Some pediatric patients with newly diagnosed acquired aplastic anemia may be more likely to have a complete response with immunosuppressive treatment combined with eltrombopag (ELTR) compared with immunosuppression alone, according to research published in Blood Advances. The 3-year overall survival rates, however, do not appear to be affected.
The standards of care for aplastic anemia are allogeneic hematopoietic stem cell transplantation (HSCT) or immunosuppression with horse antithymocyte globulin and cyclosporine. Early data suggest that adding ELTR to immunosuppression may be effective.
For this randomized phase 2 study (ClinicalTrials.gov Identifier: NCT03413306), researchers compared the safety and efficacy of immunosuppression plus ELTR vs immunosuppression alone among pediatric patients with newly diagnosed severe or very severe aplastic anemia (SAA and vSAA, respectively).
Overall, 98 patients were enrolled and treated on this study; 49 were randomly assigned to the ELTR group while 49 were assigned to receive immunosuppression only.
At baseline, in the ELTR and immunosuppression-only groups, the median ages were 10.5 and 8.7 years, 71.4% and 61.2% of patients were male sex, and 36.7% and 42.9% had SAA within their respective group while 63.3% and 57.1% had vSAA, respectively. Where there was no response for 4 months, group crossover was permitted.
Overall response rates (ORR) at 4 months were similar between the 2 groups (65% in the ELTR group vs 53% in the control group; P =.218), though the complete response rate was higher in the ELTR group (31% vs 12%, respectively; P =.027).
Patients with SAA, furthermore, had an improved ORR in the ELTR group (89% vs 57% with immunosuppression alone; P =.028), although no ORR difference was noted among patients with vSAA.
The 3-year overall survival and event-free survival rates, however, were similar between the 2 groups (P =.673 and P =.326, respectively).
“However, these findings do not change the current algorithm of SAA treatment in children where HSCT from [a matched sibling donor] remains the first-line option, and the use of matched unrelated donor HSCT upfront is supported based on the ability to provide the graft within 3 to 4 months of diagnosis,” the authors noted in their report.
Disclosure: Some study authors declared affiliations with biotech, pharmaceutical, or device companies. Please see the original reference for a full list of authors’ disclosures.
Goronkova O, Novichkova G, Salimova T, et al. Efficacy of combined immunosuppression with or without eltrombopag in children with newly diagnosed aplastic anemia. Blood Adv. 2023;7(6):953-962. doi:10.1182/bloodadvances.2021006716