{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,8,18]],"date-time":"2026-08-18T00:51:28Z","timestamp":1787014288969,"version":"build-2736575974"},"reference-count":53,"publisher":"Wiley","issue":"S3","license":[{"start":{"date-parts":[[2024,3,19]],"date-time":"2024-03-19T00:00:00Z","timestamp":1710806400000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Haemophilia"],"published-print":{"date-parts":[[2024,4]]},"abstract":"<jats:title>Abstract<\/jats:title>\n                  <jats:sec>\n                    <jats:title>Introduction<\/jats:title>\n                    <jats:p>Advances in haemophilia treatment have resulted in a near\u2010normal life expectancy, lower burden of bleeding and treatment, and improved quality of life in high\u2010income countries. Bleeding rate is approaching zero and novel parameters should be evaluated to assess the efficacy of treatment not only from the clinical point of view by using new methodologies (e.g. joint health assessment), but also from the patient's perspective (e.g. pain, quality of life, treatment satisfaction).<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Methods and results<\/jats:title>\n                    <jats:p>This approach should be aimed at combining objective clinical methodologies and patient\u2010reported outcomes (PROs). However, some instruments used for assessing PROs are still suboptimal and not properly validated. Recent evidence suggests that these tools can take advantage from a more personalized designed approach and could be effectively improved and serve to facilitate the patient's self\u2010evaluation. For other congenital bleeding disorders (BDs), a set of patient\u2010relevant outcomes has been also defined that overlap substantially those of haemophilia, including bleeding, side effects and complications, and PROs, such as pain, physical functioning, impact on daily life including school and work and mental health. There is a growing focus on addressing women\u2010specific outcomes in BDs, reflecting an increased awareness of the unique challenges faced by women in this context. However, the development of tailored tools is imperative to further advance the progress in managing women with BDs, ensuring more accurate monitoring and personalized care.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Conclusions<\/jats:title>\n                    <jats:p>How incorporating these outcome measures in the process of approval of novel treatments for these disorders by regulatory authorities remains to be established.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.1111\/hae.14990","type":"journal-article","created":{"date-parts":[[2024,3,20]],"date-time":"2024-03-20T00:48:54Z","timestamp":1710895734000},"page":"112-119","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":8,"title":["Outcomes and outcome measures"],"prefix":"10.1111","volume":"30","author":[{"ORCID":"https:\/\/orcid.org\/0000-0003-4973-1317","authenticated-orcid":false,"given":"Giancarlo","family":"Castaman","sequence":"first","affiliation":[{"name":"Center for Bleeding Disorders and Coagulation Department of Oncology Careggi University Hospital  Florence Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-3937-3499","authenticated-orcid":false,"given":"Victor","family":"Jimenez\u2010Yuste","sequence":"additional","affiliation":[{"name":"La Paz University Hospital Autonomous University of Madrid  Madrid Spain"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Samanta","family":"Gouw","sequence":"additional","affiliation":[{"name":"Department