Allogeneic stem cell transplantation (alloSCT) continues to be the only potentially curative treatment for patients with refractory lymphomas or relapsing after autologous stem cell transplantation. ...Until recently, alloSCT was restricted to patients who had a matched donor, sibling or unrelated. In the past years, substantial progress in haploidentical transplantation (haploSCT) has resulted in a significant increase in the number of patients treated with this procedure, worldwide. Given the fact that an HLA haplo-identical donor can be found within the immediate family for almost any patient, virtually every patient can receive an haploSCT. Another reason to use haploSCT, especially in diseases like lymphomas where the decision to perform an alloSCT is being taken sometimes late in the course of the disease, is the considerable delay to find a matched unrelated donor (MUD), when an HLA-identical sibling (MSD) is not available. In this paper, we summarize available evidence supporting the use of haploSCT in lymphoma patients and share current recommendations of the Lymphoma Working Party of the European Society for Blood and Marrow Transplantation (EBMT) on how to integrate haploSCT in this population.
The use of antihuman T-lymphocyte immunoglobulin in the setting of transplantation from an HLA-matched related donor is still much debated. Acute and chronic graft-
-host disease are the main causes ...of morbidity and mortality after allogeneic hematopoietic stem cell transplantation in patients with myelofibrosis. The aim of this study was to evaluate the effect of antihuman T-lymphocyte immunoglobulin in a large cohort of patients with myelofibrosis (n=287). The cumulative incidences of grade II-IV acute graft-
-host disease among patients who were or were not given antihuman T-lymphocyte immunoglobulin were 26% and 41%, respectively. The corresponding incidences of chronic graft-
-host disease were 52% and 55%, respectively. Non-adjusted overall survival, disease-free survival and non-relapse mortality rates were 55%
53%, 49%
45%, and 32%
31%, respectively, among the patients who were or were not given antihuman T-lymphocyte immunoglobulin. An adjusted model confirmed that the risk of acute graft-
-host disease was lower following antihuman T-lymphocyte immunoglobulin (hazard ratio, 0.54;
=0.010) while it did not decrease the risk of chronic graft-
-host disease. The hazard ratios for overall survival and non-relapse mortality were 0.66 and 0.64, with
-values of 0.05 and 0.09, respectively. Antihuman T-lymphocyte immunoglobulin did not influence disease-free survival, graft-
-host disease, relapse-free survival or relapse risk. In conclusion, in the setting of matched related transplantation in myelofibrosis patients, this study demonstrates that antihuman T-lymphocyte immunoglobulin decreases the risk of acute graft-
-host disease without increasing the risk of relapse.
Acute myeloid leukemia (AML) in first remission (CR1) with isolated NPM1 mutation (iNPM1m) is considered a good prognosis genotype, although up to one‐third relapse. To evaluate the best transplant ...strategy, we retrospectively compared autologous stem cell transplantation (auto‐SCT), related (MSD), and fully matched unrelated (MUD) allogeneic stem cell transplantation (allo‐SCT). We identified 256 adult patients including 125 auto‐SCT, 72 MSD, and 59 MUD. The 2‐year leukemia‐free survival (LFS) was 62% in auto‐SCT, 69% in MUD, and 81% in MSD (P = .02 for MSD vs others). The 2‐year overall survival (OS) was not different among auto‐SCT, MUD, and MSD, reaching 83% (P = .88). The 2‐year non‐relapse mortality (NRM) was 2.5% in auto‐SCT and 7.5% in allo‐SCT (P = .04). The 2‐year cumulative incidence of relapse (RI) was higher after auto‐SCT (30%) than after MUD (22%) and MSD (12%, P = .01). In multivariate analysis, MSD versus auto‐SCT but not MUD versus auto‐SCT was associated with lower RI (P < .01 and P = .13, respectively) and better LFS (P = .01 and P = .31, respectively). Age correlated with higher NRM (P < .01). Allo‐SCT using MSD appears as a reasonable transplant option for young patients with iNPM1m AML in CR1. Auto‐SCT was followed by worse RI and LFS, but similar OS to both allo‐SCT modalities.
