PMID- 38362793 OWN - NLM STAT- Publisher LR - 20240216 IS - 1365-2141 (Electronic) IS - 0007-1048 (Linking) DP - 2024 Feb 16 TI - Efficacy and safety of avatrombopag in Chinese children with persistent and chronic primary immune thrombocytopenia: A multicentre observational retrospective study in China. LID - 10.1111/bjh.19342 [doi] AB - Avatrombopag (AVA) is a novel thrombopoietin receptor agonist (TPO-RA) that has been recently approved as a second-line therapy for immune thrombocytopenia (ITP) in adults; however, its safety and efficacy data in children are lacking. Here, we demonstrated the efficacy and safety of AVA as second-line therapy in children with ITP. A multicentre, retrospective, observational study was conducted in children with persistent or chronic ITP who did not respond to or relapsed from previous treatment and were treated with AVA for at least 12 weeks between August 2020 and December 2022. The outcomes were the responses (defined as achieving a platelet count >/=30 x 10(9) /L, twofold increase in platelet count from baseline and absence of bleeding), including rapid response within 4 weeks, sustained response at weeks 12 and 24, bleeding control and adverse events (AEs). Thirty-four (18 males) patients with a mean age of 6.3 (range: 1.9-15.3) years were enrolled. The median number of previous treatment types was four (range: 1-6), and 41.2% patients switched from other TPO-RAs. Within 4 weeks, overall response (OR) was achieved in 79.4% patients and complete response (CR, defined as a platelet count >/=100 x 10(9) /L and the absence of bleeding) in 67.7% patients with a median response time of 7 (range: 1-27) days. At 12 weeks, OR was achieved in 88.2%, CR in 76.5% and sustained response in 44% of patients. At 24 weeks, 22/34 (64.7%) patients who achieved a response and were followed up for 24 weeks were evaluated; 12/22 (54.55%) achieved a sustained response. During AVA therapy, median platelet counts increased by week 1 and were maintained throughout the treatment period. The proportion of patients with grade 1-3 bleeding decreased from 52.95% at baseline to 2.94% at 12 weeks, while concomitant ITP medications decreased from 36.47% at baseline to 8.82% at 12 weeks, with only 9 (26.47%) patients receiving rescue therapy 23 times within 12 weeks. There were 61.8% patients with 59 AEs: 29.8% with Common Terminology Criteria for Adverse Events grade 1 and the rest with grade 2. These findings show that AVA could achieve a rapid and sustained response in children with persistent or chronic ITP as a second-line treatment, with good clinical bleeding control and reduction of concomitant ITP therapy, without significant AEs. CI - (c) 2024 British Society for Haematology and John Wiley & Sons Ltd. FAU - Wang, Zhifa AU - Wang Z AUID- ORCID: 0000-0001-7363-5101 AD - Beijing Key Laboratory of Pediatric Hematology-Oncology, National Key Discipline of Pediatrics (Capital Medical University), Key Laboratory of Major Diseases in Children, Hemophilia Comprehensive Care Centre, Hematology Centre, National Centre for Children's Health, Ministry of Education, Beijing Children's Hospital, Capital Medical University, Beijing, China. FAU - Zhang, Aijun AU - Zhang A AUID- ORCID: 0000-0002-6117-4560 AD - Department of Pediatrics, Qilu Hospital of Shandong University, Jinan, China. FAU - Xu, Zhongjin AU - Xu Z AD - Department of Hematology, Jiangxi Provincial Children's Hospital, Nanchang, China. FAU - Wang, Nan AU - Wang N AD - Department of Pharmacy, Beijing Children's Hospital, Capital Medical University, Beijing, China. FAU - Zhang, Jialu AU - Zhang J AUID- ORCID: 0000-0002-7624-9430 AD - Beijing Key Laboratory of Pediatric Hematology-Oncology, National Key Discipline of Pediatrics (Capital Medical University), Key Laboratory of Major Diseases in Children, Hemophilia Comprehensive Care Centre, Hematology Centre, National Centre for Children's Health, Ministry of Education, Beijing Children's Hospital, Capital Medical University, Beijing, China. FAU - Meng, Jinxi AU - Meng J AD - Beijing Key Laboratory of Pediatric Hematology-Oncology, National Key Discipline of Pediatrics (Capital Medical University), Key Laboratory of Major Diseases in Children, Hemophilia Comprehensive Care Centre, Hematology Centre, National Centre for Children's Health, Ministry of Education, Beijing Children's Hospital, Capital Medical University, Beijing, China. FAU - Dong, Shuyue AU - Dong S AD - Beijing Key Laboratory of Pediatric Hematology-Oncology, National Key Discipline of Pediatrics (Capital Medical University), Key Laboratory of Major Diseases in Children, Hemophilia Comprehensive Care Centre, Hematology Centre, National Centre for Children's Health, Ministry of Education, Beijing Children's Hospital, Capital Medical University, Beijing, China. FAU - Ma, Jingyao AU - Ma J AD - Beijing Key Laboratory of Pediatric Hematology-Oncology, National Key Discipline of Pediatrics (Capital Medical University), Key Laboratory of Major Diseases in Children, Hemophilia Comprehensive Care Centre, Hematology Centre, National Centre for Children's Health, Ministry of Education, Beijing Children's Hospital, Capital Medical University, Beijing, China. FAU - Hu, Yu AU - Hu Y AD - Beijing Key Laboratory of Pediatric Hematology-Oncology, National Key Discipline of Pediatrics (Capital Medical University), Key Laboratory of Major Diseases in Children, Hemophilia Comprehensive Care Centre, Hematology Centre, National Centre for Children's Health, Ministry of Education, Beijing Children's Hospital, Capital Medical University, Beijing, China. FAU - Ouyang, Juntao AU - Ouyang J AD - Hematologic Disease Laboratory, Hematology Centre, Beijing Key Laboratory of Pediatric Hematology-Oncology, National Key Discipline of Pediatrics (Capital Medical University), Key Laboratory of Major Diseases in Children, National Centre for Children's Health, Ministry of Education, Beijing Pediatric Research Institute, Beijing Children's Hospital, Capital Medical University, Beijing, China. FAU - Chen, Zhenping AU - Chen Z AD - Hematologic Disease Laboratory, Hematology Centre, Beijing Key Laboratory of Pediatric Hematology-Oncology, National Key Discipline of Pediatrics (Capital Medical University), Key Laboratory of Major Diseases in Children, National Centre for Children's Health, Ministry of Education, Beijing Pediatric Research Institute, Beijing Children's Hospital, Capital Medical University, Beijing, China. FAU - An, Qi AU - An Q AD - Department of Hematology, Xuzhou Children's Hospital, Xuzhou Medical University, Xuzhou, China. FAU - Cheng, Xiaoling AU - Cheng X AUID- ORCID: 0000-0002-0048-2658 AD - Department of Pharmacy, Beijing Children's Hospital, Capital Medical University, Beijing, China. FAU - Wu, Runhui AU - Wu R AUID- ORCID: 0000-0003-4030-209X AD - Beijing Key Laboratory of Pediatric Hematology-Oncology, National Key Discipline of Pediatrics (Capital Medical University), Key Laboratory of Major Diseases in Children, Hemophilia Comprehensive Care Centre, Hematology Centre, National Centre for Children's Health, Ministry of Education, Beijing Children's Hospital, Capital Medical University, Beijing, China. LA - eng GR - 81970111/National Natural Science Foundation of China/ GR - PX2023044/Beijing Municipal Administration of Hospitals Incubating Program/ GR - 2022-2Z-2099/Beijing Municipal Health Commission, Capital's Funds for Health Improvement and Research/ PT - Journal Article DEP - 20240216 PL - England TA - Br J Haematol JT - British journal of haematology JID - 0372544 SB - IM OTO - NOTNLM OT - avatrombopag OT - children OT - persistent or chronic immune thrombocytopenia OT - primary immune thrombocytopenia EDAT- 2024/02/16 06:43 MHDA- 2024/02/16 06:43 CRDT- 2024/02/16 05:31 PHST- 2024/01/16 00:00 [revised] PHST- 2023/10/12 00:00 [received] PHST- 2024/02/06 00:00 [accepted] PHST- 2024/02/16 06:43 [medline] PHST- 2024/02/16 06:43 [pubmed] PHST- 2024/02/16 05:31 [entrez] AID - 10.1111/bjh.19342 [doi] PST - aheadofprint SO - Br J Haematol. 2024 Feb 16. doi: 10.1111/bjh.19342.