دورية أكاديمية
أعرض في EDS

Efficacy of combined immunosuppression with or without eltrombopag in children with newly diagnosed aplastic anemia.

التفاصيل البيبلوغرافية
العنوان: Efficacy of combined immunosuppression with or without eltrombopag in children with newly diagnosed aplastic anemia.
المؤلفون: Goronkova O; Dmitry Rogachev National Medical Research Center of Pediatric Hematology, Oncology, and Immunology, Moscow, Russia., Novichkova G; Dmitry Rogachev National Medical Research Center of Pediatric Hematology, Oncology, and Immunology, Moscow, Russia., Salimova T; Dmitry Rogachev National Medical Research Center of Pediatric Hematology, Oncology, and Immunology, Moscow, Russia., Kalinina I; Dmitry Rogachev National Medical Research Center of Pediatric Hematology, Oncology, and Immunology, Moscow, Russia., Baidildina D; Dmitry Rogachev National Medical Research Center of Pediatric Hematology, Oncology, and Immunology, Moscow, Russia., Petrova U; Dmitry Rogachev National Medical Research Center of Pediatric Hematology, Oncology, and Immunology, Moscow, Russia., Antonova K; Dmitry Rogachev National Medical Research Center of Pediatric Hematology, Oncology, and Immunology, Moscow, Russia., Sadovskaya M; Dmitry Rogachev National Medical Research Center of Pediatric Hematology, Oncology, and Immunology, Moscow, Russia., Suntsova E; Dmitry Rogachev National Medical Research Center of Pediatric Hematology, Oncology, and Immunology, Moscow, Russia., Evseev D; Dmitry Rogachev National Medical Research Center of Pediatric Hematology, Oncology, and Immunology, Moscow, Russia., Matveev V; Dmitry Rogachev National Medical Research Center of Pediatric Hematology, Oncology, and Immunology, Moscow, Russia., Venyov D; Dmitry Rogachev National Medical Research Center of Pediatric Hematology, Oncology, and Immunology, Moscow, Russia., Khachatryan L; Dmitry Rogachev National Medical Research Center of Pediatric Hematology, Oncology, and Immunology, Moscow, Russia., Litvinov D; Dmitry Rogachev National Medical Research Center of Pediatric Hematology, Oncology, and Immunology, Moscow, Russia., Pshonkin A; Dmitry Rogachev National Medical Research Center of Pediatric Hematology, Oncology, and Immunology, Moscow, Russia., Ovsyannikova G; Dmitry Rogachev National Medical Research Center of Pediatric Hematology, Oncology, and Immunology, Moscow, Russia., Kotskaya N; Dmitry Rogachev National Medical Research Center of Pediatric Hematology, Oncology, and Immunology, Moscow, Russia., Gobadze D; Dmitry Rogachev National Medical Research Center of Pediatric Hematology, Oncology, and Immunology, Moscow, Russia., Olshanskaya Y; Dmitry Rogachev National Medical Research Center of Pediatric Hematology, Oncology, and Immunology, Moscow, Russia., Popov A; Dmitry Rogachev National Medical Research Center of Pediatric Hematology, Oncology, and Immunology, Moscow, Russia., Raykina E; Dmitry Rogachev National Medical Research Center of Pediatric Hematology, Oncology, and Immunology, Moscow, Russia., Mironenko O; Dmitry Rogachev National Medical Research Center of Pediatric Hematology, Oncology, and Immunology, Moscow, Russia., Voronin K; Dmitry Rogachev National Medical Research Center of Pediatric Hematology, Oncology, and Immunology, Moscow, Russia., Purbueva B; Pirogov Russian Clinical Children's Hospital, Moscow, Russia., Boichenko E; St. Petersburg Children's City Hospital No 1, St. Petersburg, Russia., Dinikina Y; Almazov National Medical Research Center, St. Petersburg, Russia., Guseynova E; Krasnoyarsk Regional Clinical Hospital, Krasnoyarsk, Russia., Sherstnev D; Shustov University Clinical Hospital No 3 of Razumovsky Saratov State Medical University, Saratov, Russia., Kalinina E; Samara Regional Clinical Children's Hospital, Samara, Russia., Mezentsev S; Perm Regional Clinical Hospital, Perm, Russia., Streneva O; Ekaterinburg Regional Clinical Children's Hospital, Ekaterinburg, Russia., Yudina N; Voronezh Regional Clinical Children's Hospital No 1, Voronezh, Russia., Plaksina O; Regional Clinical Children's Hospital, Nizhniy Novgorod, Russia., Erega E; Piotrovich Regional Clinical Children's Hospital, Khabarovsk, Russia., Maschan M; Dmitry Rogachev National Medical Research Center of Pediatric Hematology, Oncology, and Immunology, Moscow, Russia., Maschan A; Dmitry Rogachev National Medical Research Center of Pediatric Hematology, Oncology, and Immunology, Moscow, Russia.