of Paediatric Haematology Emma Children's Hospital, Amsterdam UMC University of Amsterdam  Amsterdam The Netherlands"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-4843-7805","authenticated-orcid":false,"given":"Roseline","family":"D'Oiron","sequence":"additional","affiliation":[{"name":"Bic\u00eatre Hospital AP\u2010HP University of Paris\u2010Saclay and UMR_S1176 INSERM Le Kremlin\u2010Bic\u00eatre  Paris France"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2024,3,19]]},"reference":[{"key":"e_1_2_10_2_1","doi-asserted-by":"publisher","DOI":"10.1016\/S0140-6736(20)32722-7"},{"key":"e_1_2_10_3_1","doi-asserted-by":"publisher","DOI":"10.1182\/hematology.2022000397"},{"key":"e_1_2_10_4_1","doi-asserted-by":"publisher","DOI":"10.1111\/hae.14046"},{"key":"e_1_2_10_5_1","doi-asserted-by":"publisher","DOI":"10.1191\/1740774505cn078oa"},{"key":"e_1_2_10_6_1","doi-asserted-by":"publisher","DOI":"10.1002\/rth2.12488"},{"key":"e_1_2_10_7_1","unstructured":"BenemeiS BoniL CastamanG.Outcome measures in hemophilia: current and future perspectives. Submitted."},{"key":"e_1_2_10_8_1","doi-asserted-by":"publisher","DOI":"10.1111\/hae.14943"},{"key":"e_1_2_10_9_1","doi-asserted-by":"publisher","DOI":"10.1111\/hae.14657"},{"key":"e_1_2_10_10_1","doi-asserted-by":"publisher","DOI":"10.1002\/rth2.12690"},{"key":"e_1_2_10_11_1","doi-asserted-by":"publisher","DOI":"10.1111\/hae.14180"},{"key":"e_1_2_10_12_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.jtha.2023.01.035"},{"key":"e_1_2_10_13_1","doi-asserted-by":"publisher","DOI":"10.1160\/TH12\u201011\u20100874"},{"key":"e_1_2_10_14_1","doi-asserted-by":"publisher","DOI":"10.1111\/hae.14368"},{"key":"e_1_2_10_15_1","doi-asserted-by":"publisher","DOI":"10.1002\/jum.14846"},{"key":"e_1_2_10_16_1","doi-asserted-by":"publisher","DOI":"10.1111\/hae.14441"},{"key":"e_1_2_10_17_1","doi-asserted-by":"publisher","DOI":"10.1111\/hae.14280"},{"key":"e_1_2_10_18_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.blre.2020.100781"},{"key":"e_1_2_10_19_1","doi-asserted-by":"publisher","DOI":"10.1186\/s12891-022-06042-w"},{"key":"e_1_2_10_20_1","doi-asserted-by":"publisher","DOI":"10.1111\/hae.14006"},{"key":"e_1_2_10_21_1","doi-asserted-by":"publisher","DOI":"10.1111\/hae.14208"},{"key":"e_1_2_10_22_1","unstructured":"Hemophilia WFH.World Federation of Hemophilia Report on the Annual Global Survey 2022.2022."},{"key":"e_1_2_10_23_1","doi-asserted-by":"publisher","DOI":"10.1002\/rth2.12331"},{"key":"e_1_2_10_24_1","doi-asserted-by":"publisher","DOI":"10.1111\/hae.13504"},{"key":"e_1_2_10_25_1","doi-asserted-by":"publisher","DOI":"10.1111\/hae.13456"},{"key":"e_1_2_10_26_1","doi-asserted-by":"publisher","DOI":"10.1111\/hae.14511"},{"key":"e_1_2_10_27_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.rpth.2023.102201"},{"key":"e_1_2_10_28_1","doi-asserted-by":"publisher","DOI":"10.1111\/hae.14694"},{"issue":"10","key":"e_1_2_10_29_1","article-title":"Routine provision of feedback from patient\u2010reported outcome measurements to healthcare providers and patients in clinical practice","volume":"10","author":"Gibbons C","year":"2021","journal-title":"Cochrane Database Syst Rev"},{"key":"e_1_2_10_30_1","doi-asserted-by":"publisher","DOI":"10.1186\/s41687-021-00324-y"},{"key":"e_1_2_10_31_1","doi-asserted-by":"publisher","DOI":"10.1111\/hae.13197"},{"issue":"1","key":"e_1_2_10_32_1","first-page":"1015","article-title":"Advances in patient\u2010reported outcomes: the