Improved outcome of patients with relapsed/refractory Hodgkin lymphoma with a new fotemustine-based high-dose chemotherapy regimen.

High-dose chemotherapy (HDT) with autologous stem cell transplantation is the standard of care for relapsed/refractory (RR) Hodgkin lymphoma (HL). Given that HDT may cure a sizeable proportion of patients refractory to first salvage, development of newer conditioning regimens remains a priority. We present the results of a novel HDT regimen in which carmustine was substituted by a third-generation chloroethylnitrosourea, fotemustine, with improved pharmacokinetics and safety (FEAM; fotemustine, etoposide, cytarabine, melphalan) in 122 patients with RR-HL accrued into a prospective registry-based study. Application of FEAM resulted in a 2-year progression-free survival (PFS) of 73·8% [95% confidence interval (CI), 0·64-0·81] with median PFS, overall survival and time to progression yet to be reached. The 2-year risk of progression adjusted for the competitive risk of death was 19·4% (95% CI, 0·12-0·27) for the entire patient population. Most previously established independent risk factors, except for fluorodeoxyglucose ((18) (F) FDG)-uptake, were unable to predict for disease progression and survival after FEAM. Although 32% of patients had (18) (F) FDG-positrin emission tomography-positive lesions before HDT, the 2-year risk of progression adjusted for competitive risk of death was 19·4% (95% CI; 0·12-0·27). No unusual acute toxicities or early/late pulmonary adverse events were registered. FEAM emerges as an ideal HDT regimen for RR-HL patients typically pre-exposed to lung-damaging treatments.

Long-term results of survival in patients with thalassemia major treated with bone marrow transplantation.

Allogeneic bone marrow transplantation (BMT) is the only available curative approach for thalassemia major, although long-term morbidity and mortality are not established. The aim of this study was to assess the long-term clinical and hematological results in children and adults with thalassemia major treated with BMT. We analyzed the outcome of 115 patients (median age 9 years, range 11 months to 28 years) with thalassemia major undergoing BMT from a related donor between 1983 and 2006. All patients received the same protocol, consisting of busulfan and cyclophoshamide as conditioning therapy and cyclosporin (CSA) alone or CSA and methotrexate for graft-versus-host disease (GvHD) prophylaxis. The cumulative probability of graft rejection was 6.7%. The transplant-related mortality at 1 year was 8.7%. The 20-year Kaplan-Meier estimate of overall survival and disease-free survival was 89.2% and 85.7%, respectively. Ninety-nine patients out of 103 survivors were in excellent clinical and hematological conditions at last visit following a median follow-up of 15 years (range, 1-24 years) with the exception of two patients who had invalidating chronic GvHD. This study conducted with a large cohort of patients and covering a long period of observation time, showed BMT to be curative for the majority of patients with thalassemia major. The impact of long-term transplant-related sequelae was very limited.