major adverse cardiovascular events (MACE), treatment-related bleeding or anemia were observed. Treatment discontinuation due to an adverse event was reported in 2 (3.1%) patients in the combination group and 3 (4.7%) patients in the monotherapy group.


This is the first study reporting JAK inhibition as a first-line therapy for ITP. Baricitinib at 2 mg daily plus high-dose dexamethasone was well tolerated in patients with newly diagnosed ITP and might contribute to achieving a higher rate of durable response.

Disclosures: No relevant conflicts of interest to declare.

OffLabel Disclosure: Baricitinib (Olumiant, 2MG), an orally administrated, reversible, and selective Janus-associated kinase 1/2 (JAK1/2) inhibitor, is evaluated for its efficacy and safety as first-line therapy for newly diagnosed primary ITP.

*signifies non-member of ASH