Prophylaxis use of clotting factor replacement products in people with non-severe haemophilia: A review of the literature

Alfonso Iorio; Christoph Königs; Mark T. Reding; Dawn Rotellini; Mark W. Skinner; Maria Elisa Mancuso; Erik Berntorp

doi:10.1111/hae.14676

Prophylaxis use of clotting factor replacement products in people with non-severe haemophilia: A review of the literature

Alfonso Iorio, Christoph Königs, Mark T. Reding, Dawn Rotellini, Mark W. Skinner, Maria Elisa Mancuso, Erik Berntorp

Research output: Contribution to journal › Review article › peer-review

5 Scopus citations

Abstract

Introduction: People with non-severe haemophilia appear to be under-treated in many countries, and this may lead to joint damage and worsen quality of life. Aim: To review literature for clotting factor replacement prophylaxis in people with non-severe haemophilia A and B (HA/HB) in relation to long-term outcomes to support clinical decision-making. Methods: A targeted literature search was performed to identify studies published between 2000 and 2021 that included prophylaxis in people with non-severe HA/HB and long-term outcomes, including annualized bleeding rates, joint health and quality of life. Results: Although eligible articles included 2737 and 2272 people with mild or moderate HA, respectively, only 22% (n = 609) and 29% (n = 668) reported treatment regimens. A total of 549 people with moderate HA were treated with factor replacement prophylaxis and were from high-income countries. On the contrary, nearly all people with mild HA received desmopressin (n = 599). Details of treatment regimens for women with haemophilia and people with HB were sparse. Three studies provided long-term outcomes for people with moderate haemophilia who received prophylaxis with factor concentrate, supporting early prophylaxis in people with a frequent bleeding phenotype regardless of their endogenous clotting factor level to preserve joint health. Conclusion: There remain large knowledge gaps when considering how to provide optimal treatment for people with non-severe haemophilia. Nonetheless, there is a strong rationale that prophylaxis should be considered early in life according to similar strategies as for severe haemophilia for those with a frequent severe bleeding phenotype.

Original language	English (US)
Pages (from-to)	33-44
Number of pages	12
Journal	Haemophilia
Volume	29
Issue number	1
DOIs	https://doi.org/10.1111/hae.14676
State	Published - Jan 2023

Bibliographical note

Funding Information:
The authors acknowledge other members of the Council of the Hemophilia Community (CHC), with whom the concept of the manuscript was discussed (Claude Négrier, Cliff Goodman, Johannes Oldenburg, Brian O'Mahony, Keiji Nogami, Michael Wang). AI, CK, MTR, DR, MS, EB are members of the Council of the Hemophilia Community (CHC), which is organized and funded by Bayer. Medical writing support was provided by Ingrid Koo, PhD, and Celia J. Parkyn, PhD, from Fleishman Hillard. The medical writers conducted the literature searches under the direction of EB, populated a spreadsheet with literature search results under the guidance of EB, attended meetings to take notes on discussions between the authors, collated disclosure statements from authors, and provided writing/editorial support, such as written notes on meeting discussions, journal styling the article and coordinating/facilitating author revisions, and checking the documents required for submission.

Funding Information:
Medical writing support was funded by Bayer, USA.

Funding Information:
AI, CK, MTR, DR, MS, EB are members of the Council of the Hemophilia Community (CHC), which is organized and funded by Bayer.

Funding Information:
MWS's institution has received research funding from BioMarin, Freeline, Roche, Takeda, uniQure; and fees for advisory board or educational presentations from: Bayer, BioMarin, Pfizer (DMC), Roche/Genentech, Sanofi, Spark (DMC), Takeda.

Funding Information:
CK has acted as a paid advisor or speaker for Bayer, CSL Behring, Novo Nordisk, Roche/Chugai, Sobi/Sanofi and Takeda and his institution has received research funding from Bayer, Biotest, EB has acted as paid consultant to Bayer, CSL Behring, Octapharma, Sobi, Takeda, and has received funding for research from Bayer, CSL Behring, Shire/Takeda, Sobi and Bioverativ.

Funding Information:
Medical writing support was provided by Ingrid Koo, PhD, and Celia J. Parkyn, PhD, from Fleishman Hillard, and was funded by Bayer.

Publisher Copyright:
© 2022 The Authors. Haemophilia published by John Wiley & Sons Ltd.

