Böske Anne-Kathrin, Sander Annette, Sykora Karl-Walter, Baumann Ulrich, Pfister Eva-Doreen
Clinic for Pediatric Nephrology, Hepatology and Metabolic Disorders, Hannover Medical School, Hannover, Germany.
Pediatric hematology and oncology, Hannover Medical School, Hannover, Germany.
Klin Padiatr. 2020 May;232(3):151-158. doi: 10.1055/a-1108-1553. Epub 2020 Mar 19.
Children with idiopathic acute liver failure (IALF) are at a high risk of developing life-threatening bone marrow failure (BMF). The aim of the study was to describe the development, therapy and prognosis of this hepatitis-associated aplastic anaemia (HAAA) in comparison to isolated acquired aplastic anaemia.
We retrospectively found 18 patients (9 female) of HAAA between 1984 and 2017 with an age of 1.4-16.4 years. Fifteen of them fulfilled the SAA criteria, 3 had a bone marrow hypoplasia. Eleven of these children received liver transplantation (LTx) (these were 11 of 42 (26%) children receiving LTx for IALF), 6 patients recovered without LTx. The first signs of BMF, thrombocytopaenia and leucocytopaenia, occurred before LTx in all cases. During the follow-up period 8 patients reached haematological remission, 6 received haematopoietic stem cell transplantation (HSCT). Seven children died in a median of 304 days after the first symptoms mostly because of bleedings and infections. To date, extensive investigations failed to detect a genetically, viral or immunological aetiology. No AA was diagnosed in the 41 patients receiving liver transplants during the same period for ALF of known aetiology. As a comparison group, we collected the data of patients with isolated SAA. 73% achieved a remission after Immunosuppressive therapy (IST) without HSCT, and none of them died during the follow-up period.
Blood counts should be examined early and regularly (0-22 days after onset) in patients with IALF. Aggressive treatment with LTx, IST and HSCT appears to improve the prognosis.
特发性急性肝衰竭（IALF）患儿发生危及生命的骨髓衰竭（BMF）风险很高。本研究旨在描述这种与肝炎相关的再生障碍性贫血（HAAA）相较于孤立性获得性再生障碍性贫血的发生、治疗及预后情况。
我们回顾性分析了1984年至2017年间18例HAAA患者（9例女性），年龄在1.4 - 16.4岁之间。其中15例符合重型再生障碍性贫血（SAA）标准，3例骨髓发育不全。这些患儿中有11例接受了肝移植（LTx）（这是42例因IALF接受LTx患儿中的11例（26%）），6例未接受LTx而康复。所有病例中，BMF的首发症状血小板减少和白细胞减少均出现在肝移植之前。在随访期间，8例患者达到血液学缓解，6例接受了造血干细胞移植（HSCT）。7例患儿在出现首发症状后的中位304天死亡，主要原因是出血和感染。迄今为止，广泛的调查未能发现遗传、病毒或免疫病因。同期41例因已知病因的急性肝衰竭接受肝移植的患者中未诊断出再生障碍性贫血。作为对照组，我们收集了孤立性SAA患者的数据。73%的患者在未进行HSCT的免疫抑制治疗（IST）后实现缓解，且随访期间无一人死亡。
IALF患者应在发病早期（发病后0 - 22天）尽早且定期检查血常规。积极采用LTx、IST和HSCT治疗似乎可改善预后。
