Abstract
Philadelphia negative myeloproliferative or myeloproliferative/myelodysplastic neoplasms may evolve towards secondary acute myeloid leukemia (AML). The prognosis of such secondary leukemia is very poor. At present, there are only a few reports assessing the outcome of adult patients with a philadelphia negative myeloproliferative or myeloproliferative/myelodysplastic neoplasm in blast phase (MPN-BP) who received allogeneic stem cell transplantation (allo-SCT).
in this retrospective study, inclusion criteria were: (i) adult patients with a MPN-BP (ii) who received first allo-SCT (iii) between 2000 and 2010 (iv) irrespective of the stem cell source or conditioning regimen. MPN with <20% blasts in blood/bone marrow and AML secondary to myelodysplastic syndromes were excluded from this analysis.
60 patients were included. MPN, AML and allo-SCT characteristics are described in table 1. Median age at allo-SCT was 57 (range, 30-68). Patients received allo-SCT in first complete remission (CR1), CR2 or in advanced disease in 22 (37%), 4 (7%) and 34(57%) of cases, respectively. Engraftment was achieved in 55 cases (92%). With a median follow-up of 31 months (range, 25-44), the 3-year overall survival (OS) and Leukemia-Free-Survival (LFS) were respectively 18% and 9%. The 3-year transplant-related mortality (TRM) was 24% whereas relapse incidence was 68%. The 3-year LFS of patients grafted in CR (n=26) was 18% whereas the 3-year LFS of patients allografted in advanced disease (n=34) was only 3% (p=0.008). In the CR group, the 3-year TRM was 24% whereas relapse incidence was 61%. Intermediate or good AML karyotype (3-year LFS of 33% versus 10% for adverse AML karyotype, p=0.03) and the absence of a previous thrombotic event (3-year LFS of 24% versus 0, p=0.02) were associated with an improved LFS in patients allografted in CR.
N . | 60 . |
---|---|
Patient gender | Male: 46 (77%) |
MPN | Myeloproliferative neoplasms: 43 Polycythemia vera: 10 Essential Thrombocytemia: 20 Primary myelofibrosis: 8 Other: 5 Myelodysplastic/myeloproliferative neoplasms: 17 Chronic myelomonocytic leukemia: 15 Other: 2 |
Time from MPN diagnosis to blast phase (months) | 66 (range, 4-330) |
Median age at time allo-SCT (years) | 57 (range, 30-68) |
AML karyotype | Favorable: 1 Normal: 16 Other intermediate risk: 9 Adverse: 25 Failed: 9 |
AML therapy before allo-SCT | Intensive chemotherapy: 50 Hypomethylating agent: 6 Upfront allo-SCT: 7 |
AML status at allo-SCT | CR1: 22 CR>1: 4 advanced: 34 |
Stem cell source | Bone Marrow: 10 Peripheral Blood Stem Cell: 44 Cord Blood: 6 |
Conditioning regimen | Myeloablative: 14 Reduced-intensity regimen: 33 Sequential regimen: 13 |
N . | 60 . |
---|---|
Patient gender | Male: 46 (77%) |
MPN | Myeloproliferative neoplasms: 43 Polycythemia vera: 10 Essential Thrombocytemia: 20 Primary myelofibrosis: 8 Other: 5 Myelodysplastic/myeloproliferative neoplasms: 17 Chronic myelomonocytic leukemia: 15 Other: 2 |
Time from MPN diagnosis to blast phase (months) | 66 (range, 4-330) |
Median age at time allo-SCT (years) | 57 (range, 30-68) |
AML karyotype | Favorable: 1 Normal: 16 Other intermediate risk: 9 Adverse: 25 Failed: 9 |
AML therapy before allo-SCT | Intensive chemotherapy: 50 Hypomethylating agent: 6 Upfront allo-SCT: 7 |
AML status at allo-SCT | CR1: 22 CR>1: 4 advanced: 34 |
Stem cell source | Bone Marrow: 10 Peripheral Blood Stem Cell: 44 Cord Blood: 6 |
Conditioning regimen | Myeloablative: 14 Reduced-intensity regimen: 33 Sequential regimen: 13 |
These results suggest that the outcome of patients with a MPN-BP is dismal despite allo-SCT due to a high relapse incidence even in patients transplanted in CR. Outside a clinical trial, allo-SCT should be mainly proposed to patients in CR. New strategies are mandatory to improve the outcome of patients in blast phase.
No relevant conflicts of interest to declare.
Author notes
Asterisk with author names denotes non-ASH members.
This feature is available to Subscribers Only
Sign In or Create an Account Close Modal