Big picture initiatives in bleeding disorders.

Publisher: Blackwell Science Country of Publication: England NLM ID: 9442916 Publication Model: Print Cited Medium: Internet ISSN: 1365-2516 (Electronic) Linking ISSN: 13518216 NLM ISO Abbreviation: Haemophilia Subsets: MEDLINE

Original Publication: Osney Mead, Oxford, UK : Blackwell Science, [1994-

Hemophilia A*/therapy ; Quality of Life*; Adult ; Female ; Hemorrhage ; Humans ; Pregnancy ; Rare Diseases

Introduction: The inherited bleeding disorders (IBD) community has witnessed significant therapeutic advances recently, yet important gaps persist, particularly for those with rare disorders and historically underserved populations.
Aims: -To create a national research blueprint agenda, led by the National Hemophilia Foundation (NHF), enhancing patient-centric principles, accelerate research progress and address important gaps in care. -To review critical gaps that remain to be addressed in women with IBDs, who face specific bleeding challenges.
Methods: The NHF research blueprint research agenda was defined by input from across the community, including caregivers and patients who are considered subject matter experts of their IBD, research leaders, allied health professionals and specialists, and representatives of the biopharmaceutical industry. In addition, two medical experts in the field of IBDs performed a comprehensive review to address the knowledge gaps in women with IBDs.
Results: Two foundational principles of the NHF blueprint are: (1) it must deliver on key issues that significantly impact the lives of those affected by IBDs, and (2) the priorities defined are relevant and actionable aimed to achieve health equity among all those affected by IBDs. A multidisciplinary approach is necessary for an optimal management of puberty, transition to adulthood and pregnancy. Even if strict guidelines are followed, recent studies show that women with IBDs are still facing a high burden.
Conclusion: NHF blueprint will be issued in 2022. A specific research agenda is needed in women with IBDs to further improve their management and quality of life.
(© 2022 John Wiley & Sons Ltd.)

