Erythrocytapheresis versus phlebotomy in the maintenance treatment of HFE hemochromatosis patients: results from a randomized crossover trial

E. Rombout-Sestrienkova, B. Winkens, Brigitte Essers, Fred Nieman, P.A. Noord, M.C. Janssen, Cees Th B.M. van Deursen, A. Boonen, E.P. Reuser-Kaasenbrood, J. Heeremans, M. van Kraaij, A. Masclee, Ger Koek

Research output: Contribution to journalArticleAcademicpeer-review

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Abstract

BACKGROUNDPhlebotomy 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 METHODSWe 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 mu g/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.

RESULTSThe 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 235Euro for phlebotomy versus 511Euro for erythrocytapheresis. Eighty percent of patients preferred erythrocytapheresis as treatment method.

CONCLUSIONErythrocytapheresis 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.

Original languageEnglish
Pages (from-to)261-270
Number of pages10
JournalTransfusion
Volume56
Issue number1
DOIs
Publication statusPublished - 1 Jan 2016

Keywords

  • HEREDITARY HEMOCHROMATOSIS
  • IRON OVERLOAD
  • IDIOPATHIC HEMOCHROMATOSIS
  • ARTHROPATHY
  • ARTHRITIS
  • THERAPY
  • HEALTH
  • ERYTHROPOIETIN
  • GUIDELINE
  • DIAGNOSIS

Cite this

Rombout-Sestrienkova, E. ; Winkens, B. ; Essers, Brigitte ; Nieman, Fred ; Noord, P.A. ; Janssen, M.C. ; van Deursen, Cees Th B.M. ; Boonen, A. ; Reuser-Kaasenbrood, E.P. ; Heeremans, J. ; van Kraaij, M. ; Masclee, A. ; Koek, Ger. / Erythrocytapheresis versus phlebotomy in the maintenance treatment of HFE hemochromatosis patients: results from a randomized crossover trial. In: Transfusion. 2016 ; Vol. 56, No. 1. pp. 261-270.
@article{835bc46d6d7e4a02af3176156a3e9e71,
title = "Erythrocytapheresis versus phlebotomy in the maintenance treatment of HFE hemochromatosis patients: results from a randomized crossover trial",
abstract = "BACKGROUNDPhlebotomy 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 METHODSWe 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 mu g/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.RESULTSThe 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 235Euro for phlebotomy versus 511Euro for erythrocytapheresis. Eighty percent of patients preferred erythrocytapheresis as treatment method.CONCLUSIONErythrocytapheresis 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.",
keywords = "HEREDITARY HEMOCHROMATOSIS, IRON OVERLOAD, IDIOPATHIC HEMOCHROMATOSIS, ARTHROPATHY, ARTHRITIS, THERAPY, HEALTH, ERYTHROPOIETIN, GUIDELINE, DIAGNOSIS",
author = "E. Rombout-Sestrienkova and B. Winkens and Brigitte Essers and Fred Nieman and P.A. Noord and M.C. Janssen and {van Deursen}, {Cees Th B.M.} and A. Boonen and E.P. Reuser-Kaasenbrood and J. Heeremans and {van Kraaij}, M. and A. Masclee and Ger Koek",
year = "2016",
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doi = "10.1111/trf.13328",
language = "English",
volume = "56",
pages = "261--270",
journal = "Transfusion",
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publisher = "Wiley",
number = "1",

}

Erythrocytapheresis versus phlebotomy in the maintenance treatment of HFE hemochromatosis patients: results from a randomized crossover trial. / Rombout-Sestrienkova, E.; Winkens, B.; Essers, Brigitte; Nieman, Fred; Noord, P.A.; Janssen, M.C.; van Deursen, Cees Th B.M.; Boonen, A.; Reuser-Kaasenbrood, E.P.; Heeremans, J.; van Kraaij, M.; Masclee, A.; Koek, Ger.

In: Transfusion, Vol. 56, No. 1, 01.01.2016, p. 261-270.

Research output: Contribution to journalArticleAcademicpeer-review

TY - JOUR

T1 - Erythrocytapheresis versus phlebotomy in the maintenance treatment of HFE hemochromatosis patients: results from a randomized crossover trial

AU - Rombout-Sestrienkova, E.

AU - Winkens, B.

AU - Essers, Brigitte

AU - Nieman, Fred

AU - Noord, P.A.

AU - Janssen, M.C.

AU - van Deursen, Cees Th B.M.

AU - Boonen, A.

AU - Reuser-Kaasenbrood, E.P.

AU - Heeremans, J.

AU - van Kraaij, M.

AU - Masclee, A.

AU - Koek, Ger

PY - 2016/1/1

Y1 - 2016/1/1

N2 - BACKGROUNDPhlebotomy 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 METHODSWe 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 mu g/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.RESULTSThe 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 235Euro for phlebotomy versus 511Euro for erythrocytapheresis. Eighty percent of patients preferred erythrocytapheresis as treatment method.CONCLUSIONErythrocytapheresis 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.

AB - BACKGROUNDPhlebotomy 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 METHODSWe 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 mu g/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.RESULTSThe 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 235Euro for phlebotomy versus 511Euro for erythrocytapheresis. Eighty percent of patients preferred erythrocytapheresis as treatment method.CONCLUSIONErythrocytapheresis 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.

KW - HEREDITARY HEMOCHROMATOSIS

KW - IRON OVERLOAD

KW - IDIOPATHIC HEMOCHROMATOSIS

KW - ARTHROPATHY

KW - ARTHRITIS

KW - THERAPY

KW - HEALTH

KW - ERYTHROPOIETIN

KW - GUIDELINE

KW - DIAGNOSIS

U2 - 10.1111/trf.13328

DO - 10.1111/trf.13328

M3 - Article

VL - 56

SP - 261

EP - 270

JO - Transfusion

JF - Transfusion

SN - 0041-1132

IS - 1

ER -