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Gene Therapy in Hemophilia

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Gene Therapy in Hemophilia : A Transformational Patient Experience. / Rasul, Enayet; Hallock, Ryan; Hellmann, Magnus; Konduros, Jay; Pembroke, Luke; LeCleir, Gregory; Malacan, Jean; von Mackensen, Sylvia.

in: J PATIENT EXPERIENCE, Jahrgang 10, 2023, S. 23743735231193573.

Publikationen: SCORING: Beitrag in Fachzeitschrift/Zeitung › SCORING: Zeitschriftenaufsatz › Forschung › Begutachtung

Harvard

Rasul, E, Hallock, R, Hellmann, M, Konduros, J, Pembroke, L, LeCleir, G, Malacan, J & von Mackensen, S 2023, 'Gene Therapy in Hemophilia: A Transformational Patient Experience', J PATIENT EXPERIENCE, Jg. 10, S. 23743735231193573. https://doi.org/10.1177/23743735231193573

APA

Rasul, E., Hallock, R., Hellmann, M., Konduros, J., Pembroke, L., LeCleir, G., Malacan, J., & von Mackensen, S. (2023). Gene Therapy in Hemophilia: A Transformational Patient Experience. J PATIENT EXPERIENCE, 10, 23743735231193573. https://doi.org/10.1177/23743735231193573

Vancouver

Rasul E, Hallock R, Hellmann M, Konduros J, Pembroke L, LeCleir G et al. Gene Therapy in Hemophilia: A Transformational Patient Experience. J PATIENT EXPERIENCE. 2023;10:23743735231193573. https://doi.org/10.1177/23743735231193573

Bibtex

@article{853be1f24caf4c19a6d307e900f44aa8,
title = "Gene Therapy in Hemophilia: A Transformational Patient Experience",
abstract = "Hemophilia is a bleeding disorder caused by a single absent/defective gene and characterized by a lack of functional clotting factors. People with hemophilia may experience joint damage, pain, and psychological impairments, all of which could contribute to reduced health-related quality of life (HRQoL). The current standard of care is clotting factor replacement, which is associated with regular infusions; therefore, alternative treatments such as gene therapy (GT) are in development. GT involves the delivery of a functional copy of the clotting factor 8/9 gene by a single infusion into the patient's cells, enabling them to produce their own clotting factor VIII/IX. The impact of treatment on patients' HRQoL can be assessed using hemophilia-specific patient-reported outcome (PRO) measures. Since these measures were designed before the advent of GT, there is a need for updated individualized PRO measures. Patient groups and regulatory authorities emphasize the need for increased patient engagement when considering clinical trial design. Here, we provide patients' perspective on undergoing GT and discuss how to capture the patient voice when measuring the therapy's transformative impact. ",
author = "Enayet Rasul and Ryan Hallock and Magnus Hellmann and Jay Konduros and Luke Pembroke and Gregory LeCleir and Jean Malacan and {von Mackensen}, Sylvia",
note = "{\textcopyright} The Author(s) 2023.",
year = "2023",
doi = "10.1177/23743735231193573",
language = "English",
volume = "10",
pages = "23743735231193573",
journal = "J PATIENT EXPERIENCE",
issn = "2374-3743",
publisher = "Sage",

}

RIS

TY - JOUR

T1 - Gene Therapy in Hemophilia

T2 - A Transformational Patient Experience

AU - Rasul, Enayet

AU - Hallock, Ryan

AU - Hellmann, Magnus

AU - Konduros, Jay

AU - Pembroke, Luke

AU - LeCleir, Gregory

AU - Malacan, Jean

AU - von Mackensen, Sylvia

N1 - © The Author(s) 2023.

PY - 2023

Y1 - 2023

N2 - Hemophilia is a bleeding disorder caused by a single absent/defective gene and characterized by a lack of functional clotting factors. People with hemophilia may experience joint damage, pain, and psychological impairments, all of which could contribute to reduced health-related quality of life (HRQoL). The current standard of care is clotting factor replacement, which is associated with regular infusions; therefore, alternative treatments such as gene therapy (GT) are in development. GT involves the delivery of a functional copy of the clotting factor 8/9 gene by a single infusion into the patient's cells, enabling them to produce their own clotting factor VIII/IX. The impact of treatment on patients' HRQoL can be assessed using hemophilia-specific patient-reported outcome (PRO) measures. Since these measures were designed before the advent of GT, there is a need for updated individualized PRO measures. Patient groups and regulatory authorities emphasize the need for increased patient engagement when considering clinical trial design. Here, we provide patients' perspective on undergoing GT and discuss how to capture the patient voice when measuring the therapy's transformative impact.

AB - Hemophilia is a bleeding disorder caused by a single absent/defective gene and characterized by a lack of functional clotting factors. People with hemophilia may experience joint damage, pain, and psychological impairments, all of which could contribute to reduced health-related quality of life (HRQoL). The current standard of care is clotting factor replacement, which is associated with regular infusions; therefore, alternative treatments such as gene therapy (GT) are in development. GT involves the delivery of a functional copy of the clotting factor 8/9 gene by a single infusion into the patient's cells, enabling them to produce their own clotting factor VIII/IX. The impact of treatment on patients' HRQoL can be assessed using hemophilia-specific patient-reported outcome (PRO) measures. Since these measures were designed before the advent of GT, there is a need for updated individualized PRO measures. Patient groups and regulatory authorities emphasize the need for increased patient engagement when considering clinical trial design. Here, we provide patients' perspective on undergoing GT and discuss how to capture the patient voice when measuring the therapy's transformative impact.

U2 - 10.1177/23743735231193573

DO - 10.1177/23743735231193573

M3 - SCORING: Journal article

C2 - 37663068

VL - 10

SP - 23743735231193573

JO - J PATIENT EXPERIENCE

JF - J PATIENT EXPERIENCE

SN - 2374-3743

ER -