Outcome of Ph negative myeloproliferative neoplasms transforming to accelerated or leukemic phase

Abstract

Myeloproliferative neoplasms (MPN) are chronic disorders that can sometimes evolve into accelerated or leukemic phases. We retrospectively identified 122 patients with such blastic phases. The overall median survival was four months: 10.2 months for patients treated with intensive treatments compared to three months for best supportive care (p = .005). Azacytidine, intensive chemotherapies, or allogeneic stem cell transplantation gave the highest median survivals with 9, 10.2, and 19.4 months, respectively. Accelerated phases (AP) had a longer median survival compared to acute leukemia (4.8 months vs. 3.1 months; p = .02). In this retrospective and observational study, we observe that the longest survivals are seen in patients eligible for intensive treatments. Azacytidine shows interesting results in patients non-fit for intensive chemotherapy. Supportive care should probably be restricted to elderly patients and those with unfavorable karyotype. An early diagnosis of AP could also result in a better survival rate.

Treatment of Philadelphia‐negative myeloproliferative neoplasms in accelerated/blastic phase with azacytidine. Clinical results and identification of prognostic factors

“…Myeloproliferative neoplasms (MPN) are chronic diseases characterized by a relatively long survival with the current available treatments. However, evolution into an accelerated or blastic phase (MPN‐AP/BP) may occur during the clinical follow‐up; this event is similar to de novo acute myeloid leukemia (AML) and is associated with a very poor outcome with only a few months of survival likelihood …”

“…Treatment options for MPN‐AP/BP are at present very limited. Palliative care is generally used in elderly patients, while intensive chemotherapy followed by allogeneic stem cell transplantation (SCT) has been employed in younger subjects, but the results have been largely disappointing with high rates of treatment‐related deaths and disease recurrence …”

Successful Decitabine Treatment in Unfit, Elderly Patients with Acute Myeloid Leukemia following Chronic Myeloproliferative Neoplasm

“…The blastic phase of MPNs is a challenging therapeutic dilemma [2-5]. It is associated with poor treatment response and a median OS of 4 months, ranging from 3 to 10.2 months for patients receiving best supportive care or allogeneic SCT, respectively [3, 5]. Although some reports have suggested the useful role of hypomethylating agents to treat MPNs [7-11], no controlled clinical trials have been performed.…”

“…The remaining 2 patients, who achieved PR, progressed to overt AML and were managed with supportive care after decitabine withdrawal until death. The blastic phase of MPNs is a challenging therapeutic dilemma [2-5]. It is associated with poor treatment response and a median OS of 4 months, ranging from 3 to 10.2 months for patients receiving best supportive care or allogeneic SCT, respectively [3, 5].…”

“…Approximately 5–10% of patients with a Ph-negative MPN progress into acute myeloid leukemia (AML) with a dismal outcome [2-4]. Treatment options for these patients are extremely limited [5-7], with only a minority suitable for intensive chemotherapy and/or allogeneic stem cell transplantation (SCT) [3]. Although a beneficial clinical role for hypomethylating compounds has been suggested [6-11], the effect of these agents in this difficult setting has not been clearly established.…”