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Master Thesis

Place des agents hypométhylants dans les hémopathies malignes de l’enfant : étude rétrospective de l’administration d’Azacitidine dans trois centres d’hématologie d’Île-de-France

Abstract : Purpose: data on the interest of hypomethylating agents in pediatric hematologic malignancies are missing. Administration of Azacitidine (Aza), regarding indication, efficacy and toxicity, in a cohort of children and young adults, has been studied. Methods: we retrospectively collected data of patients aged under 25 years old, treated with Aza in 3 university hospitals, between 2009 and 2019. Results: 32 patients (median age = 11.7 y), treated with 35 courses of Aza, have been included. Diagnosis were: 16 acute myeloid leukemias (AML), 6 juvenile myelomonocytic leukemias (JMML), 7 myelodysplastic syndromes (MDS), 2 mixed-type acute leukemias (AL), and 1 sarcoma. 137 cycles have been administrated and were well tolerated. At the end of course 23/35 treatments with Aza had failed. 24/32 patients died, and overall survival at 5 years was 23%, IC95% [11 ;48]. No difference on overall survival according to diagnosis has been observed (p=0,85). Four patients with refractory AML achieved remission prior to bone marrow transplant and were alive in complete remission (median follow-up = 29.5 months). 3/6 AL treated with Sorafenib and Aza, and 2/3 JMML with PTPN11 mutation have presented a response. 1 MDS presented an extended stable disease after palliative treatment with Aza. Conclusion: Aza was well tolerated, but overall outcome was poor, probably due to the fact that most of the patients have been heavily pretreated. Further international prospective investigations are needed in order to confirm some of the results, and better define pediatric patients who might benefit from hypomethylating agents, in particular in combination with kinase inhibitors or proapoptotic agents.
Keywords : MDS JMML AML Childhood
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Master Thesis
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https://dumas.ccsd.cnrs.fr/dumas-03190565
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Submitted on : Thursday, September 2, 2021 - 3:30:47 PM
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Émilie Saucier. Place des agents hypométhylants dans les hémopathies malignes de l’enfant : étude rétrospective de l’administration d’Azacitidine dans trois centres d’hématologie d’Île-de-France. Médecine humaine et pathologie. 2019. ⟨dumas-03190565⟩

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