Eva Rombout-Sestrienkova1,2, Bjorn Winkens3, Brigitte A B Essers4, Fred H M Nieman4, Paulus A H Noord5, Mirian C H Janssen6, Cees Th B M van Deursen7, Annelies Boonen8, Ellen P J M Reuser-Kaasenbrood1, Judith Heeremans1, Marian van Kraaij1, Ad Masclee2, Ger H Koek2. 1. Department of Transfusion Medicine, Sanquin Blood Supply, Amsterdam, the Netherlands. 2. Department of Internal Medicine, Division of Gastroenterology, Maastricht University Medical Centre, Maastricht, the Netherlands. 3. Department of Methodology and Statistic, School for Public Health and Primary Care (CAPHRI), Maastricht University. 4. Department of Clinical Epidemiology and Medical Technology Assessment. 5. Department of Research, Sanquin Blood Supply. 6. Department of Internal Medicine, Radboud University Nijmegen Medical Centre, Nijmegen, the Netherlands. 7. Department of Internal Medicine, Zuyderland Medical Center, Heerlen/Brunssum/Sittard, the Netherlands. 8. Department of Internal Medicine, Division of Rheumatology.
Abstract
BACKGROUND:Phlebotomy is standard maintenance treatment of patients with hereditary hemochromatosis (HH). Erythrocytapheresis, which selectively removes red blood cells, provides a new, potentially more effective treatment option. Our aim was to evaluate the effectiveness of erythrocytapheresis over phlebotomy for maintenance therapy in patients with HH. STUDY DESIGN AND METHODS: We conducted a two-treatment-arms, randomized, crossover clinical trial, involving 46 patients, treated for 1 year with either erythrocytapheresis or phlebotomy to keep the ferritin level at not more than 50 µg/L. After 1 year, patients were switched to the other treatment modality. Primary endpoint was the number of treatment procedures per treatment year. Secondary endpoints were intertreatment intervals, several aspects of health-related quality of life, costs, and patient discomfort as well as preference for one of both treatments. RESULTS: The mean number of required treatment procedures per treatment year was significantly higher using phlebotomy versus erythrocytapheresis (3.3 vs. 1.9; mean difference, 1.4; 95% confidence interval, 1.1-1.7). The median intertreatment time was 2.3 times longer for erythrocytapheresis. There was no significant difference in overall health assessed by SF-36 and EQ-5D, respectively, between both treatments arms. The number of self-reported swollen joints was significantly higher during phlebotomy treatment. The mean treatment costs of one treatment year were 235€ for phlebotomy versus 511€ for erythrocytapheresis. Eighty percent of patients preferred erythrocytapheresis as treatment method. CONCLUSION: Erythrocytapheresis significantly reduced the number of treatment procedures per treatment year, although the mean treatment costs per year are higher in our health care system. It is the preferred treatment for the majority of patients.
RCT Entities:
BACKGROUND: Phlebotomy is standard maintenance treatment of patients with hereditary hemochromatosis (HH). Erythrocytapheresis, which selectively removes red blood cells, provides a new, potentially more effective treatment option. Our aim was to evaluate the effectiveness of erythrocytapheresis over phlebotomy for maintenance therapy in patients with HH. STUDY DESIGN AND METHODS: We conducted a two-treatment-arms, randomized, crossover clinical trial, involving 46 patients, treated for 1 year with either erythrocytapheresis or phlebotomy to keep the ferritin level at not more than 50 µg/L. After 1 year, patients were switched to the other treatment modality. Primary endpoint was the number of treatment procedures per treatment year. Secondary endpoints were intertreatment intervals, several aspects of health-related quality of life, costs, and patient discomfort as well as preference for one of both treatments. RESULTS: The mean number of required treatment procedures per treatment year was significantly higher using phlebotomy versus erythrocytapheresis (3.3 vs. 1.9; mean difference, 1.4; 95% confidence interval, 1.1-1.7). The median intertreatment time was 2.3 times longer for erythrocytapheresis. There was no significant difference in overall health assessed by SF-36 and EQ-5D, respectively, between both treatments arms. The number of self-reported swollen joints was significantly higher during phlebotomy treatment. The mean treatment costs of one treatment year were 235€ for phlebotomy versus 511€ for erythrocytapheresis. Eighty percent of patients preferred erythrocytapheresis as treatment method. CONCLUSION: Erythrocytapheresis significantly reduced the number of treatment procedures per treatment year, although the mean treatment costs per year are higher in our health care system. It is the preferred treatment for the majority of patients.
Authors: Elena Buzzetti; Maria Kalafateli; Douglas Thorburn; Brian R Davidson; Emmanuel Tsochatzis; Kurinchi Selvan Gurusamy Journal: Cochrane Database Syst Rev Date: 2017-03-08
Authors: Luis Alfredo Utria Acevedo; Aline Morgan Alvarenga; Paula Fernanda Silva Fonseca; Nathália Kozikas da Silva; Rodolfo Delfini Cançado; Flavio Augusto Naoum; Carla Luana Dinardo; Alexandre Costa Pereira; Pierre Brissot; Paulo Caleb Junior Lima Santos Journal: Genes (Basel) Date: 2022-01-10 Impact factor: 4.096
Authors: Eva Rombout-Sestrienkova; Bjorn Winkens; Marian van Kraaij; Cees Th B M van Deursen; Mirian C H Janssen; Alexander M J Rennings; Dorothea Evers; Jean-Louis Kerkhoffs; Ad Masclee; Ger H Koek Journal: J Clin Apher Date: 2020-12-24 Impact factor: 2.821