A Iorio1,2, E Iserman1, V Blanchette3, G Dolan4, C Escuriola Ettingshausen5, C Hermans6, C Negrier7, J Oldenburg8, A Reininger9, C Rodriguez-Merchan10, M Spannagl11, L A Valentino12, G Young13, K N Steinitz-Trost9, A Gringeri9. 1. Department of Health Research Methods, Evidence, and Impact, McMaster University, Hamilton, Ontario, Canada. 2. Department of Medicine, McMaster University, Hamilton, Ontario, Canada. 3. Department of Pediatrics, Division of Hematology/Oncology, Hospital for Sick Children, University of Toronto, Toronto, Canada. 4. Guy's and St Thomas' Hospital, London, UK. 5. Haemophilia Centre Rhine Main - HZRM, Moerfelden-Walldorf, Germany. 6. Haemostasis and Thrombosis Unit, Division of Haematology, Cliniques universitaires Saint-Luc, Brussels, Belgium. 7. Division of Haematology, Louis Pradel Cardiology hospital, University Claude Bernard Lyon1, Lyon, France. 8. University Clinic Bonn, Bonn, Germany. 9. Global Medical Affairs Hematology, Baxalta Innovations GmbH, Vienna, Austria. 10. Department of Orthopaedic Surgery, La Paz University Hospital, Madrid, Spain. 11. Department of Transfusion Medicine and Hemostasis, University Hospital Munich, Munich, Germany. 12. Rush University and Baxalta, US, Inc., Deerfield, IL, USA. 13. Children's Hospital Los Angeles, University of Southern California Keck School of Medicine, Los Angeles, CA, USA.
Abstract
BACKGROUND: Prophylactic replacement with factor concentrate is the optimal treatment for persons with severe haemophilia to avoid or minimize bleeding. This ultimately prevents or reduces joint disease and improves life expectancy and quality of life towards values matching those in the normal population. However, uncertainty still exists around the optimal regimens to be prescribed for prophylaxis. An increasing number of treating physicians and patients are showing interest in patient-tailored approaches to prophylaxis, which aim to harmonize the prophylaxis regimen with the patients' bleeding phenotype, levels of physical activity and a variety of other variables. METHODS: A modified Delphi technique was adopted to generate consensus. The expert panel met in person to set the objectives, be trained on the Delphi technique and agree on the desired level of consensus. Three iterations were used to identify the targets, the scenarios and their combinations. RESULTS: Twenty-eight scenarios and eight target levels were identified and used to issue recommendations. The panel reached the desired level of consensus on positive or negative recommendations. Areas where consensus was not reached were identified and proposed as areas for future research. Prospective assessment of the validity of most of the proposed targets is recommended. CONCLUSIONS: We have generated, by expert consensus, target plasma levels of factor concentrate to be used to tailor treatment for persons with haemophilia.
BACKGROUND: Prophylactic replacement with factor concentrate is the optimal treatment for persons with severe haemophilia to avoid or minimize bleeding. This ultimately prevents or reduces joint disease and improves life expectancy and quality of life towards values matching those in the normal population. However, uncertainty still exists around the optimal regimens to be prescribed for prophylaxis. An increasing number of treating physicians and patients are showing interest in patient-tailored approaches to prophylaxis, which aim to harmonize the prophylaxis regimen with the patients' bleeding phenotype, levels of physical activity and a variety of other variables. METHODS: A modified Delphi technique was adopted to generate consensus. The expert panel met in person to set the objectives, be trained on the Delphi technique and agree on the desired level of consensus. Three iterations were used to identify the targets, the scenarios and their combinations. RESULTS: Twenty-eight scenarios and eight target levels were identified and used to issue recommendations. The panel reached the desired level of consensus on positive or negative recommendations. Areas where consensus was not reached were identified and proposed as areas for future research. Prospective assessment of the validity of most of the proposed targets is recommended. CONCLUSIONS: We have generated, by expert consensus, target plasma levels of factor concentrate to be used to tailor treatment for persons with haemophilia.
Authors: Tim Preijers; Laura Bukkems; Max van Spengler; Frank Leebeek; Marjon Cnossen; Ron Mathôt Journal: Eur J Clin Pharmacol Date: 2021-02-24 Impact factor: 2.953
Authors: M Eva Mingot-Castellano; M Teresa Álvarez Román; Luis Fernando Fernández Fuertes; Tomás José González-López; José María Guinea de Castro; Isidro Jarque; M Fernanda López-Fernández; Maria Luisa Lozano; Blanca Sánchez González; David Valcárcel Ferreiras; José Ramón González Porras Journal: Adv Hematol Date: 2019-08-22
Authors: Alfonso Iorio; Andrea N Edginton; Victor Blanchette; Jan Blatny; Ana Boban; Marjon Cnossen; Peter Collins; Stacy E Croteau; Katheljin Fischer; Daniel P Hart; Shinya Ito; Joan Korth-Bradley; Stefan Lethagen; David Lillicrap; Mike Makris; Ron Mathôt; Massimo Morfini; Ellis J Neufeld; Jeffrey Spears Journal: Res Pract Thromb Haemost Date: 2018-05-20