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1.
Am J Hematol ; 91(8): 782-6, 2016 08.
Article in English | MEDLINE | ID: mdl-27135784

ABSTRACT

Waldenstrom's macroglobulinemia is generally treated with alkylating agents, purine analogs and monoclonal antibodies, alone or in combination. We report the outcomes of 82 patients (median age 61 years) treated with the RFC combination. Twenty-five patients were treatment-naive. RFC was administered every 4 weeks, for a median of five cycles. At treatment discontinuation, the overall response rate was 85.4%. The responses improved after treatment discontinuation in 25 patients, with a median time to best response achievement of 10.8 months, raising the major response rate (PR, VGPR and CR) from 64.6% to 76.8%. With a median follow-up of 47 months, the median progression-free survival time had not been reached (67% PFS at 48 months) and was influenced by age and treatment status before RFC. Likewise, the median time to next therapy had not been reached. Two cases of myelodysplastic syndrome/AML and 3 cases of transformation to aggressive lymphoma occurred. Thirteen patients died. The 3-year overall survival rate was 90%. Long-lasting cytopenias occurred in 19 patients. The RFC combination thus gave a high response rate and durable responses, even in heavily treatment-experienced patients. The high incidence of long-lasting cytopenia might be reduced by giving fewer courses and thereby minimizing myelotoxicity. Am. J. Hematol. 91:782-786, 2016. © 2016 Wiley Periodicals, Inc.


Subject(s)
Antineoplastic Combined Chemotherapy Protocols/therapeutic use , Waldenstrom Macroglobulinemia/drug therapy , Aged , Antineoplastic Combined Chemotherapy Protocols/adverse effects , Cell Transformation, Neoplastic/chemically induced , Cyclophosphamide/administration & dosage , Female , Humans , Male , Middle Aged , Pancytopenia/chemically induced , Retrospective Studies , Rituximab/administration & dosage , Survival Rate , Treatment Outcome , Vidarabine/administration & dosage , Vidarabine/analogs & derivatives , Waldenstrom Macroglobulinemia/mortality , Waldenstrom Macroglobulinemia/pathology
2.
Hematology ; 14(6): 315-22, 2009 Dec.
Article in English | MEDLINE | ID: mdl-19941737

ABSTRACT

Deferoxamine (DFO) is an iron chelator used to treat iron overload in patients receiving chronic blood transfusions, and is usually administered as overnight subcutaneous infusions. ISOSFER was a prospective, observational, cross-sectional study conducted in metropolitan France that evaluated patient characteristics, quality of life (QoL), compliance and patient satisfaction with DFO monotherapy. Of 70 patients with either thalassemia, sickle cell disease or myelodysplastic syndromes, 30% were 'satisfied' or 'very satisfied' with DFO. Patients' SF-36 scores were lower than those of the general French population, and lower among patients with comorbidities and those dissatisfied with treatment. Although 72% of patients had good compliance to DFO, 57% reported missing at least one infusion in the previous month, and 82% of patients expressed a preference for oral therapy. These results suggest that QoL is severely compromised in patients receiving DFO, and that compliance is not optimal.


Subject(s)
Chelation Therapy/psychology , Deferoxamine/therapeutic use , Iron Overload/psychology , Quality of Life , Siderophores/therapeutic use , Adult , Aged , Aged, 80 and over , Blood Transfusion/psychology , Chelation Therapy/adverse effects , Child , Cross-Sectional Studies , Deferoxamine/adverse effects , Female , France , Hematologic Diseases/psychology , Hematologic Diseases/therapy , Humans , Iron Overload/drug therapy , Male , Middle Aged , Patient Compliance , Prospective Studies , Siderophores/adverse effects , Transfusion Reaction
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