PMID- 34665865 OWN - NLM STAT- MEDLINE DCOM- 20220307 LR - 20220307 IS - 1528-0020 (Electronic) IS - 0006-4971 (Linking) VI - 139 IP - 3 DP - 2022 Jan 20 TI - All-trans retinoic acid plus low-dose rituximab vs low-dose rituximab in corticosteroid-resistant or relapsed ITP. PG - 333-342 LID - 10.1182/blood.2021013393 [doi] AB - The study aimed to compare the efficacy and safety of all-trans retinoic acid (ATRA) plus low-dose rituximab (LD-RTX) with LD-RTX monotherapy in corticosteroid-resistant or relapsed immune thrombocytopenia (ITP) patients. Recruited patients were randomized at a ratio of 2:1 into 2 groups: 112 patients received LD-RTX plus ATRA, and 56 patients received LD-RTX monotherapy. Overall response (OR), defined as achieving a platelet count of >/=30 x 109/L confirmed on >/=2 separate occasions (>/=7 days apart), at least a doubling of the baseline platelet count without any other ITP-specific treatment, and the absence of bleeding within 1 year after enrollment, was observed in more patients in the LD-RTX plus ATRA group (80%) than in the LD-RTX monotherapy group (59%) (between-group difference, 0.22; 95% CI, 0.07-0.36). Sustained response (SR), defined as maintenance of a platelet count >30 x 109/L, an absence of bleeding, and no requirement for any other ITP-specific treatment for 6 consecutive months after achievement of OR during 1 year following enrollment, was achieved by 68 (61%) patients in the combination group and 23 (41%) patients in the monotherapy group (between-group difference, 0.20; 95% CI, 0.04-0.35). The 2 most common adverse events (AEs) for the combination group were dry skin and headache or dizziness. Our findings demonstrated that ATRA plus LD-RTX significantly increased the overall and sustained response, indicating a promising treatment option for corticosteroid-resistant or relapsed adult ITP. This study is registered at www.clinicaltrials.gov as #NCT03304288. CI - (c) 2022 by The American Society of Hematology. FAU - Wu, Ye-Jun AU - Wu YJ AD - Peking University People's Hospital, Peking University Institute of Hematology, Beijing, China. AD - Collaborative Innovation Center of Hematology, Peking University, Beijing, China. AD - Beijing Key Laboratory of Hematopoietic Stem Cell Transplantation, Beijing, China. AD - National Clinical Research Center for Hematologic Disease, Beijing, China. FAU - Liu, Hui AU - Liu H AD - Department of Hematology, Beijing Hospital, Ministry of Health, Beijing, China. FAU - Zeng, Qiao-Zhu AU - Zeng QZ AD - Peking University People's Hospital, Peking University Institute of Hematology, Beijing, China. AD - Collaborative Innovation Center of Hematology, Peking University, Beijing, China. AD - Beijing Key Laboratory of Hematopoietic Stem Cell Transplantation, Beijing, China. AD - National Clinical Research Center for Hematologic Disease, Beijing, China. FAU - Liu, Yi AU - Liu Y AD - Department of Geriatric Hematology, Chinese PLA General Hospital, Beijing, China. FAU - Wang, Jing-Wen AU - Wang JW AD - Department of Hematology, Beijing Tongren Hospital, Capital Medical University, Beijing, China. FAU - Wang, Wen-Sheng AU - Wang WS AD - Department of Hematology, Peking University First Hospital, Beijing, China. FAU - Jia-Feng AU - Jia-Feng AD - Department of Hematology, Peking University Shenzhen Hospital, Shenzhen, China; and. FAU - Zhou, He-Bing AU - Zhou HB AD - Department of Hematology, The Affiliated Beijing Luhe Hospital of Capital Medical University, Beijing, China. FAU - Huang, Qiu-Sha AU - Huang QS AD - Peking University People's Hospital, Peking University Institute of Hematology, Beijing, China. AD - Collaborative Innovation Center of Hematology, Peking University, Beijing, China. AD - Beijing Key Laboratory of Hematopoietic Stem Cell Transplantation, Beijing, China. AD - National Clinical Research Center for Hematologic Disease, Beijing, China. FAU - He, Yun AU - He Y AD - Peking University People's Hospital, Peking University Institute of Hematology, Beijing, China. AD - Collaborative Innovation Center of Hematology, Peking University, Beijing, China. AD - Beijing Key Laboratory of Hematopoietic Stem Cell Transplantation, Beijing, China. AD - National Clinical Research Center for Hematologic Disease, Beijing, China. FAU - Fu, Hai-Xia AU - Fu HX AD - Peking University People's Hospital, Peking University Institute of Hematology, Beijing, China. AD - Collaborative Innovation Center of Hematology, Peking University, Beijing, China. AD - Beijing Key Laboratory of Hematopoietic Stem Cell Transplantation, Beijing, China. AD - National Clinical Research Center for Hematologic Disease, Beijing, China. FAU - Zhu, Xiao-Lu AU - Zhu XL AD - Peking University People's Hospital, Peking University Institute of Hematology, Beijing, China. AD - Collaborative Innovation Center of Hematology, Peking University, Beijing, China. AD - Beijing Key Laboratory of Hematopoietic Stem Cell Transplantation, Beijing, China. AD - National Clinical Research Center for Hematologic Disease, Beijing, China. FAU - Jiang, Qian AU - Jiang Q AD - Peking University People's Hospital, Peking University Institute of Hematology, Beijing, China. AD - Collaborative Innovation Center of Hematology, Peking University, Beijing, China. AD - Beijing Key Laboratory of Hematopoietic Stem Cell Transplantation, Beijing, China. AD - National Clinical Research Center for Hematologic Disease, Beijing, China. FAU - Jiang, Hao AU - Jiang H AD - Peking University People's Hospital, Peking University Institute of Hematology, Beijing, China. AD - Collaborative Innovation Center of Hematology, Peking University, Beijing, China. AD - Beijing Key Laboratory of Hematopoietic Stem Cell Transplantation, Beijing, China. AD - National Clinical Research Center for Hematologic Disease, Beijing, China. FAU - Chang, Ying-Jun AU - Chang YJ AD - Peking University People's Hospital, Peking University Institute of Hematology, Beijing, China. AD - Collaborative Innovation Center of Hematology, Peking University, Beijing, China. AD - Beijing Key Laboratory of Hematopoietic Stem Cell Transplantation, Beijing, China. AD - National Clinical Research Center for Hematologic Disease, Beijing, China. FAU - Xu, Lan-Ping AU - Xu LP AD - Peking University People's Hospital, Peking University Institute of Hematology, Beijing, China. AD - Collaborative Innovation Center of Hematology, Peking University, Beijing, China. AD - Beijing Key Laboratory of Hematopoietic Stem Cell Transplantation, Beijing, China. AD - National Clinical Research Center for Hematologic Disease, Beijing, China. FAU - Huang, Xiao-Jun AU - Huang XJ AUID- ORCID: 0000-0002-2145-6643 AD - Peking University People's Hospital, Peking University Institute of Hematology, Beijing, China. AD - Collaborative Innovation Center of Hematology, Peking University, Beijing, China. AD - Beijing Key Laboratory of Hematopoietic Stem Cell Transplantation, Beijing, China. AD - National Clinical Research Center for Hematologic Disease, Beijing, China. FAU - Zhang, Xiao-Hui AU - Zhang XH AUID- ORCID: 0000-0003-4730-256X AD - Peking University People's Hospital, Peking University Institute of Hematology, Beijing, China. AD - Collaborative Innovation Center of Hematology, Peking University, Beijing, China. AD - Beijing Key Laboratory of Hematopoietic Stem Cell Transplantation, Beijing, China. AD - National Clinical Research Center for Hematologic Disease, Beijing, China. LA - eng SI - ClinicalTrials.gov/NCT03304288 PT - Clinical Trial PT - Journal Article PT - Multicenter Study PT - Randomized Controlled Trial PT - Research Support, Non-U.S. Gov't PL - United States TA - Blood JT - Blood JID - 7603509 RN - 0 (Adrenal Cortex Hormones) RN - 0 (Antineoplastic Agents) RN - 0 (Immunologic Factors) RN - 4F4X42SYQ6 (Rituximab) RN - 5688UTC01R (Tretinoin) SB - IM CIN - Blood. 2022 Jan 20;139(3):307-308. PMID: 35050337 MH - Adrenal Cortex Hormones/therapeutic use MH - Adult MH - Antineoplastic Agents/administration & dosage/*therapeutic use MH - Drug Resistance MH - Drug Therapy, Combination MH - Female MH - Humans MH - Immunologic Factors/administration & dosage/*therapeutic use MH - Male MH - Middle Aged MH - Purpura, Thrombocytopenic, Idiopathic/*drug therapy MH - Recurrence MH - Rituximab/administration & dosage/*therapeutic use MH - Secondary Prevention MH - Tretinoin/administration & dosage/*therapeutic use EDAT- 2021/10/20 06:00 MHDA- 2022/03/08 06:00 CRDT- 2021/10/19 17:20 PHST- 2021/07/19 00:00 [received] PHST- 2021/10/13 00:00 [accepted] PHST- 2021/10/20 06:00 [pubmed] PHST- 2022/03/08 06:00 [medline] PHST- 2021/10/19 17:20 [entrez] AID - S0006-4971(21)01759-6 [pii] AID - 10.1182/blood.2021013393 [doi] PST - ppublish SO - Blood. 2022 Jan 20;139(3):333-342. doi: 10.1182/blood.2021013393.