Objective To evaluate efficacy, complications and contributing factors for response of immunosuppressive therapy(IST) and allogenic hematopoietic stem cell transplantation(allo-HSCT) in children with acquired aplastic anemia; to explore optimal therapy for different clinical types of aplastic anemia.

Methods 230 children diagnosed with aplastic anemia accepted immunosuppressive therapy from January 1, 2006 to July 15, 2019 in Department of pediatrics, Sun Yat-sen memorial hospital and Department of pediatrics, The seventh affiliated hospital, Sun Yat-sen University, the response rate for 3, 6, 9, 12months for IST, complications,predictors of response and overall survival were analyzed and response rates were compared according to clinical types.

Results 230 children were followed with median time 47.3 months. Among all AA patients, there are 67 NSAA patients, 95 SAA patients and 68 VSAA patients. 214 patients had received IST initially, due to economic, medical insurance related factors or lack of well-matched donors at the time, and 16 patients had undergone allo-HSCT initially. And 21 patients , who had failed to had a CR/PR responds to IST, received allo-HSCT as a salvage regimen. The overall survival(OS) rate of all patients was 79.6±2.9% with a 46.2 months median follow-up.

For those who had received IST therapy overall CR rate was 27.1%(58/214), PR rate 37.4%(80/214), therefore overall response (CR+PR)rate was 64.5%(138/214). CR rate at 3,6,9,12 months were 7.9%, 15.9%, 16.4%, 27.1%, respectively; meanwhile PR rate 3,6,9,12 monthsatwere 28.0%, 34.1%, 34.1%, 37.4%, respectively. When we termed"transplantation and death"as an even, the transplantation-free survival(TFS) for NSAA was 90.7±4.0%, significantly superior to SAA(59.7±6.5%) and VSAA(50.5±7.5%). As for complications, serum sickness and infection are most commonly seen.

Among 37 patients who had recieved allo-HSCT, 33 patients had long term CR, while 2 died of infection, 1 of extensive aGVHD, 1 of thrombotic microangiopathy. 21 patients who underwent HSCT as a salvage regimen had 85.4±7.8% OS with a 55.6 months median follow-up, and 16 patients who recieved HSCT as initial treatment had 93.8±6.1% OS with a 69.1 months median follow-up.

Conclusions In children with AA, overall response rate to IST goes higher over time with a highest response rate 64.5% at 12 month. TFS for NSAA IST-group is significantly superior to SAA and VSAA IST-group. However, well-matched allo-HSCT still achieve nearly 90% OS as both initial or salvage treatment. Hence, we agure that IST is a pratical treatment for pediatric AA, especially NSAA, in conditions of economic status and poor medical insurance, and HSCT is still a cure for those who failed IST.

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Disclosures

No relevant conflicts of interest to declare.

Topics:
acquired aplastic anemia, china, pediatrics, therapeutic immunosuppression, transplantation, treatment outcome, inappropriate sinus tachycardia, hematopoietic stem cell transplantation, allopurinol, follow-up

Author notes

*

Asterisk with author names denotes non-ASH members.

© 2019 by The American Society of Hematology
2019
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