Keywords

arthropathy
bleeding
episodic/on-demand treatment
non-severe (mild or moderate) haemophilia A or B
prophylactic FVIII or FIX
women with haemophilia (symptomatic carriers)

PubMed: MeSH publication types

Journal Article
Review

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10.1111/hae.14676

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@article{1ac28be4f10d49a3b2dcb75b6fddbd99,

title = "Prophylaxis use of clotting factor replacement products in people with non-severe haemophilia: A review of the literature",

abstract = "Introduction: People with non-severe haemophilia appear to be under-treated in many countries, and this may lead to joint damage and worsen quality of life. Aim: To review literature for clotting factor replacement prophylaxis in people with non-severe haemophilia A and B (HA/HB) in relation to long-term outcomes to support clinical decision-making. Methods: A targeted literature search was performed to identify studies published between 2000 and 2021 that included prophylaxis in people with non-severe HA/HB and long-term outcomes, including annualized bleeding rates, joint health and quality of life. Results: Although eligible articles included 2737 and 2272 people with mild or moderate HA, respectively, only 22% (n = 609) and 29% (n = 668) reported treatment regimens. A total of 549 people with moderate HA were treated with factor replacement prophylaxis and were from high-income countries. On the contrary, nearly all people with mild HA received desmopressin (n = 599). Details of treatment regimens for women with haemophilia and people with HB were sparse. Three studies provided long-term outcomes for people with moderate haemophilia who received prophylaxis with factor concentrate, supporting early prophylaxis in people with a frequent bleeding phenotype regardless of their endogenous clotting factor level to preserve joint health. Conclusion: There remain large knowledge gaps when considering how to provide optimal treatment for people with non-severe haemophilia. Nonetheless, there is a strong rationale that prophylaxis should be considered early in life according to similar strategies as for severe haemophilia for those with a frequent severe bleeding phenotype.",

keywords = "arthropathy, bleeding, episodic/on-demand treatment, non-severe (mild or moderate) haemophilia A or B, prophylactic FVIII or FIX, women with haemophilia (symptomatic carriers)",

author = "Alfonso Iorio and Christoph K{\"o}nigs and Reding, {Mark T.} and Dawn Rotellini and Skinner, {Mark W.} and Mancuso, {Maria Elisa} and Erik Berntorp",

note = "Funding Information: The authors acknowledge other members of the Council of the Hemophilia Community (CHC), with whom the concept of the manuscript was discussed (Claude N{\'e}grier, Cliff Goodman, Johannes Oldenburg, Brian O'Mahony, Keiji Nogami, Michael Wang). AI, CK, MTR, DR, MS, EB are members of the Council of the Hemophilia Community (CHC), which is organized and funded by Bayer. Medical writing support was provided by Ingrid Koo, PhD, and Celia J. Parkyn, PhD, from Fleishman Hillard. The medical writers conducted the literature searches under the direction of EB, populated a spreadsheet with literature search results under the guidance of EB, attended meetings to take notes on discussions between the authors, collated disclosure statements from authors, and provided writing/editorial support, such as written notes on meeting discussions, journal styling the article and coordinating/facilitating author revisions, and checking the documents required for submission. Funding Information: Medical writing support was funded by Bayer, USA. Funding Information: AI, CK, MTR, DR, MS, EB are members of the Council of the Hemophilia Community (CHC), which is organized and funded by Bayer. Funding Information: MWS's institution has received research funding from BioMarin, Freeline, Roche, Takeda, uniQure; and fees for advisory board or educational presentations from: Bayer, BioMarin, Pfizer (DMC), Roche/Genentech, Sanofi, Spark (DMC), Takeda. Funding Information: CK has acted as a paid advisor or speaker for Bayer, CSL Behring, Novo Nordisk, Roche/Chugai, Sobi/Sanofi and Takeda and his institution has received research funding from Bayer, Biotest, EB has acted as paid consultant to Bayer, CSL Behring, Octapharma, Sobi, Takeda, and has received funding for research from Bayer, CSL Behring, Shire/Takeda, Sobi and Bioverativ. Funding Information: Medical writing support was provided by Ingrid Koo, PhD, and Celia J. Parkyn, PhD, from Fleishman Hillard, and was funded by Bayer. Publisher Copyright: {\textcopyright} 2022 The Authors. Haemophilia published by John Wiley & Sons Ltd.",

year = "2023",

month = jan,

doi = "10.1111/hae.14676",

language = "English (US)",

volume = "29",

pages = "33--44",

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T1 - Prophylaxis use of clotting factor replacement products in people with non-severe haemophilia

T2 - A review of the literature

AU - Iorio, Alfonso

AU - Königs, Christoph

AU - Reding, Mark T.

AU - Rotellini, Dawn

AU - Skinner, Mark W.

