1 research outputs found
HSCT may lower leukemia risk in ELANE neutropenia: a beforeâafter study from the French Severe Congenital Neutropenia Registry
International audienceELANE neutropenia is associated with myelodysplasia and acute leukemia (MDSâAL), and severe infections. Because the MDSâAL risk has also been shown to be associated with exposure to GCSF, since 2005, in France, patients receiving high daily GCSF doses (>15âÎŒg/kg/day) are eligible for HSCT, in addition to classic indications (MDSâAL or GCSF refractoriness). We analyzed the effect of this policy. Among 144 prospectively followed ELANE-neutropenia patients enrolled in the French Severe Congenital Neutropenia Registry, we defined two groups according to period: âbefore 2005â for those born before 2005 and followed until 31/12/2004 (1588 person-years); and âafter 2005â comprised of those born after 2005 or born before 2005 but followed after 2005 until 31/03/2019 (1327 person-years). Sixteen of our cohort patients underwent HSCT (14 long-term survivors) and six developed MDSâALs. Six leukemic transformations occurred in the before-2005 group and none after 2005 (respective frequencies 3.8âĂâ10â3 vs. 0; Pâ<â0.01), while four HSCTs were done before 2005 and 12 since 2005 (respective HSCT rates increased 2.5âĂâ10â3 vs. 9âĂâ10â3; Pâ<â0.01). Our results support early HSCT for patients with ELANE mutations who received high GCSF doses, as it might lower the risk of leukemic transformation