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

Dmitry Rogachev National Medical Research Center of Pediatric Hematology, Oncology and Immunology, Moscow, Russian Federation. Dmitriy Rogachev National Center for pediatric hematology, oncology and immunology. Dmitriy Rogachev National Medical Center of pediatric hematology, oncology and immunology, Moscow, Russian Federation. Dmitriy Rogachev National Center for Pediatric Hematology, Oncology and Immunology, Moscow, Russian Federation. Federal Research Centre of Pediatric Hematology, Oncology and Immunology. National Research Center for Pediatric Hematology, Oncology and Immunology, Moscow, Russian Federation. Dmitry Rogachev National Medical Center of Pediatric Hematology, Oncology and Immunology, Moscow, Russian Federation. Pirogov Russian Clinical Children Hospital, Moscow, Russia, Moscow, Russian Federation. Saint-Petersburg Children City Hospital No 1, Saint Petersburg, Russian Federation. Almazov National Medical Research Center, Saint Petersburg, Russia, Saint Petersburg, Russian Federation. Krasnoyarsk Regional Clinical Hospital, Krasnoyarsk, Russian Federation. Saratov State Medical University, Saratov, Russian Federation. Samara Regional Clinical Children Hospital, Samara, Russian Federation. Perm Regional Clinical Hospital, Perm, Russian Federation. Regional Children Hospital, Ekaterinburg, Russian Federation. Regional Clinical Children's Hospital No 1, Voronezh, Russian Federation. Regional Clinical Children Hospital, Nizhniy Novgorod, Nizhniy Novgorod, Russian Federation. Piotrovich Regional Clinical Children Hospital, Khabarovsk, Khabarovsk, Russian Federation. Dmtri Rogachev Research Center for Pediatric Hematology, Oncology and Immunology, Moscow, Russian Federation.

Blood advances. 2022
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Abstract
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 and cyclosporin A 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=0.218); however, the complete response (CR) rate was significantly higher in ELTR+IST group (31% vs. 12%, p=0.027). In severity subgroups, the ORR was 89% vs. 57% (p=0.028) in favor of IST+ELTR in SAA, but it did not differ in patients with vSAA (52% vs. 50%, p=0.902). At 6 months after the crossover, 61% of initial ELTR(-) patients achieved a response compared to 17% of initial ELTR(+) patients (p=0.016). No significant difference in ELTR+IST and IST groups was observed in the 3-year OS (89% vs. 91%, p=0.673) or the 3-year EFS (53% vs. 41%, p=0.326). 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. Clinicaltrials.gov #NCT03413306.
Study details
Language : eng
Credits : Bibliographic data from MEDLINE®/PubMed®, a database of the U.S. National Library of Medicine