Acquired aplastic anaemia in children – Optimal outcomes depend on optimal delivery of immunosuppressive therapy

摘要

Acquired aplastic anemia (AA) is a haematological emer- gency in children and we have gained enormous knowledge about the pathogenesis and immune mediated self-destruction of stem cells and immunosuppressive therapy. Due to its rarity, we have limited data of this entity, especially, its course and outcome in resource limited countries. Patients and methods: The case records of children who had been diag- nosed to have AA since 2002 at our centre were reviewed. The diagnosis of AA was made in children with pancytopenia without organomegaly and confirmed by hypocellular bone marrow showing decreased expression of all three hematopoietic lineages. Clinical findings including dysmorphism, thumb abnormalities, congenital malformations and skin hyperpigmen- tation were noted down. Mitomycin C induced chromosomal instability to rule out Fanconi anaemia, Ham's test to look for paroxysmal nocturnal haemoglobinuria and chromosomal analysis to look for complex karyo- typic abnormalities seen in myelodysplastic syndrome were documented in all patients. After initial stabilization, the option of haematopoietic stem cell trans- plantation (HSCT) was offered to all those who had matched related do- nors. If not, immunosuppressive therapy was delivered within twomonths from diagnosis after the child was infection free. A combination of 40mg/ kg/day of Horse derived antithymocyte globulin (ATGAM) and methyl- prednisolone at 2 mg/kg/day were started for the first 4 days through a central venous catheter. Oral cyclosporine and G-CSF were started on the fifth day and since 2015 Eltrombopag was added to the IST regimen. Revolade and GCSF were continued for about 8 to 12 weeks and cyclo- sporine from 6 to 12 months. All patient received Voriconazole and Acyclovir prophylaxis till neutrophil recovery to 1000. Results: A total of 109 children had been diagnosed as AA, of whom, 42 had acquired AA. Themean age of childrenwith acquired AAwas 8.16 years (2-15 yrs). Boys were twice more commonly affected (ratio M: F ? 2:1). Details are listed in table 1. Of the 41, 21 underwent HSCT as they had fully matched related donor and 20 children received IST. Of the 20 children who received ATG, 10 children (70%) had complete remission in a median duration of 78 days. Two of the three non responderswere treated with second IST and all three succumbed to the illness. One child developed acute myeloid leukaemia after achieving partial remission and was salvaged with HSCT, and 1 more non responder had an unrelated HSCT and is doing well. One child has suffered a relapse after a durable remission of over 18 months off immunosuppression. The 21 children who have been transplanted have an overall survival rate of 77 %. Conclusion: Immunosuppressive regimen including Horse ATGAM, Methyl prednisolone, Cyclosporine, GCSF and Eltrombopag along with adequate and meticulous supportive care including neutropenic care, prompt infection management and the use of irradiated and leukode- pleted blood products results in a 70% response rate and should be offered to all children with aplastic anaemia with no matched family donor. Theaddition of Elthrombopag has resulted in early recovery of all three cell lines and must be added to the IST protocol in children. RBC-1_V1.2 DISCORDANT BEATS e A PROSPECTIVE STUDY OF CARDIAC MRI TO ASSESS IRON OVERLOAD IN PATIENTS WITH THALASSAEMIA MAJOR S. Divya 1, M. Venaktadesiaklu 2, V. Mythili 2, Bharathi 2, U. Ramya 1, V.S. Venakateswaran1, Ravikanth Balaji 1, S. Premsekar 2, Revathi Raj 1. 1Department of Paediatric Haematology, Oncology, Blood and Marrow Transplantation, Apollo Speciality Hosital, Chennai, India; 2VHS Thalassaemia Centre, Taramani, Chennai, India Introduction: Advances in blood banking has ensured optimal delivery of monthly transfusion needed to save the lives of about 10,000 new thal- assaemia children born each year in our country. The burden of iron che- lation has now increased as these children survive into their second and third decade of life. Cardiac failure secondary to myocardial haemoside- rosis is the most common cause of mortality in these patients. Our study has been done to evaluate the prevalence and severity of cardiac ironload in children with beta thalassemia major and the effectiveness of serum ferritin as a marker of iron overload. Patients and Background: Acquired aplastic anemia (AA) is a haematological emergency in children and we have gained enormous knowledge about the pathogenesis and immune mediated self-destruction of stem cells and immunosuppressive therapy. Due to its rarity, we have limited data of this entity, especially, its course and outcome in resource limited countries.