Atiq F, Saes JL, Punt MC, et al. WiN, RBiN and TiN study groups. Major differences in clinical presentation, diagnosis and management of men and women with autosomal inherited bleeding disorders. EClinicalMedicine. 2021;32:100726.
Leebeck FW, Eikenboom JC. Von Willebrand's disease. N Engl J Med. 2016;375(21):2067-2080.
Punt MC, Ruigrok ND, Bloemenkamp KWM, Uitslager N, Urbanus RT, Groot E, et al. Trombocytopathy in the Netherlands (TiN) Study Group. Prevalence, burden and treatment effects of vaginal bleeding in women with (suspected) congenital platelet disorders throughout life: a cross-sectional study. Br J Haematol. 2022;196(1):215-223.
Paroskie A, Gailani D, DeBaun MR, Jr SidonioRF. A cross-sectional study of bleeding phenotype in haemophilia A carriers. Br J Haematol. 2015;170(2):223-228.
Radakovic B. Grgic O. Von Willebrand disease and recurrent hematoperitoneum due to the rupture of haemorrhagic ovarian cysts. Haemophilia. 2009;15(2):607-609.
James AH, Jamison MG. Bleeding events and other complications during pregnancy and childbirth in women with von Willebrand disease. J Thromb Haemost. 2007;5(6):1165-1169.
De Wee EM, Knol HM, Mauser-Bunschoten EP, et al. WiN study group. Gynaecological and obstetric bleeding in moderate and severe von Willebrand disease. Thromb Haemost. 2011;106(5):885-892.
Punt MC, Waning ML, Mauser-Bunschoten EP, et al. Maternal and neonatal bleeding complications in relation to peripartum management in women with Von Willebrand disease: a systematic review. Blood Rev. 2020;39:100633.
Munro MG, Critchley HOD, Fraser IS. Committee FMD; FIGO Menstrual Disorders Committee. The two FIGO systems for normal and abnormal uterine bleeding symptoms and classification of causes of abnormal uterine bleeding in the reproductive years: 2018 revisions. Int J Gynaecol Obstet. 2018;143(3):393-408.
Borzutzky C, Jaffray J. Diagnosis and Management of Heavy Menstrual Bleeding and Bleeding Disorders in Adolescents. JAMA Pediatr. 2020;174(2):186-194.
Shankar M, Lee CA, Sabin CA, Economides DL, Kadir RA. von Willebrand disease in women with menorrhagia: a systematic review. BJOG. 2004;111(7):734-740.
Screening and Management of Bleeding Disorders in Adolescents With Heavy Menstrual Bleeding: aCOG COMMITTEE OPINION, Number 785. Obstet Gynecol. 2019; 134(3):e71-e83.
James PD, Connell NT, Ameer B, Di Paola J, Eikenboom J, Giraud N, et al. ASH ISTH NHF WFH 2021 guidelines on the diagnosis of von Willebrand disease. Blood Adv. 2021;5(1):280-300.
Zia A, Jain S, Kouides P, Zhang S, et al. Bleeding disorders in adolescents with heavy menstrual bleeding in a multicenter prospective US cohort. Haematologica. 2020;105(7):1969-1976.
Byams VR, Anderson BL, Grant AM, Atrash H, Schulkin J. Evaluation of bleeding disorders in women with menorrhagia: a survey of obstetrician-gynecologists. Am J Obstet Gynecol. 2012;207(4):269.e1-5.
Powers JM, Stanek JR, Srivaths L, Haamid FW, O'Brien SH. Hematologic considerations and management of adolescent girls with heavy menstrual bleeding and anemia in US children's hospitals. J Pediatr Adolesc Gynecol. 2018;31(5):446-450.
Higham JM, O'Brien PM, Shaw RW. Assessment of menstrual blood loss using a pictorial chart. Br J Obstet Gynaecol. 1990;97(8):734-739.
Dkeidek A, Mattinson R, Grover SR. Bleeding disorders: are gynaecologists testing women at risk?. Haemophilia. 2021;27(4):e534-e536.
James PD. Women and bleeding disorders: diagnostic challenges. Hematology Am Soc Hematol Educ Program. 2020;2020(1):547-552.
James AH. Heavy menstrual bleeding: work-up and management. Hematology Am Soc Hematol Educ Program. 2016(1):236-242.
Mauser-Bunschoten EP, Kadir RA, Laan ETM, Elfvinge P, Haverman L, Teela L, et al. Managing women-specific bleeding in inherited bleeding disorders: a multidisciplinary approach. Haemophilia. 2021;27(3):463-469.
Lee CA, Chi C, Shiltagh N, Pollard D, Griffioen A, Dunn N, et al. Review of a multidisciplinary clinic for women with inherited bleeding disorders. Haemophilia. 2009;15(1):359-360.
Connell NT, Flood VH, Brignardello-Petersen R, Abdul-Kadir R, Arapshian A, Couper S, et al. ASH ISTH NHF WFH 2021 guidelines on the management of von Willebrand disease. Blood Adv. 2021;5(1):301-325.
Bryant-Smith AC, Lethaby A, Farquhar C, Hickey M. Antifibrinolytics for heavy menstrual bleeding. Cochrane Database Syst Rev. 2018;4(4). CD000249.
Kouides PA, Byams VR, Philipp CS, Stein SF, Heit JA, Lukes AS, et al. Multisite management study of menorrhagia with abnormal laboratory haemostasis: a prospective crossover study of intranasal desmopressin and oral tranexamic acid. Br J Haematol. 2009;145(2):212-220.
Ray S, Ray A. Non-surgical interventions for treating heavy menstrual bleeding (menorrhagia) in women with bleeding disorders. Cochrane Database Syst Rev. 2016;11(11). CD010338.
Matteson KA, Rahn DD, Wheeler TL, 2nd, Casiano E, Siddiqui NY. Harvie HSet al; society of gynecologic surgeons systematic review group. nonsurgical management of heavy menstrual bleeding: a systematic review. Obstet Gynecol. 2013;121(3):632-643. 2nd.
