Novel treatment for congenital FXIII deficiency.

نویسنده

  • Hans P Kohler
چکیده

light of the results of the French study, one can only agree with the recommendation of Lepretre et al that FCA should not be used for the treatment of CLL outside of clinical trials. The question of whether alemtuzumab should no longer be used in any chemoimmu-notherapy is an entirely different issue. It should be emphasized that a recent random-ized trial showed that the second-line use of fludarabine plus intravenous alemtuzumab, FA, led to a significant improvement of the overall survival rate without excess toxicity, in particular in CLL patients with advanced Rai stages. 5 Therefore, it is likely that alemtu-zumab will have a defined, valuable role in the management of CLL, for example, for high-risk patients or at relapse. Conflict-of-interest disclosure: The author declares no competing financial interests. ■ REFERENCES 1. Lepretre S, Aurran T, Mahé B, et al. Excess mortality after treatment with fludarabine and cyclophosphamide in combination with alemtuzumab in previously untreated patients with chronic lymphocytic leukemia in a random-ized phase 3 trial. of rituximab to fludarabine and cyclophosphamide in patients with chronic lymphocytic leukaemia: a randomised, open-label, phase 3 trial. Osterborg A. Variability in B-cell antigen expression: implications for the treatment of B-cell lymphomas and leuke-mias with monoclonal antibodies. combination with alemtuzumab is effective and feasible in patients with relapsed or refractory B-cell chronic lympho-cytic leukemia: results of a phase II trial. et al. Fludarabine plus alemtuzumab versus fludarabine alone in patients with previously treated chronic lympho-cytic leukaemia: a randomised phase 3 trial.bine plus cyclophosphamide versus fludarabine alone in first line therapy of younger patients with chronic lymphocytic leukemia. of alemtuzumab in combination with fludarabine in patients with relapsed or refractory B-cell chronic lymphocytic leukemia.netics of alemtuzumab used for in vivo and in vitro T-cell depletion in allogeneic transplantations: relevance for early adoptive immunotherapy and infectious complications.trations of alemtuzumab and antiglobulin responses in patients with chronic lymphocytic leukemia following intravenous or subcutaneous routes of administration.imab plus fludarabine and cyclophosphamide prolongs progression free survival compared with fludarabine and cyclo-phosphamide alone in previously treated chronic lymphocytic leukemia. Congenital Factor XIII (FXIII) deficiency is a rare autosomal recessive inherited disease leading to severe bleeding diathesis. In this issue of Blood, Inbal and colleagues report on a safe and novel treatment of this rare disorder with recombinant FXIII (rFXIII). 1 I n 1960, Duckert et al described the first clinical case of congenital FXIII deficiency. 2 They investigated a young …

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Recombinant factor XIII: a safe and novel treatment for congenital factor XIII deficiency.

Congenital factor XIII (FXIII) deficiency is a rare, autosomal-recessive disorder, with most patients having an A-subunit (FXIII-A) deficiency. Patients experience life-threatening bleeds, impaired wound healing, and spontaneous abortions. In many countries, only plasma or cryoprecipitate treatments are available, but these carry a risk for allergic reactions and infection with blood-borne path...

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Coagulation factor XIII (FXIII) has a major role in the final stage of blood coagulation, is important for wound healing and maintaining pregnancy. Severe congenital FXIII deficiency is a rare disorder with 1 patient in 1-3 million. Untreated, it causes bleeding events, with intracranial haemorrhage being the major cause of death, impaired wound healing, and abortion. FXIII deficiency was tradi...

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عنوان ژورنال:
  • Blood

دوره 119 22  شماره 

صفحات  -

تاریخ انتشار 2012