AU - Mancuso, Maria Elisa

AU - Berntorp, Erik

N1 - Funding Information: The authors acknowledge other members of the Council of the Hemophilia Community (CHC), with whom the concept of the manuscript was discussed (Claude Négrier, Cliff Goodman, Johannes Oldenburg, Brian O'Mahony, Keiji Nogami, Michael Wang). AI, CK, MTR, DR, MS, EB are members of the Council of the Hemophilia Community (CHC), which is organized and funded by Bayer. Medical writing support was provided by Ingrid Koo, PhD, and Celia J. Parkyn, PhD, from Fleishman Hillard. The medical writers conducted the literature searches under the direction of EB, populated a spreadsheet with literature search results under the guidance of EB, attended meetings to take notes on discussions between the authors, collated disclosure statements from authors, and provided writing/editorial support, such as written notes on meeting discussions, journal styling the article and coordinating/facilitating author revisions, and checking the documents required for submission. Funding Information: Medical writing support was funded by Bayer, USA. Funding Information: AI, CK, MTR, DR, MS, EB are members of the Council of the Hemophilia Community (CHC), which is organized and funded by Bayer. Funding Information: MWS's institution has received research funding from BioMarin, Freeline, Roche, Takeda, uniQure; and fees for advisory board or educational presentations from: Bayer, BioMarin, Pfizer (DMC), Roche/Genentech, Sanofi, Spark (DMC), Takeda. Funding Information: CK has acted as a paid advisor or speaker for Bayer, CSL Behring, Novo Nordisk, Roche/Chugai, Sobi/Sanofi and Takeda and his institution has received research funding from Bayer, Biotest, EB has acted as paid consultant to Bayer, CSL Behring, Octapharma, Sobi, Takeda, and has received funding for research from Bayer, CSL Behring, Shire/Takeda, Sobi and Bioverativ. Funding Information: Medical writing support was provided by Ingrid Koo, PhD, and Celia J. Parkyn, PhD, from Fleishman Hillard, and was funded by Bayer. Publisher Copyright: © 2022 The Authors. Haemophilia published by John Wiley & Sons Ltd.

PY - 2023/1

Y1 - 2023/1

N2 - Introduction: People with non-severe haemophilia appear to be under-treated in many countries, and this may lead to joint damage and worsen quality of life. Aim: To review literature for clotting factor replacement prophylaxis in people with non-severe haemophilia A and B (HA/HB) in relation to long-term outcomes to support clinical decision-making. Methods: A targeted literature search was performed to identify studies published between 2000 and 2021 that included prophylaxis in people with non-severe HA/HB and long-term outcomes, including annualized bleeding rates, joint health and quality of life. Results: Although eligible articles included 2737 and 2272 people with mild or moderate HA, respectively, only 22% (n = 609) and 29% (n = 668) reported treatment regimens. A total of 549 people with moderate HA were treated with factor replacement prophylaxis and were from high-income countries. On the contrary, nearly all people with mild HA received desmopressin (n = 599). Details of treatment regimens for women with haemophilia and people with HB were sparse. Three studies provided long-term outcomes for people with moderate haemophilia who received prophylaxis with factor concentrate, supporting early prophylaxis in people with a frequent bleeding phenotype regardless of their endogenous clotting factor level to preserve joint health. Conclusion: There remain large knowledge gaps when considering how to provide optimal treatment for people with non-severe haemophilia. Nonetheless, there is a strong rationale that prophylaxis should be considered early in life according to similar strategies as for severe haemophilia for those with a frequent severe bleeding phenotype.

AB - Introduction: People with non-severe haemophilia appear to be under-treated in many countries, and this may lead to joint damage and worsen quality of life. Aim: To review literature for clotting factor replacement prophylaxis in people with non-severe haemophilia A and B (HA/HB) in relation to long-term outcomes to support clinical decision-making. Methods: A targeted literature search was performed to identify studies published between 2000 and 2021 that included prophylaxis in people with non-severe HA/HB and long-term outcomes, including annualized bleeding rates, joint health and quality of life. Results: Although eligible articles included 2737 and 2272 people with mild or moderate HA, respectively, only 22% (n = 609) and 29% (n = 668) reported treatment regimens. A total of 549 people with moderate HA were treated with factor replacement prophylaxis and were from high-income countries. On the contrary, nearly all people with mild HA received desmopressin (n = 599). Details of treatment regimens for women with haemophilia and people with HB were sparse. Three studies provided long-term outcomes for people with moderate haemophilia who received prophylaxis with factor concentrate, supporting early prophylaxis in people with a frequent bleeding phenotype regardless of their endogenous clotting factor level to preserve joint health. Conclusion: There remain large knowledge gaps when considering how to provide optimal treatment for people with non-severe haemophilia. Nonetheless, there is a strong rationale that prophylaxis should be considered early in life according to similar strategies as for severe haemophilia for those with a frequent severe bleeding phenotype.

KW - arthropathy

KW - bleeding

KW - episodic/on-demand treatment

KW - non-severe (mild or moderate) haemophilia A or B

KW - prophylactic FVIII or FIX

KW - women with haemophilia (symptomatic carriers)

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Prophylaxis use of clotting factor replacement products in people with non-severe haemophilia: A review of the literature

Abstract

Bibliographical note

Keywords

PubMed: MeSH publication types

Publisher link

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