title: Weekly standard doses of rh-EPO are highly effective for the treatment of anemic patients with low-intermediate 1 risk myelodysplastic syndromes.
abstract: For more than 20 years erythropoietin (rHEPO) has largely been used to treat anemia in myelodysplastic syndromes (MDS). Early clinical trials showed erythroid responses in no more than 15-25% of patients. In the last decade, a better selection of MDS patients suitable for a therapeutic challenge with rHEPO, alone or in combination with G-CSF, allowed for an increased response-rate, averaging around 40%. More recently, an even higher percentage of responses have been obtained using higher-doses of rHEPO (up to 80,000 IU/weekly) in lower-risk MDS patients. This treatment however, especially at such high doses, is costly and not easily affordable for prolonged periods. The aim of this study was to verify if the use of "standard" doses of rHEPO could induce a satisfying response-rate with a less expensive treatment schedule in IPSS-defined "lower-risk" MDS anemic patients. From January 2005 to December 2009 a total of 55 consecutive anemic (Hb ≤ 10 g/dL) patients (29 males, 26 females, median age 78 years) with low-intermediate-1 risk MDS were treated after informed consent with rHEPO (40,000 IU once a week subcutaneously) for at least 3 months; at the end of this period, erythroid response was assessed, and responders were allowed to continue the treatment indefinitely, whereas non-responders were considered "off study". Both efficacy and safety of the treatment were recorded and evaluated. After 3 months of treatment, 36 out of 55 (65.5%) patients achieved an erythroid response to rHEPO according to IWG 2006 criteria. Higher response-rates to rHEPO were related with both lower IPSS and particularly WPSS scores. Treatment was safe, and only 1 patient had to discontinue the treatment because of unmanageable side-effects. Among the 36 responders, 28 (77%) maintained the response after a median follow-up of 46 months. Our data indicate that standard doses of rHEPO are at least as effective as higher-doses for correcting anemia in lower-risk MDS patients; in this clinical scenario, this schedule allows for a consistent reduction of costs without precluding the achievement of a durable erythroid response.
authors.affiliation: Department of Hematology and Oncology, Azienda Ospedaliera Universitaria San Martino di Genova, Genova, Italy. balleari@unige.it
authors.forename: Enrico
authors.initials: E
authors.lastname: Balleari
authors.forename: Marino
authors.initials: M
authors.lastname: Clavio
authors.forename: Eleonora
authors.initials: E
authors.lastname: Arboscello
authors.forename: Andrea
authors.initials: A
authors.lastname: Bellodi
authors.forename: Andrea
authors.initials: A
authors.lastname: Bruzzone
authors.forename: Lisette
authors.initials: L
authors.lastname: Del Corso
authors.forename: Maria Vita
authors.initials: MV
authors.lastname: Lucchetti
authors.forename: Maurizio
authors.initials: M
authors.lastname: Miglino
authors.forename: Caterina
authors.initials: C
authors.lastname: Passalia
authors.forename: Ivana
authors.initials: I
authors.lastname: Pierri
authors.forename: Irene
authors.initials: I
authors.lastname: Ponassi
authors.forename: Caterina
authors.initials: C
authors.lastname: Oneto
authors.forename: Omar
authors.initials: O
authors.lastname: Racchi
authors.forename: Marco
authors.initials: M
authors.lastname: Scudeletti
authors.forename: Luana
authors.initials: L
authors.lastname: Vignolo
authors.forename: Gabriele
authors.initials: G
authors.lastname: Zoppoli
authors.forename: Marco
authors.initials: M
authors.lastname: Gobbi
authors.forename: Riccardo
authors.initials: R
authors.lastname: Ghio
mesh_terms: D000368:Aged; D000369:Aged, 80 and over; D000740:Anemia / Q000188:drug therapy* / Q000209:etiology; D004305:Dose-Response Relationship, Drug; D004921:Erythropoietin / Q000627:therapeutic use*; D005260:Female; D005500:Follow-Up Studies; D006801:Humans; D008297:Male; D008875:Middle Aged; D009190:Myelodysplastic Syndromes / Q000150:complications* / Q000628:therapy; D011379:Prognosis; D011994:Recombinant Proteins / Q000627:therapeutic use*; D012307:Risk Factors
chemical_list: D011994:Recombinant Proteins; D004921:Erythropoietin