{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,7,7]],"date-time":"2026-07-07T19:03:54Z","timestamp":1783451034452,"version":"3.55.0"},"reference-count":0,"publisher":"Georg Thieme Verlag KG","issue":"03","license":[{"start":{"date-parts":[[2019,1,27]],"date-time":"2019-01-27T00:00:00Z","timestamp":1548547200000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Thromb Haemost"],"published-print":{"date-parts":[[2019,3]]},"abstract":"<jats:p>\n            Background\u2003Performing individual pharmacokinetics (PK) studies in clinical practice can be simplified by adopting population PK-based profiling on limited post-infusion samples. The objective of this study was to assess the impact of population PK in tailoring prophylaxis in patients with haemophilia A.<\/jats:p><jats:p>\n            Patients and Methods\u2003Individual weekly treatment plans were developed considering predicted plasma factor activity levels and patients' lifestyle. Patients were trained using a visual traffic-light scheme to help modulate their level of physical activity with respect to factor infusions timing. Annualized joint bleeding rate (ABJR), haemophilia-specific quality of life questionnaire for adults (Haemo-QoL-A) and factor utilization were measured for 12 months before and after tailoring, compared within patients and analysed separately for those previously on prophylaxis (P), situational prophylaxis (SP) or on-demand (OD).<\/jats:p><jats:p>\n            Results\u2003Sixteen patients previously on P, 10 on SP and 10 on OD were enrolled in the study. The median (lower, upper quartile) ABJR changed from 2.0 (0, 4.0) to 0 (0, 1.6) for P (p\u2009=\u20090.003), from 2.0 (2.0, 13.6) to 3.0 (1.4, 7.2) for SP (p\u2009=\u20090.183) and from 16.0 (13.0, 25.0) to 2.3 (0, 5.0) for OD (p\u2009=\u20090.003). The Haemo-QoL-A total score improved for 58% of P, 50% of SP and 29% of OD patients. Factor utilization (IU\/kg\/patient\/year) increased by 2,400 (121; 2,586) for P, 1,052 (308; 1,578) for SP and 2,086 (1,498; 2,576) for OD. One of 138 measurements demonstrated a factor activity level below the critical threshold of 0.03 IU\/mL while the predicted level was above the threshold.<\/jats:p><jats:p>\n            Conclusion\u2003Implementing tailored prophylaxis using a Bayesian forecasting approach in a routine clinical practice setting may improve haemophilia clinical outcomes.<\/jats:p>","DOI":"10.1055\/s-0039-1677700","type":"journal-article","created":{"date-parts":[[2019,1,27]],"date-time":"2019-01-27T18:31:57Z","timestamp":1548613917000},"page":"368-376","source":"Crossref","is-referenced-by-count":23,"title":["Impact of Adopting Population Pharmacokinetics for Tailoring Prophylaxis in Haemophilia A Patients: A Historically Controlled Observational Study"],"prefix":"10.1055","volume":"119","author":[{"given":"Michaela","family":"Stemberger","sequence":"additional","affiliation":[{"name":"Medizinische Klinik und Poliklinik IV, Klinikum der Universit\u00e4t M\u00fcnchen, Munich, Germany"},{"name":"Abteilung f\u00fcr Transfusionsmedizin, Zelltherapeutika und H\u00e4mostaseologie, Klinikum der Universit\u00e4t M\u00fcnchen, Munich, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Felix","family":"Kallenbach","sequence":"additional","affiliation":[{"name":"Abteilung f\u00fcr Transfusionsmedizin, Zelltherapeutika und H\u00e4mostaseologie, Klinikum der Universit\u00e4t M\u00fcnchen, Munich, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Elisabeth","family":"Schmit","sequence":"additional","affiliation":[{"name":"Abteilung f\u00fcr Transfusionsmedizin, Zelltherapeutika und H\u00e4mostaseologie, Klinikum der Universit\u00e4t M\u00fcnchen, Munich, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Alanna","family":"McEneny-King","sequence":"additional","affiliation":[{"name":"School of Pharmacy, University of Waterloo, Waterloo, Ontario, Canada"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Federico","family":"Germini","sequence":"additional","affiliation":[{"name":"Department of Health Research Methods, Evidence, and Impact, McMaster University, Hamilton, Ontario, Canada"},{"name":"Department of Health Sciences, Universit\u00e0 degli Studi di Milano, Milan, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Cindy","family":"Yeung","sequence":"additional","affiliation":[{"name":"Department of Health Research Methods, Evidence, and Impact, McMaster University, Hamilton, Ontario, Canada"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Andrea","family":"Edginton","sequence":"additional","affiliation":[{"name":"School of Pharmacy, University of Waterloo, Waterloo, Ontario, Canada"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sylvia","family":"von Mackensen","sequence":"additional","affiliation":[{"name":"Institut und Poliklinik f\u00fcr Medizinische Psychologie, Universit\u00e4tsklinikum Hamburg-Eppendorf, Hamburg, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Karin","family":"Kurnik","sequence":"additional","affiliation":[{"name":"Zentrum f\u00fcr P\u00e4diatrische H\u00e4mostaseologie, Dr. von Haunersches Kinderspital, Klinikum der Universit\u00e4t M\u00fcnchen, Munich, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Alfonso","family":"Iorio","sequence":"additional","affiliation":[{"name":"Department of Health Research Methods, Evidence, and Impact, McMaster University, Hamilton, Ontario, Canada"},{"name":"McMaster-Bayer Chair for Clinical Epidemiology Research in Bleeding Disorders, Department of Medicine, McMaster University, Hamilton, Ontario, Canada"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"194","published-online":{"date-parts":[[2019,1,27]]},"container-title":["Thrombosis and Haemostasis"],"original-title":[],"language":"en","link":[{"URL":"http:\/\/www.thieme-connect.de\/products\/ejournals\/pdf\/10.1055\/s-0039-1677700.pdf","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2019,5,6]],"date-time":"2019-05-06T17:20:41Z","timestamp":1557163241000},"score":1,"resource":{"primary":{"URL":"http:\/\/www.thieme-connect.de\/DOI\/DOI?10.1055\/s-0039-1677700"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2019,1,27]]},"references-count":0,"journal-issue":{"issue":"03","published-online":{"date-parts":[[2019,2,25]]},"published-print":{"date-parts":[[2019,3]]}},"URL":"https:\/\/doi.org\/10.1055\/s-0039-1677700","relation":{},"ISSN":["0340-6245","2567-689X"],"issn-type":[{"value":"0340-6245","type":"print"},{"value":"2567-689X","type":"electronic"}],"subject":[],"published":{"date-parts":[[2019,1,27]]}}}