Off-label prescription of hydroxycarbamide (hydroxyurea, HU) for severe anemia: preliminary results from European non-interventional, multicentric, prospective escort-HU study | oneSCDvoice
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abstracts & posters

Off-label prescription of hydroxycarbamide (hydroxyurea, HU) for severe anemia: preliminary results from European non-interventional, multicentric, prospective escort-HU study

key information

source: American Society of Hematology

year: 2017

authors: Mariane De Montalembert, Gylna Loko, Jerome Clouzeau, Valentine Brousse, Frederic Galacteros, Corinne Charneau, Marie Belloy, Malika Benkerrou

summary/abstract:

HU is licensed in Europe in the prevention of recurrent painful vaso-occlusive crises (VOC) including acute chest syndromes in adults, adolescents and children older than 2 years with sickle-cell disease (SCD). However, based on US and European expert panel recommendations (Yawn 2014, Habibi 2015) and results from placebo-controlled clinical trials, HU could be useful in SCD patients with severe anemia without VOC since it has been demonstrated to increase total Hb level (Wang 2011) and to decrease the need for blood transfusion. We hereby present preliminary results on effectiveness and safety data related to the prescription of HU for anemia from ESCORT-HU (European Sickle Cell Disease COhoRT – HydroxyUrea), a multicentric, prospective, non-interventional European study designed to collect long-term safety data on HU in SCD population. Between January 2009 and June 2017, 1841 patients were enrolled from 63 centers in France, Germany, Greece and Italy, amongst which 126 patients (6.8%) were started on HU for anemia from 34 centers. Of these 126, 96 were HU-naive. These HU-naive patients treated for anemia (‘anemic’ subpopulation) were selected for analysis to evaluate effectiveness and safety of HU in this indication and compared with data in HU-naive patients treated for other SCD indications. Demographic data and Hb genotypes are displayed in Table 1. The mean age, distribution of gender, Hb genotype and the mean HU dose at initiation were comparable in the ‘anemic’ subpopulation and the ‘non-anemic’ HU-naive cohort. Not surprisingly, mean Hb level at initiation was markedly lower in the ‘anemic’ subpopulation (7.07 ± 0.88 g/dl) than in the ‘non-anemic’ HU-naive cohort (8.71 ± 1.51 g/dl), with a lower proportion of patients with history of VOC and SCD-related hospitalization prior to HU initiation. The mean HU dose after 6 months was comparable in both groups (15.6 ± 3.83 mg/kg/day and 15.4 ± 4.11 mg/kg/day, respectively).

organisation: Necker Children’s Hospital, Assistance Publique-Hôpitaux de Paris, Paris, France; Laboratory of Excellence, GR-Ex, Paris, France; Martinique University Hospital, Le Lamentin, Martinique; Franck-Joly Hospital, Saint-Laurent du Maroni, Guyana; Henri Mondor Hospital - APHP, Creteil, France; Basse-Terre Hospital, Basse-Terre, Guadeloupe; Robert-Ballanger Hospital, Aulnay-sous-Bois, France; Robert Debré Hospital - APHP, Paris, France

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