Korean J Hematol 2009; 44(4):
Published online December 30, 2009
https://doi.org/10.5045/kjh.2009.44.4.205
© The Korean Society of Hematology
유기영 최용묵 박영실
한국혈우재단,
경희대학교 의과대학 소아과학교실
Background: Eleven percent of severe hemophilia A patients and 5% of severe hemophilia B patients may develop inhibitors. We have conducted aPCC-based maintenance therapy for hemophilia patients with high levels of responding inhibitors and we analyzed the efficacy, safety, the factor consumption and the expense of this treatment, as compared to on-demand therapy.
Methods: Eleven hemophilia patients with high levels of responding inhibitors were eligible for the study. We tried to evaluate the longitudinal bleeding episodes, the inhibitor titers, the X-ray findings, the adverse events and the factor consumption between on-demand therapy and maintenance therapy. The bypassing agent in this study was aPCC having a longer half-life. The dosage was 30∼50 U/kg, 3 times a week.
Results: The mean follow-up period was 6.8 months for on-demand therapy and 10.6 months for maintenance therapy. The mean dosage of aPCC was 45.2 U/kg. The episodes of hemarthrosis decreased by 61.4% (P=0.003) and other significant bleedings decreased by 45.2% (P=0.109). The inhibitor titers decreased in 7 patients and these increased in 4 patients, but anamnesis took place in only 1 patient. Radiologically, 2 patients improved, 1 patient got worse and 7 patients were stable. Neither adverse signs nor symptoms were noticed. The mean factor consumption changed from 55.8×103 U for aPCC and 48.6 mg for rFVIIa on-demand therapy to 216×103 U for aPCC and 4.8 mg rFVIIa for maintenance therapy. Maintenance therapy cost 67% more than on-demand therapy monthly (P=0.041).
Conclusion: aPCC-based maintenance therapy for hemophilia patients with high responding inhibitors cost 67% more than on-demand therapy, but it reduced by 61.4% the episodes of hemarthrosis and 45.2% of the other significant bleedings. aPCC-based maintenance therapy can very effectively reduce the bleeding episodes of hemophilia patients with high levels of responding inhibitors. (Korean J Hematol 2009;44:205-211.)
Keywords Hemophilia, Inhibitor, aPCC, Maintenance therapy
Korean J Hematol 2009; 44(4): 205-211
Published online December 30, 2009 https://doi.org/10.5045/kjh.2009.44.4.205
Copyright © The Korean Society of Hematology.
유기영 최용묵 박영실
한국혈우재단,
경희대학교 의과대학 소아과학교실
Ki Young Yoo, Yong Mook Choi, Young Shil Park
Korea Hemophilia Foundation
Department of Pediatrics, College of Medicine, Kyung Hee University, Seoul, Korea
Background: Eleven percent of severe hemophilia A patients and 5% of severe hemophilia B patients may develop inhibitors. We have conducted aPCC-based maintenance therapy for hemophilia patients with high levels of responding inhibitors and we analyzed the efficacy, safety, the factor consumption and the expense of this treatment, as compared to on-demand therapy.
Methods: Eleven hemophilia patients with high levels of responding inhibitors were eligible for the study. We tried to evaluate the longitudinal bleeding episodes, the inhibitor titers, the X-ray findings, the adverse events and the factor consumption between on-demand therapy and maintenance therapy. The bypassing agent in this study was aPCC having a longer half-life. The dosage was 30∼50 U/kg, 3 times a week.
Results: The mean follow-up period was 6.8 months for on-demand therapy and 10.6 months for maintenance therapy. The mean dosage of aPCC was 45.2 U/kg. The episodes of hemarthrosis decreased by 61.4% (P=0.003) and other significant bleedings decreased by 45.2% (P=0.109). The inhibitor titers decreased in 7 patients and these increased in 4 patients, but anamnesis took place in only 1 patient. Radiologically, 2 patients improved, 1 patient got worse and 7 patients were stable. Neither adverse signs nor symptoms were noticed. The mean factor consumption changed from 55.8×103 U for aPCC and 48.6 mg for rFVIIa on-demand therapy to 216×103 U for aPCC and 4.8 mg rFVIIa for maintenance therapy. Maintenance therapy cost 67% more than on-demand therapy monthly (P=0.041).
Conclusion: aPCC-based maintenance therapy for hemophilia patients with high responding inhibitors cost 67% more than on-demand therapy, but it reduced by 61.4% the episodes of hemarthrosis and 45.2% of the other significant bleedings. aPCC-based maintenance therapy can very effectively reduce the bleeding episodes of hemophilia patients with high levels of responding inhibitors. (Korean J Hematol 2009;44:205-211.)
Keywords: Hemophilia, Inhibitor, aPCC, Maintenance therapy
Myung Hee Han, and Young Shil Park
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