Erythropoietin treatment of the anaemia of myelofibrosis with myeloid metaplasia: results in 20 patients and review of the literature

Abstract

Summary Recombinant human erythropoietin (rHuEPO) is an effective treatment for the anaemia that occurs secondary to various conditions, but its role in myelofibrosis with myeloid metaplasia (MMM) is not well established. rHuEPO, at an initial dose of 10 000 U thrice a week, was given to 20 patients with MMM and anaemia. Complete response (CR) was defined as transfusion cessation with normal haemoglobin (Hb) levels and partial response (PR) as a transfusion decrease ≥50% and Hb > 10 g/dl maintained for at least 8 weeks. Nine patients (45%) showed a favourable response to treatment, including four CR and five PR, four of whom have maintained their response at a median follow‐up of 12·5 months (range: 4–21 months) from the start of treatment. The pretreatment factors associated with a favourable response were lack of transfusion requirement (P = 0·002) and higher Hb at start treatment of (P = 0·01). An analysis of the present series (n = 20) and 31 patients from the literature identified 28 (55%) favourable responses to rHuEPO, including 16 CR and 12 PR. In the multivariate analysis, serum erythropoietin level <125 U/l was found to be associated with a favourable response to rHuEPO, whereas lack of transfusional support had borderline significance.

Efficacy and tolerability of hydroxyurea in the treatment of the hyperproliferative manifestations of myelofibrosis: results in 40 patients

“…Thus, a wait-and-see approach is often adopted in asymptomatic patients, delaying treatment start until a change in the clinical situation is observed. In patients with anemia, besides transfusion therapy, treatment is based on the use of androgens [19,20], erythropoietin or erythropoietin-stimulating agents [17,18] and, more recently, immuuomodulatory drugs such as thalidomide, lenalidomide or pomalidomide [21][22][23]. With regard to symptoms of hyperproliferation of MF, they are usually managed with oral cytoreductive drugs, mainly HU [9,10], but also busulfan [24] or melphalan [25].…”

“…The minimum time to assess the response was established as 3 months. In case of appearance or worsening of a preexisting anemia, either erythropoietin/darbepoetin-alfa or danazol were administered, depending on the patient's serum erythropoietin levels, as previously reported [17][18][19]. Table 1 shows the main characteristics of the 40 patients.…”

Modern management of myelofibrosis

“…Most responses, but not all, are seen in patients with inappropriate erythropoietin levels. In a recent analysis of 20 patients with IM and anaemia treated with erythropoietin, the response rate was 45%, with 20% of patients maintaining the response in the long-term (Cervantes et al, 2004). Pretreatment variables associated with a favourable response were lack of transfusion requirement and higher Hb value at treatment start.…”

Guideline for the diagnosis and management of myelofibrosis

“…In an analysis of 20 anaemic MF patients treated with rEPO, responses were seen in 45% of cases but only maintained long-term in 20% (Cervantes et al, 2004); responses to rEPO being more likely in transfusion-independent patients with higher baseline Hb. A pooled analysis of this 20-patient series with 31 patients from the literature demonstrated an overall rEPO response rate of 55% [31% complete response (CR)] with a median duration of 12 months (Cervantes et al, 2004).…”