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

J 2023

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

ASTERMARK, Jan, Jan BLATNÝ, Christoph KOENIGS, Cedric HERMANS, Victor JIMENEZ-YUSTE et. al.

Basic information

Original name

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

Authors

ASTERMARK, Jan, Jan BLATNÝ (203 Czech Republic, belonging to the institution), Christoph KOENIGS, Cedric HERMANS, Victor JIMENEZ-YUSTE and Daniel P HART

Edition

THERAPEUTIC ADVANCES IN HEMATOLOGY, London, SAGE PUBLICATIONS LTD, 2023, 2040-6207

Other information

Language

English

Type of outcome

Článek v odborném periodiku

Field of Study

30205 Hematology

Country of publisher

United Kingdom of Great Britain and Northern Ireland

Confidentiality degree

není předmětem státního či obchodního tajemství

References:

URL

Impact factor

Impact factor: 3.400 in 2022

RIV identification code

RIV/00216224:14110/23:00133676

Organization unit

Faculty of Medicine

DOI

http://dx.doi.org/10.1177/20406207231165857

UT WoS

000971562400001

Keywords in English

hemophilia; previously untreated patient; prophylaxis; therapy

Abstract

V originále

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 summaryPoints to be taken into account to help families make decisions to best care for children born with hemophiliaMedical 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.

Citovat

ASTERMARK, Jan, Jan BLATNÝ, Christoph KOENIGS, Cedric HERMANS, Victor JIMENEZ-YUSTE and Daniel P HART. Considerations for shared decision management in previously untreated patients with hemophilia A or B. THERAPEUTIC ADVANCES IN HEMATOLOGY. London: SAGE PUBLICATIONS LTD, 2023, vol. 14, January-December 2023, p. 1-21. ISSN 2040-6207. Available from: https://dx.doi.org/10.1177/20406207231165857.

@article{2379582,
   author = {Astermark, Jan and Blatný, Jan and Koenigs, Christoph and Hermans, Cedric and JimenezandYuste, Victor and Hart, Daniel P},
   article_location = {London},
   article_number = {January-December 2023},
   doi = {http://dx.doi.org/10.1177/20406207231165857},
   keywords = {hemophilia; previously untreated patient; prophylaxis; therapy},
   language = {eng},
   issn = {2040-6207},
   journal = {THERAPEUTIC ADVANCES IN HEMATOLOGY},
   title = {Considerations for shared decision management in previously untreated patients with hemophilia A or B},
   url = {https://journals.sagepub.com/doi/10.1177/20406207231165857},
   volume = {14},
   year = {2023}
}

TY  - JOUR
ID  - 2379582
AU  - Astermark, Jan - Blatný, Jan - Koenigs, Christoph - Hermans, Cedric - Jimenez-Yuste, Victor - Hart, Daniel P
PY  - 2023
TI  - Considerations for shared decision management in previously untreated patients with hemophilia A or B
JF  - THERAPEUTIC ADVANCES IN HEMATOLOGY
VL  - 14
IS  - January-December 2023
SP  - 1-21
EP  - 1-21
PB  - SAGE PUBLICATIONS LTD
SN  - 20406207
KW  - hemophilia
KW  - previously untreated patient
KW  - prophylaxis
KW  - therapy
UR  - https://journals.sagepub.com/doi/10.1177/20406207231165857
N2  - 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 summaryPoints to be taken into account to help families make decisions to best care for children born with hemophiliaMedical 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.
ER  -

ASTERMARK, Jan, Jan BLATNÝ, Christoph KOENIGS, Cedric HERMANS, Victor JIMENEZ-YUSTE and Daniel P HART. Considerations for shared decision management in previously untreated patients with hemophilia A or B. \textit{THERAPEUTIC ADVANCES IN HEMATOLOGY}. London: SAGE PUBLICATIONS LTD, 2023, vol.~14, January-December 2023, p.~1-21. ISSN~2040-6207. Available from: https://dx.doi.org/10.1177/20406207231165857.

Detailed Information on Publication Record