The myeloproliferative neoplasms, including polycythemia vera, essential thrombocythemia and myelofibrosis, are distinguished by their debilitating symptom profiles, life-threatening complications ...and profound impact on quality of life. The role gender plays in the symptomatology of myeloproliferative neoplasms remains under-investigated. In this study we evaluated how gender relates to patients' characteristics, disease complications and overall symptom expression. A total of 2,006 patients (polycythemia vera=711, essential thrombocythemia=830, myelofibrosis=460, unknown=5) were prospectively evaluated, with patients completing the Myeloproliferative Neoplasm-Symptom Assessment Form and Brief Fatigue Inventory Patient Reported Outcome tools. Information on the individual patients' characteristics, disease complications and laboratory data was collected. Consistent with known literature, most female patients were more likely to have essential thrombocythemia (48.6% versus 33.0%; P<0.001) and most male patients were more likely to have polycythemia vera (41.8% versus 30.3%; P<0.001). The rate of thrombocytopenia was higher among males than females (13.9% versus 8.2%; P<0.001) and males also had greater red-blood cell transfusion requirements (7.3% versus 4.9%; P=0.02) with shorter mean disease duration (6.4 versus 7.2 years, P=0.03). Despite there being no statistical differences in risk scores, receipt of most therapies or prior complications (hemorrhage, thrombosis), females had more severe and more frequent symptoms for most individual symptoms, along with overall total symptom score (22.8 versus 20.3; P<0.001). Females had particularly high scores for abdominal-related symptoms (abdominal pain/discomfort) and microvascular symptoms (headache, fatigue, insomnia, concentration difficulties, dizziness; all P<0.01). Despite complaining of more severe symptom burden, females had similar quality of life scores to those of males. The results of this study suggest that gender contributes to the heterogeneity of myeloproliferative neoplasms by influencing phenotypic profiles and symptom expression.
Allogeneic stem cell transplantation is usually considered the only curative treatment option for patients with advanced or transformed myelodysplastic syndromes in complete remission, but ...post-remission chemotherapy and autologous stem cell transplantation are potential alternatives, especially in patients over 45 years old.
We evaluated, after intensive anti-leukemic remission-induction chemotherapy, the impact of the availability of an HLA-identical sibling donor on an intention-to treat basis. Additionally, all patients without a sibling donor in complete remission after the first consolidation course were randomized to either autologous peripheral blood stem cell transplantation or a second consolidation course consisting of high-dose cytarabine.
The 4-year survival of the 341 evaluable patients was 28%. After achieving complete remission, the 4-year survival rates of patients under 55 years old with or without a donor were 54% and 41%, respectively, with an adjusted hazard ratio of 0.81 (95% confidence interval 95% CI, 0.49-1.35) for survival and of 0.67 (95% CI, 0.42-1.06) for disease-free survival. In patients with intermediate/high risk cytogenetic abnormalities the hazard ratio in multivariate analysis was 0.58 (99% CI, 0.22-1.50) (P=0.14) for survival and 0.46 (99% CI, 0.22-1.50) for disease-free survival (P=0.03). In contrast, in patients with low risk cytogenetic characteristics the hazard ratio for survival was 1.17 (99% CI, 0.40-3.42) and that for disease-free survival was 1.02 (99% CI, 0.40-2.56). The 4-year survival of the 65 patients randomized to autologous peripheral blood stem cell transplantation or a second consolidation course of high-dose cytarabine was 37% and 27%, respectively. The hazard ratio in multivariate analysis was 1.22 (95% CI, 0.65-2.27) for survival and 1.02 (95% CI, 0.56-1.85) for disease-free survival.
Patients with a donor and candidates for allogeneic stem cell transplantation in first complete remission may have a better disease-free survival than those without a donor in case of myelodysplastic syndromes with intermediate/high-risk cytogenetics. Autologous peripheral blood stem cell transplantation does not provide longer survival than intensive chemotherapy.
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