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Master Thesis

Comparaison du devenir à long terme des patients atteints d'aplasie médullaire pédiatrique : allogreffe d'emblée versus traitement médical initial

Abstract : The aim of this cross-sectional study was to compare quality of life (QoL) and academic achievement between two groups of long-term survivors of childhood aplastic anaemia (AA), defined by their first treatment: patients who first received allogenic hematopoietic stem cells (allo-HSCT); or patients who first underwent immunosuppressive therapy. Validated health-related quality of life instruments appropriate for each age group were used: VSPAe for children aged 8 to 10; VSPA for adolescents aged 11 to 18; and SF36 for adults. Academic data was self-reported by adult participants and reported by the parents of younger participants. 26 participants treated for AA between January 2004 and December 2017 were included in the study: 16 first underwent underwent immunosuppressive therapy; 10 first received allo-HSCT. QoL scores reported by initially transplanted patients were significantly better than the other group. Children, adolescents and adults all report similar QoL than overall population. Academic achievement reported were similar in the two treatment groups. Our study could be an argument to prioritize allo-HSCT in first line of treatment in childhood AA. Because of the rarity of the condition, the use of multicentre studies is crucial for further evaluation of outcomes of survivors of childhood AA.
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Master Thesis
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https://dumas.ccsd.cnrs.fr/dumas-03321111
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Submitted on : Tuesday, August 17, 2021 - 9:57:48 AM
Last modification on : Wednesday, November 3, 2021 - 6:29:40 AM
Long-term archiving on: : Thursday, November 18, 2021 - 6:24:36 PM

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Manon Taourel. Comparaison du devenir à long terme des patients atteints d'aplasie médullaire pédiatrique : allogreffe d'emblée versus traitement médical initial. Sciences du Vivant [q-bio]. 2020. ⟨dumas-03321111⟩

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