Long-term follow-up of myelodysplastic syndrome patients with moderate/severe anaemia receiving human recombinant erythropoietin + 13-cis-retinoic acid and dihydroxylated vitamin D3: independent positive impact of erythroid response on survival.

Abstract

We previously reported a 60% erythroid response rate with recombinant erythropoietin + 13-cis retinoic acid + dihydroxylated vitamin D3 in 63 elderly myelodysplastic patients (median age 75 years) with unfavourable features for response to erythropoietin alone [70% transfusion-dependent, 35% refractory anaemia with ring sideroblasts/refractory anaemia with excess of blasts type 1 (RAEB1), 70% with International Prognostic Scoring System (IPSS) Intermediate-1 or -2]. This report updates that case study at a 7-year follow-up, and compared the impact on overall survival of erythroid response to known prognostic factors. The erythroid response duration (median 17 months; 22 in non-RAEB patients, with 20% patients in response after 6 years of therapy) was longer than in most studies with erythropoietin alone. Overall survival (median 55 months in non-RAEB, 15 in RAEB1 patients) was negatively affected by RAEB1 diagnosis, IPSS and WPSS intermediate scores and transfusion-dependence. In the multivariate analysis, erythroid response maintained an independent positive impact on survival, particularly in non-RAEB patients in the first 3 years from diagnosis (90% survival compared to 50% of non-responders). In conclusion, the long-term follow-up confirmed the achievement, by our combined treatment, of fairly long-lasting erythroid response in the majority of MDS patients with unfavourable prognostic features for response to erythropoietin: this translated in a survival benefit that was independent from other prognostic features.

• Authors: Elena Crisà, Cristina Foli, Roberto Passera, Antonella Darbesio, Kimberly B Garvey, Mario Boccadoro, Dario Ferrero
• Journal: British journal of haematology (Br J Haematol) Vol. 158 Issue 1 Pg. 99-107 (Jul 2012) ISSN: 1365-2141 [Electronic] England
• PMID: 22571649 (Publication Type: Clinical Trial, Journal Article, Research Support, Non-U.S. Gov't)
• Copyright: © 2012 Blackwell Publishing Ltd.

Chemical References

• Recombinant Proteins
• Erythropoietin
• Isotretinoin
• Calcitriol

Topics

• Adult
• Aged
• Aged, 80 and over
• Anemia, Refractory (drug therapy)
• Calcitriol (therapeutic use)
• Drug Administration Schedule
• Drug Therapy, Combination
• Erythropoietin (therapeutic use)
• Female
• Humans
• Isotretinoin (therapeutic use)
• Male
• Middle Aged
• Myelodysplastic Syndromes (classification, drug therapy)
• Prognosis
• Recombinant Proteins (therapeutic use)
• Survival Analysis
• Treatment Outcome