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Eltrombopag in children with severe aplastic anemia
Harry Lesmana1,2, Timothy Jacobs3, Michelle Boals1
1Department of Hematology, St. Jude Children's Research Hospital, Memphis, Tennessee, USA.
Insights
Eltrombopag added to standard immunosuppressive therapy (IST) improved hematological response in children with severe aplastic anemia (SAA). This combination therapy was well-tolerated, showing promise for pediatric SAA treatment.
Area of Science:
- Pediatric Hematology
- Aplastic Anemia Research
- Immunosuppressive Therapy
Background:
- Standard therapy for pediatric severe aplastic anemia (SAA) lacking HLA-identical siblings includes horse antithymocyte globulin and cyclosporine.
- Eltrombopag, a thrombopoietin receptor agonist, is approved for SAA patients aged 2 years and older, but pediatric data are limited.
Purpose of the Study:
- To evaluate the safety and efficacy of eltrombopag combined with standard immunosuppressive therapy (IST-Epag) versus standard IST alone (IST-Std) in pediatric SAA patients.
- To assess objective response (OR) and complete response (CR) rates at 6 and 12 months post-treatment.
Main Methods:
- Retrospective study of pediatric SAA patients (≤18 years) diagnosed between 2000-2018.
- Patients received either IST-Std or IST-Epag (upfront or after failed IST).
- Primary outcome: OR (partial and complete response) at 6 and 12 months.
Main Results:
- IST-Epag showed high OR (100% at 12 months) and CR (29% at 12 months) in upfront treatment, comparable to IST-Std (OR 100%, CR 58% at 12 months).
- Two patients with prior IST failure did not respond to eltrombopag.
- No significant differences in infections; one patient had transient transaminitis. No changes in PNH clone size or cytogenetics were observed.
Conclusions:
- Addition of eltrombopag to standard IST is well-tolerated and yields satisfactory hematological response in pediatric SAA.
- Further research with larger cohorts and longer follow-up is needed to confirm response durability.
Background:
Immunosuppressive therapy with horse antithymocyte globulin and cyclosporine currently remains the standard therapy for children with severe aplastic anemia (SAA) who lack human leukocyte antigen (HLA)-identical sibling. The thrombopoietin receptor agonist eltrombopag has been recently approved for SAA patients 2 years and older. However, there are limited data on its safety and efficacy in pediatric cohorts.
Methods:
We conducted a retrospective study of patients ≤18 years old consecutively diagnosed with SAA between 2000 and 2018. Patients received either standard immunosuppressive therapy (IST-Std) or IST with eltrombopag (IST-Epag). The primary outcome was the objective response (OR), including partial and complete response (CR), at 6 and 12 months after starting therapy.
Results:
We identified 16 patients receiving IST-Std and nine IST-Epag treatment (seven of nine as upfront therapy and two of seven after previously failed IST). The OR at 6 and 12 months in IST-Std arm was 71% and 100%, with CR in 29% and 58%, respectively. Seven patients receiving upfront IST-Epag had OR at 6 and 12 months, with two of seven (29%) achieving CR at 6 and 12 months. Two patients who previously failed standard IST did not respond to eltrombopag. No significant differences were observed in both cohorts with regard to infections. One IST-Epag-treated patient developed transient grade 3 transaminitis. Finally, no changes in paroxysmal nocturnal hemoglobinuria (PNH) clone size and cytogenetic abnormalities were seen in either cohort.
Conclusion:
The addition of eltrombopag to standard IST was well tolerated and resulted in satisfactory hematological response at 6 and 12 months in this single-institution experience. A larger cohort with longer follow-up is required to assess response durability.
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