Low Dose of Eltrombopag with Long-Term Response in Children with Severe Aplastic Anemia, Less Morbi-Mortality Better Quality of Life

Refractory severe aplastic anemia (RSAA) has poor prognosis. Eltrombopag is a therapeutic option in patients that failed to ATG+CyA; in countries with poor outcomes their use is limited. Described experience with low doses of Eltrombopag 25mg/d.

CASE 1. Female 4 yo diagnosis SAA not responded after 2 cycles ATG, the first one with mesterolone for 6 months, continued pancytopenic with multiple transfusions (every 2/3 weeks) complicated by iron overload, and serious infections; presented CNS infection with secondary spastic paralysis, due chelation therapy development 2 cholestasis outbreak. Eltrombopag was started 25mg/d, after the first month, no more transfusion was needed with increase of neutrophils and platelet counts. After 7 months Eltrombopag for economic reasons was stopped with partial response. Hb >10g/dL, CAN >1000 m3 & platelets 99 X103. For 4 years; until now; she is doing well, no infections and no transfusions needed.

CASE 2. Male 2 yo, SAA not responded ATG course, he was inpatient during 8 months with multiple infections, bleeding complications and consequence transfusion was needed. Iron overload required iron chelation therapy, the medical orders and CyA was irregularly administrated, the parents not accept considered BMT, so Eltrombopag 25mg/d was started, 6 weeks later no more transfusion was required, the bleeding stopped and no mores infections were observed; after 7 months had a partial response Hb > 11g/dL CAN > 1000m3 & platelets 45 x103, and Eltrombopag was stopped.

This two patients use Eltrombopag as compassionate use, with good clinical results, in spite of low dose to get a partial hematological response.

Low dose of Eltrombopag is useful to induce partial response rapidily with an excellent clinical results even when it is discontinued.

Actually all patients with new SAA diagnostic are register in our Institution in a prospective protocol to receive ATG+CyA and start Eltrombopag 50mg/d with increase according to the response.

The low dose could be a good option for patients with refractory aplastic anemia to get a partial response in development countries with less morbidity-mortality and a better quality of life.