Gallo MF, Nanda K, Grimes DA, Lopez LM, Schulz KF. 20 μg versus >20 μg estrogen combined oral contraceptives for contraception. Cochrane Database Syst Rev. 2013;2013(8). CD003989.
Kingman CE, Kadir RA, Lee CA. Economides DL. The use of levonorgestrel-releasing intrauterine system for treatment of menorrhagia in women with inherited bleeding disorders. BJOG. 2004;111(12):1425-1428.
Rimmer E, Jamieson MA, James P. Malposition and expulsion of the levonorgestrel intrauterine system among women with inherited bleeding disorders. Haemophilia. 2013;19(6):933-938.
Moretti D, Goede JS, Zeder C, Jiskra M, Chatzinakou V, Tjalsma H, et al. Oral iron supplements increase hepcidin and decrease iron absorption from daily or twice-daily doses in iron-depleted young women. Blood. 2015;126(17):1981-1989.
Van Galen K, Lavin M, Skouw-Rasmussen N, Fischer K, Noone D, Pollard D, et al, European Haemophilia Consortium (EHC) and the European Association for Haemophilia and Allied Disorders (EAHAD). European principles of care for women and girls with inherited bleeding disorders. Haemophilia. 2021;27(5):837-847.
Leebeek FWG, Duvekot J, Kruip MJHA. How I manage pregnancy in carriers of hemophilia and patients with von Willebrand disease. Blood. 2020;136(19):2143-2150.
Casini A, de Moerloose P. Congenital fibrinogen disorders group. management of congenital quantitative fibrinogen disorders: a delphi consensus. Haemophilia. 2016;22(6):898-905.
Bouttefroy S, Meunier S, Milien V, Boucekine M, Chamouni P, Desprez D, et al. Congenital factor XIII deficiency: comprehensive overview of the francecoag cohort. Br J Haematol. 2020;188(2):317-320. CoDeC study group.
Management of inherited bleeding disorders in Pregnancy: green-top Guideline No. 71 (joint with UKHCDO). BJOG. 2017; 124(8):e193-e263.
MASAC guidelines for perinatal management of women with bleeding disorders and carriers of hemophilia A or B (document 251).
Andersson NG, Chalmers EA, Kenet G, Ljung R, Mäkipernaa A, Chambost H. PedNet haemophilia research foundation. mode of delivery in hemophilia: vaginal delivery and cesarean section carry similar risks for intracranial hemorrhages and other major bleeds. Haematologica. 2019;104(10):2100-2106.
Kouides PA. Antifibrinolytic therapy for preventing VWD-related postpartum hemorrhage: indications and limitations. Blood Adv. 2017;1(11):699-702.
Punt MC, Aalders TH, Bloemenkamp KWM, Driessens MHE, Fischer K, Schrijvers MH, et al. The experiences and attitudes of hemophilia carriers around pregnancy: a qualitative systematic review. J Thromb Haemost. 2020;18(7):1626-1636.
Arya S, Wilton P, Page D, Boma-Fischer L, Floros G, Winikoff R, et al. Everything was blood when it comes to me”: understanding the lived experiences of women with inherited bleeding disorders. J Thromb Haemost. 2020;18(12):3211-3221.
Arya S, Wilton P, Page D, Boma-Fischer L, Floros G, Winikoff R, et al. They don't really take my bleeds seriously”: barriers to care for women with inherited bleeding disorders. J Thromb Haemost. 2021;19(6):1506-1514.
O'Brien SH, Stanek JR, Kaur D, McCracken K, Vesely SK. Laboratory monitoring during pregnancy and post-partum hemorrhage in women with von willebrand disease. J Thromb Haemost. 2020;18(3):604-608.
Roberts JC, Malec LM, Halari I, Hale SA, Oladapo A. Bleeding patterns in patients before and after diagnosis of von willebrand disease: analysis of a US medical claims database. Haemophilia. 2021. http://doi.org/10.1111/hae.14448.
Candy V, Whitworth H, Grabell J, Thibeault L, Harpell L, Bowman M, et al. A decreased and less sustained desmopressin response in hemophilia A carriers contributes to bleeding. Blood Adv. 2018;2(20):2629-2636.
Van Galen KPM, d'Oiron R, James P, Abdul-Kadir R, Kouides PA, Kulkarni R, et al. A new hemophilia carrier nomenclature to define hemophilia in women and girls: communication from the SSC of the ISTH. J Thromb Haemost. 2021;19(8):1883-1887.
Punt MC, Waning ML, Mauser-Bunschoten EP, Kruip MJHA, Eikenboom J, Nieuwenhuizen L, et al. Maternal and neonatal bleeding complications in relation to peripartum management in hemophilia carriers: a systematic review. Blood Rev. 2021;49:100826.
Makris M, Hermans C. The 2021 von willebrand disease guidelines: clarity and controversy. Haemophilia. 2022;28(1):1-3.
Gallinaro L, Cattini MG, Sztukowska M, et al. A shorter von willebrand factor survival in O blood group subjects explains how ABO determinants influence plasma von willebrand factor. Blood. 2008;111(7):3540-3545.
Srivaths L, Minard CG, O'Brien SH, et al. The spectrum and severity of bleeding in adolescents with low von willebrand factor-associated heavy menstrual bleeding. Blood Adv. 2020;4(13):3209-3216.
Lavin M, Aguila S, Dalton N, Nolan M, Byrne M, Ryan K, et al. Significant gynecological bleeding in women with low von willebrand factor levels. Blood Adv. 2018;2(14):1784-1791.

Keywords: heavy menstrual bleeding; inherited bleeding disorders; postpartum haemorrhage; pregnancy

Date Created: 20220506 Date Completed: 20220509 Latest Revision: 20220509

20231215

10.1111/hae.14532

35521733