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There is no clear consensus on the best practice for long-term prophylaxis in adults with severe haemophilia A. This is a single-centre prospective case series study. We describe here the demographic data, type and reason of prophylaxis in adult patients (>18 years old) with severe (<1%) haemophilia A, treated in our centre from 2006 to 2013. Prophylaxis was tailored according to pharmacokinetic studies and posterior factor VIII (FVIII) trough level adjustment. We analysed FVIII consumption, bleeding rate, adherence and adverse events in this group of patients. In adult patients who initiated long-term prophylaxis during this period, we compared FVIII consumption and bleeding rate with the previous on-demand period. We analysed data from 18 patients. Median annual FVIII consumption was 2374.2 IU/kg/year. Among the patients receiving tertiary prophylaxis, initiated from 2006 onwards, the annual FVIII consumption was 2557.8 vs. 1696.8 IU/kg per year during the on-demand period (P = 0.312). In this group of patients, there was a decrease in annual bleeding events of 88.3% during prophylaxis compared with the on-demand therapy (P < 0.0001). A high adherence to prophylaxis was observed (84%). No cases of anaphylaxis or symptomatic thromboembolic events were recorded. In adult severe haemophilia A patients, the type of and reason to indicate long-term prophylaxis are diverse nowadays. FVIII consumption varies depending on the justification of prophylaxis. The observations reported provide further support for the efficacy of long-term prophylaxis in adult haemophilia A patients.

Objective: To evaluate effectiveness of treatment with plerixafor in patients undergoing posterior mobilization for hematopoietic transplant at our hospital., Methods: Retrospective study of all patients who until September 2012, received plerixafor in their scheme of mobilization into peripheral blood hematopoietic progenitors. We reviewed the medical records and records of drug dispensing outpatient consultation. Effectiveness variables used were: CD34/kg in apheresis product obtained, and day dose received colony stimulating factor (G-CSF) and Plerixafor. Each patient was compared to the effectiveness of the drug results with those obtained earlier mobilization schemes where Plerixafor not used, if present. Data were analyzed using IBM SPSS v19., Results: A total of 24 patients received plerixafor in our hospital. Diagnoses were distributed: 15 NHL, 6 patients with multiple myeloma, 2 Hodgkin's disease, and one metastatic choriocarcinoma. The effectiveness outcomes were no plerixafor mobilization (n = 18): 5 patients were mobilized with G-CSGF only, 13 with G-CSF and chemotherapy. The G-CSF dose / day was mcg 931.1 (± 179.5) for 9.5 days (± 4.7). The average product obtained CD34/kg in cells was 0.2 (± 0.5). No patient received sufficient product (≥ 2 x 106 cells/kg) for subsequent autologous transplantation. 100% of the demonstrations failed. Mobilization with plerixafor (n = 24): 13 patients were mobilized with GCSGF only, 11 with G-CSF and chemotherapy. The G-CSF dose/ day and averaged Plerixafor mcg 885.1 (± 240.1) and 19.8 (± 4.4), respectively, administered for 8.9 (± 5.1) and 1 , 5 (± 0.6) days, respectively. The average product obtained in CD34/kg was 2.3 x 106 cells (± 1.7) (p = 0.014 in relation to the demonstrations without Plerixafor). Only 12.5% (n = 3) patients were unable to undergo autologous transplant., Conclusions: In our patients, plerixafor has proven effective in mobilizing hematopoietic progenitors for autologous back., (Copyright © 2013 SEFH. Published by AULA MEDICA. All rights reserved.)