Prolonged response to recombinant human erythropoietin treatment in patients with myelodysplastic syndrome at a single referral centre in Brazil

Abstract

OBJECTIVES: To evaluate the effects of epoetin (EPO) alfa treatment on overall survival, event-free survival and response duration in patients with myelodysplastic syndrome (MDS) who were treated at a haematological referral centre in northeastern Brazil. METHODS: This was a retrospective cohort study of 36 patients diagnosed with MDS and treated with EPO alfa at 30,000 to 60,000 IU per week. Clinical data were collected from medical records. The events assessed were non-response to treatment and progression to acute myeloid leukaemia (AML). Statistical analyses were performed using GraphPad Prism 7 and SPSS 24 software. RESULTS: The overall survival of patients who received EPO alfa treatment was 51.64%, with a median of 65 months of treatment, and the overall survival of this group was 100% during the first 24 months. We detected a 43.5-month median event-free survival, with a response rate of 80.5%. We observed responses from 25 to 175 months. Patients with transfusion dependence and those with a high-risk stratification, as determined by the International Prognostic Scoring System (IPSS), the Revised International Prognostic Scoring System (IPSS-R), the WHO classification-based Prognostic Scoring System (WPSS) and the WHO 2016, had a lower event-free survival than other patients. CONCLUSIONS: Despite the wide use of EPO alfa in the treatment of anaemia in patients with MDS, the median response duration is approximately only 24 months. Our data provide encouraging results concerning the benefits of using EPO alfa for the improvement of the quality of life, as patients treated with EPO showed higher overall survival, event-free survival rates and longer response durations than have been previously described in the literature.

Long-Term Efficacy of Erythropoiesis-Stimulating Agents in Patients with Low-Risk or Intermediate-1-Risk Myelodysplastic Syndrome: Multicenter Real-Life Data

“…In this study, the treatment response rates at the 24-month, 36-month, and 48-month visits in the epoetin alfa (43.0%, 33.6%, and 27.1%) and darbepoetin alfa (29.9%, 22.7%, and 16.5%) groups were lower than the 12-month response rates in each group (44.9% and 46.4%, respectively). These findings are consistent with the overall response rates ranging from 20% to 40% and durations of response ranging from 10 to 24 months reported in previous studies on ESAs, considering the refractoriness rate for ESAs to be approximately 40%-50% after 2 years of treatment [ 6 , 10 , 11 ]. Specifically, multicenter studies of epoetin-treated patients with MDS revealed response rates of approximately 30%-50% with durations of 12-24 months [ 1 , 6 , 15 , 16 , 17 , 18 , 19 , 20 , 21 ], while single-arm darbepoetin alfa studies reported durations of response ranging from 1 to 36 months [ 17 , 22 , 23 , 24 ].…”

“…Hence, the increased likelihood of transfusion independency with long-term epoetin treatment and lower-risk MDS in our cohort seems to be particularly notable given that the primary target in managing patients with lower-risk MDS in daily practice is to achieve transfusion independence, which is associated with improved survival [ 6 , 15 , 20 , 33 , 34 , 35 ]. Indeed, epoetin alfa treatment for patients with MDS is considered likely to achieve durable responses, thereby guaranteeing an improvement in the quality of life for these patients [ 10 ].…”

“…In a retrospective cohort study of 36 patients diagnosed with MDS and treated with epoetin alfa (30,000 to 60,000 IU per week), the authors reported a response rate of 80.5% with surprisingly prolonged responses (range: 25-175 months) in 82.8% of patients, who were also transfusion-independent and had favorable response stratifications [ 10 ]. Hemoglobin values were reported to increase significantly after 24 months and up to 175 months after the initiation of epoetin alfa treatment, and they remained above the baseline values, while the overall survival rate was 51.64% after a median of 65 months of treatment along with 43.5-month median event-free survival [ 10 ].…”

“…In a retrospective cohort study of 36 patients diagnosed with MDS and treated with epoetin alfa (30,000 to 60,000 IU per week), the authors reported a response rate of 80.5% with surprisingly prolonged responses (range: 25-175 months) in 82.8% of patients, who were also transfusion-independent and had favorable response stratifications [ 10 ]. Hemoglobin values were reported to increase significantly after 24 months and up to 175 months after the initiation of epoetin alfa treatment, and they remained above the baseline values, while the overall survival rate was 51.64% after a median of 65 months of treatment along with 43.5-month median event-free survival [ 10 ]. The authors indicated an association of epoetin alfa with higher overall survival and event-free survival rates and longer response durations in their study than previously described in the literature, and they emphasized that the likelihood of sustained rates of erythroid response following the initiation of epoetin alfa treatment also reflected the need for prolonged treatment to obtain the full clinical benefits [ 10 ].…”

“…However, the ESA response rates in routine practice may differ from those reported in clinical trials with the use of higher doses of ESAs [ 6 , 7 ]. The value of continuing ESAs in the absence of an early response also remains unclarified due to investigations of response rates rather than durations of response in most previous ESA studies [ 3 , 8 , 9 , 10 , 11 ].…”