NIH PROMIS(\u00ae) measures","volume":"1","author":"Broderick JE","year":"2013","journal-title":"EGEMS (Wash DC)"},{"key":"e_1_2_10_33_1","doi-asserted-by":"publisher","DOI":"10.1111\/hae.14534"},{"key":"e_1_2_10_34_1","doi-asserted-by":"publisher","DOI":"10.1002\/rth2.12621"},{"key":"e_1_2_10_35_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.rpth.2023.102159"},{"key":"e_1_2_10_36_1","doi-asserted-by":"publisher","DOI":"10.1111\/jth.15454"},{"key":"e_1_2_10_37_1","doi-asserted-by":"publisher","DOI":"10.1111\/hae.14321"},{"key":"e_1_2_10_38_1","doi-asserted-by":"crossref","unstructured":"BucknerTW DaoudN LeeL Lim E Schwartz J Raimundo K.Recht M. Blood.2023;142(suppl1):3998.","DOI":"10.1182\/blood-2023-174092"},{"key":"e_1_2_10_39_1","doi-asserted-by":"publisher","DOI":"10.1007\/s11136-020-02522-5"},{"key":"e_1_2_10_40_1","doi-asserted-by":"publisher","DOI":"10.1007\/s11136-021-02950-x"},{"key":"e_1_2_10_41_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.ejogrb.2011.04.024"},{"key":"e_1_2_10_42_1","doi-asserted-by":"publisher","DOI":"10.1111\/hae.14379"},{"key":"e_1_2_10_43_1","doi-asserted-by":"publisher","DOI":"10.1002\/ijgo.12666"},{"key":"e_1_2_10_44_1","doi-asserted-by":"publisher","DOI":"10.3109\/08880018.2012.699165"},{"key":"e_1_2_10_45_1","doi-asserted-by":"publisher","DOI":"10.1111\/j.1471\u20100528.1990.tb02473"},{"key":"e_1_2_10_46_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.jpag.2023.11.011"},{"key":"e_1_2_10_47_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.jpag.2023.10.003"},{"key":"e_1_2_10_48_1","doi-asserted-by":"publisher","DOI":"10.1186\/s12905\u2010020\u20100887\u2010y"},{"key":"e_1_2_10_49_1","doi-asserted-by":"publisher","DOI":"10.1186\/s12905\u2010023\u201002312\u20104"},{"key":"e_1_2_10_50_1","doi-asserted-by":"publisher","DOI":"10.1111\/1471\u20100528.13273"},{"key":"e_1_2_10_51_1","doi-asserted-by":"publisher","DOI":"10.1186\/1472-6874-14-71"},{"key":"e_1_2_10_52_1","volume-title":"WHO Recommendations for the Prevention and Treatment of Postpartum Haemorrhage","author":"World Health Organization","year":"2021"},{"key":"e_1_2_10_53_1","doi-asserted-by":"publisher","DOI":"10.3166\/rmp-2018-0037"},{"key":"e_1_2_10_54_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.ajogmf.2023.101065"}],"container-title":["Haemophilia"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1111\/hae.14990","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2024,4,21]],"date-time":"2024-04-21T03:05:56Z","timestamp":1713668756000},"score":1,"resource":{"primary":{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/10.1111\/hae.14990"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2024,3,19]]},"references-count":53,"journal-issue":{"issue":"S3","published-print":{"date-parts":[[2024,4]]}},"alternative-id":["10.1111\/hae.14990"],"URL":"https:\/\/doi.org\/10.1111\/hae.14990","archive":["Portico"],"relation":{},"ISSN":["1351-8216","1365-2516"],"issn-type":[{"value":"1351-8216","type":"print"},{"value":"1365-2516","type":"electronic"}],"subject":[],"published":{"date-parts":[[2024,3,19]]},"assertion":[{"value":"2024-01-19","order":0,"name":"received","label":"Received","group":{"name":"publication_history","label":"Publication History"}},{"value":"2024-03-01","order":1,"name":"accepted","label":"Accepted","group":{"name":"publication_history","label":"Publication History"}},{"value":"2024-03-19","order":2,"name":"published","label":"Published","group":{"name":"publication_history","label":"Publication History"}}]}}