title: Darbepoetin alfa and ferric gluconate ameliorate the anemia associated with recessive dystrophic epidermolysis bullosa.
abstract: Our objective was to describe the efficacy of darbepoetin alfa and ferric gluconate complex in the treatment of the anemia associated with recessive dystrophic epidermolysis bullosa. To accomplish this aim, we retrospectively reviewed a series of patients with this disease treated in a single institution with darbepoetin alfa and parenteral iron for anemia. Four patients with recessive dystrophic epidermolysis bullosa were treated for a mean length of treatment of 14.5 months (4-18 months). Three patients received parenteral iron in the form of ferric gluconate complex and one received iron dextran. The mean pretreatment hemoglobin was 6.8 g/dL (4.9-9.6 g/dL). All four had improvements in their hemoglobin levels and energy levels with a mean increase in hemoglobin level of 2.8 g/dL (p = 0.003). We found darbepoetin alfa and ferric gluconate complex to be effective in the treatment of anemia associated with recessive dystrophic epidermolysis bullosa and to have distinct advantages over previously described formulations, and we concluded that they should be considered in the supportive care of this disease. We recommend the development of a standardized protocol for the evaluation and management of recessive dystrophic epidermolysis bullosa-associated anemia.
authors.affiliation: Department of Pediatrics, Stanford University School of Medcine, Stanford, CA, USA.
authors.forename: Dennis J
authors.initials: DJ
authors.lastname: Kuo
authors.forename: Anna L
authors.initials: AL
authors.lastname: Bruckner
authors.forename: Michael R
authors.initials: MR
authors.lastname: Jeng
mesh_terms: D000293:Adolescent; D000740:Anemia / Q000188:drug therapy* / Q000209:etiology / Q000235:genetics; D002648:Child; D000068256:Darbepoetin alfa; D016108:Epidermolysis Bullosa Dystrophica / Q000150:complications / Q000188:drug therapy* / Q000235:genetics; D004921:Erythropoietin / Q000031:analogs & derivatives* / Q000627:therapeutic use; D005260:Female; D005290:Ferric Compounds / Q000627:therapeutic use*; D005808:Genes, Recessive; D006397:Hematinics / Q000627:therapeutic use*; D006801:Humans; D008297:Male; D012189:Retrospective Studies
chemical_list: D005290:Ferric Compounds; D006397:Hematinics; D004921:Erythropoietin; D000068256:Darbepoetin alfa; C035552:ferric gluconate