Considerations for shared decision management in previously untreated patients with hemophilia A or B

Abstract

Recent advances in therapeutics are now providing a wide range of options for adults and children living with hemophilia. Although therapeutic choices are also increasing for the youngest individuals with severe disease, challenges remain about early management decisions, as supporting data are currently limited. Parents and healthcare professionals are tasked with helping children achieve an inclusive quality of life and maintain good joint health into adulthood. Primary prophylaxis is the gold standard to optimize outcomes and is recommended to start before 2 years of age. A range of topics need to be discussed with parents to aid their understanding of the decisions they can make and how these will affect the management of their child/children. For those with a family history of hemophilia, prenatal considerations include the possibility of genetic counseling, prenatal investigations, and planning for delivery, together with monitoring of the mother and neonate, as well as diagnosis of the newborn and treatment of any birth-associated bleeding. Subsequent considerations, which are also applicable to families where infant bleeding has resulted in a new diagnosis of sporadic hemophilia, involve explaining bleed recognition and treatment options, practical aspects of initiating/continuing prophylaxis, dealing with bleeds, and ongoing aspects of treatment, including possible inhibitor development. Over time, optimizing treatment efficacy, in which individualizing therapy around activities can play a role, and long-term considerations, including retaining joint health and tolerance maintenance, become increasingly important. The evolving treatment landscape is creating a need for continually updated guidance. Multidisciplinary teams and peers from patient organizations can help provide relevant information. Easily accessible, multidisciplinary comprehensive care remains a foundation to care. Equipping parents early with the knowledge to facilitate truly informed decision-making will help achieve the best possible longer-term health equity and quality of life for the child and family living with hemophilia. Plain language summary Points to be taken into account to help families make decisions to best care for children born with hemophilia Medical advances are providing a range of treatment options for adults and children with hemophilia. There is, however, relatively limited information about managing newborns with the condition. Doctors and nurses can help parents to understand the choices for infants born with hemophilia. We describe the various points doctors and nurses should ideally discuss with families to enable informed decision-making. We focus on infants who require early treatment to prevent spontaneous or traumatic bleeding (prophylaxis), which is recommended to start before 2 years of age. Families with a history of hemophilia may benefit from discussions before pregnancy, including how an affected child would be treated to protect against bleeds. When mothers are pregnant, doctors can explain investigations that can provide information about their unborn child, plan for the birth, and monitor mother and baby to minimize bleed risks at delivery. Testing will confirm whether the baby is affected by hemophilia. Not all infants with hemophilia will be born to families with a history of the condition. Identification of hemophilia for the first time in a family (which is ‘sporadic hemophilia’) occurs in previously undiagnosed infants who have bleeds requiring medical advice and possibly hospital treatment. Before any mothers and babies with hemophilia are discharged from hospital, doctors and nurses will explain to parents how to recognize bleeding and available treatment options can be discussed. Over time, ongoing discussions will help parents to make informed treatment decisions: • When and how to start, then continue, prophylaxis. • How to deal with bleeds (reinforcing previous discussions about bleed recognition and treatment) and other ongoing aspects of treatment. ○ For instance, children may develop neutralizing antibodies (inhibitors) to treatment they are receiving, requiring a change to the planned approach. • Ensuring treatment remains effective as their child grows, considering the varied needs and activities of their child.

Impact of Replacement Therapy on Pregnancy Outcomes in Hemophilia Carriers: A Historical Cohort Study in Saudi Arabia

“…Interestingly, the logistic regression analysis revealed a trend towards increased odds of adverse events when replacement therapy was initiated for surgery preparation (OR = 1.57, p = 0.158). This observation underscores the potential challenges and risks associated with invasive procedures in hemophilia carriers, necessitating careful consideration and planning during replacement therapy [57,58].…”

Shared decision‐making related to treatment of haemophilia: A scoping review of influential factors and available support tools

“…28 Provision of information via tools used by patient organizations and peers, particularly before the initial HCP visit, may therefore help raise awareness regarding novel treatment options and support informed decision-making. 11,40 The findings of this scoping review highlight other potential unmet needs related to treatment SDM in haemophilia. For example, the results emphasize that HCPs and PWH may not perceive and/or be influenced by in-clinic interactions and SDM-related factors in a similar manner.…”

“…8 Simultaneously, treatment selection has become increasingly challenging, being influenced by factors such as safety and efficacy, mechanism of action, administration route, availability, insurance coverage/reimbursement, healthcare practitioner (HCP) preferences and influence, and patient preferences and priorities. [9][10][11][12] Given the complex therapeutic landscape for haemophilia, it is critical that persons with haemophilia (PWH) actively participate in discussions regarding their medical care. 9 Shared decision-making (SDM) is a patient-centred approach involving reciprocal sharing of information between PWH (or their caregiver) and the HCP.…”

Current Status and Challenges in Delivering Comprehensive Care for Patients with Hemophilia

“…In preparing the text, we searched for articles published in PuBMed over the past 10 years using the keywords "hemophilia", "comprehensive care", and "review", and selected five articles to serve as reference materials. [19][20][21][22][23]…”