المصدر: Blood advances [Blood Adv] 2023 Mar 28; Vol. 7 (6), pp. 953-962.
نوع المنشور: Randomized Controlled Trial; Journal Article
اللغة: English
بيانات الدورية: Publisher: American Society of Hematology Country of Publication: United States NLM ID: 101698425 Publication Model: Print Cited Medium: Internet ISSN: 2473-9537 (Electronic) Linking ISSN: 24739529 NLM ISO Abbreviation: Blood Adv Subsets: MEDLINE
أسماء مطبوعة: Original Publication: Washington, DC : American Society of Hematology, [2016]-
مواضيع طبية MeSH: Immunosuppressive Agents*/adverse effects , Anemia, Aplastic*/diagnosis , Anemia, Aplastic*/drug therapy, Humans ; Treatment Outcome ; Immunosuppression Therapy
مستخلص: We compared the efficacy and safety of eltrombopag (ELTR) combined with immunosuppressive therapy (IST) and IST alone in treatment-naïve children with severe (SAA) and very severe (vSAA) aplastic anemia. Ninety-eight pediatric patients were randomized to receive horse antithymocyte globulin (hATG) and cyclosporin A (CsA) with (n = 49) or without (n = 49) ELTR. The primary endpoint was the overall response rate (ORR) at 4 months. After 4 months, nonresponders were crossed over to the alternative group. In all patients, the ORR in ELTR + IST and IST groups was similar (65% vs 53%; P = .218); however, the complete response (CR) rate was significantly higher in the ELTR + IST group (31% vs 12%; P = .027). In severity subgroups, the ORR was 89% vs 57% (P = .028) in favor of IST + ELTR in SAA, but it did not differ in patients with vSAA (52% vs 50%; P = .902). At 6 months after the crossover, 61% of initial ELTR(-) patients achieved a response compared with 17% of initial ELTR(+) patients (P = .016). No significant difference in ELTR + IST and IST groups was observed in the 3-year overall survival (OS) (89% vs 91%; P = .673) or the 3-year event-free survival (EFS) (53% vs 41%; P = .326). There was no unexpected toxicity related to ELTR. Adding ELTR to standard IST was well tolerated and increased the CR rate. The greatest benefit from ELTR combined with IST was observed in patients with SAA but not in those with vSAA. The second course of IST resulted in a high ORR in initial ELTR(-) patients who added ELTR and had limited efficacy among patients who received ELTR upfront. This trial was registered at Clinicaltrials.gov as #NCT03413306.
(© 2023 by The American Society of Hematology. Licensed under Creative Commons Attribution-NonCommercial-NoDerivatives 4.0 International (CC BY-NC-ND 4.0), permitting only noncommercial, nonderivative use with attribution. All other rights reserved.)
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سلسلة جزيئية: ClinicalTrials.gov NCT03413306
المشرفين على المادة: 0 (Immunosuppressive Agents)
S56D65XJ9G (eltrombopag)
تواريخ الأحداث: Date Created: 20220421 Date Completed: 20230321 Latest Revision: 20230323
رمز التحديث: 20240628
مُعرف محوري في PubMed: PMC10027512
DOI: 10.1182/bloodadvances.2021006716
PMID: 35446936
قاعدة البيانات: MEDLINE
الوصف
تدمد:2473-9537
DOI:10.1182/bloodadvances.2021006716