Systematic Review: Efficacy of Medical Therapy on Outcomes Important to Adult Patients with X-Linked Hypophosphatemia

Dalal S Ali; Reza D Mirza; Farah Alsarraf; Salma Hussein; Hajar Abu Alrob; Natasha M Appelman-Dijkstra; Signe Sparre Beck-Nielsen; Martin Biosse-Duplan; Maria Luisa Brandi; Thomas O Carpenter; Catherine Chaussain; Martine Cohen-Solal; Rachel K Crowley; Karel Dandurand; Pablo Florenzano; Seiji Fukumoto; Claudia Gagnon; Paul Goodyer; Corinna Grasemann; Erik A Imel; Suzanne M Jan de Beur; Anna Lehman; E Michael Lewiecki; Emmett Morgante; Leanne M Ward; Aliya A Khan; Gordon Guyatt

doi:10.1210/clinem/dgae890

Systematic Review: Efficacy of Medical Therapy on Outcomes Important to Adult Patients with X-Linked Hypophosphatemia

Dalal S Ali, Reza D Mirza, Farah Alsarraf, Salma Hussein, Hajar Abu Alrob, Natasha M Appelman-Dijkstra, Signe Sparre Beck-Nielsen, Martin Biosse-Duplan, Maria Luisa Brandi, Thomas O Carpenter, Catherine Chaussain, Martine Cohen-Solal, Rachel K Crowley, Karel Dandurand, Pablo Florenzano, Seiji Fukumoto, Claudia Gagnon, Paul Goodyer, Corinna Grasemann, Erik A ImelSuzanne M Jan de Beur, Anna Lehman, E Michael Lewiecki, Emmett Morgante, Leanne M Ward, Aliya A Khan, Gordon Guyatt

Institut for Klinisk Medicin - Børn og Unge

Publikation: Bidrag til tidsskrift/Konferencebidrag i tidsskrift /Bidrag til avis › Review › Forskning › peer review

Abstract

OBJECTIVE: To examine the highest certainty evidence addressing the management of X-linked hypophosphatemia (XLH) in adults to inform treatment recommendations.

METHODS: We searched Embase, MEDLINE, Web of Science, and Cochrane Central up to May 2023. Eligible studies included RCTs and observational studies of individuals 18+ with clinically or genetically confirmed XLH. Manuscripts comparing burosumab to no treatment or conventional therapy (phosphate and active vitamin D) and conventional therapy to no treatment were included. Two reviewers independently determined eligibility, extracted data, and assessed risk of bias (RoB). GRADE methodology was used to assess evidence certainty.

RESULTS: We screened 4,114 records, after removing duplicates, and assessed 254 full texts. One RCT and two observational studies were eligible. The RCT of burosumab versus no treatment had low RoB. Burosumab probably improves pain from fracture/pseudofracture healing (moderate certainty) but has little or no impact on direct pain measures (moderate certainty). Burosumab may reduce the need for parathyroidectomy (low certainty) but has little or no impact on fatigue (high certainty), stiffness (moderate certainty), and mobility (low certainty) over 24 weeks. Burosumab may increase dental abscess risk (low certainty). Indirect evidence comparing burosumab to conventional therapy provided low certainty regarding burosumab versus conventional therapy. Two observational studies on conventional therapy versus no treatment had high RoB and very low certainty regarding the impact of conventional therapy on patient-important outcomes.

CONCLUSION: No formal comparisons between burosumab and conventional therapy in adults exist. Evidence for conventional therapy versus no treatment is very uncertain. Our review highlights the need for more data on the long-term effects of burosumab and conventional therapy on patient-important outcomes in adult patients with XLH.

Originalsprog	Engelsk
Tidsskrift	The Journal of clinical endocrinology and metabolism
ISSN	0021-972X
DOI	https://doi.org/10.1210/clinem/dgae890
Status	E-pub / Early view - 23 dec. 2024

Adgang til dokumentet

10.1210/clinem/dgae890Licens: CC BY-NC-ND

Citationsformater

Ali, D. S., Mirza, R. D., Alsarraf, F., Hussein, S., Abu Alrob, H., Appelman-Dijkstra, N. M., Beck-Nielsen, S. S., Biosse-Duplan, M., Brandi, M. L., Carpenter, T. O., Chaussain, C., Cohen-Solal, M., Crowley, R. K., Dandurand, K., Florenzano, P., Fukumoto, S., Gagnon, C., Goodyer, P., Grasemann, C., ... Guyatt, G. (2024). Systematic Review: Efficacy of Medical Therapy on Outcomes Important to Adult Patients with X-Linked Hypophosphatemia. The Journal of clinical endocrinology and metabolism. Advance online publication. https://doi.org/10.1210/clinem/dgae890

@article{0cdeb31c3f6048739101c1196baf30e9,

title = "Systematic Review: Efficacy of Medical Therapy on Outcomes Important to Adult Patients with X-Linked Hypophosphatemia",

abstract = "OBJECTIVE: To examine the highest certainty evidence addressing the management of X-linked hypophosphatemia (XLH) in adults to inform treatment recommendations.METHODS: We searched Embase, MEDLINE, Web of Science, and Cochrane Central up to May 2023. Eligible studies included RCTs and observational studies of individuals 18+ with clinically or genetically confirmed XLH. Manuscripts comparing burosumab to no treatment or conventional therapy (phosphate and active vitamin D) and conventional therapy to no treatment were included. Two reviewers independently determined eligibility, extracted data, and assessed risk of bias (RoB). GRADE methodology was used to assess evidence certainty.RESULTS: We screened 4,114 records, after removing duplicates, and assessed 254 full texts. One RCT and two observational studies were eligible. The RCT of burosumab versus no treatment had low RoB. Burosumab probably improves pain from fracture/pseudofracture healing (moderate certainty) but has little or no impact on direct pain measures (moderate certainty). Burosumab may reduce the need for parathyroidectomy (low certainty) but has little or no impact on fatigue (high certainty), stiffness (moderate certainty), and mobility (low certainty) over 24 weeks. Burosumab may increase dental abscess risk (low certainty). Indirect evidence comparing burosumab to conventional therapy provided low certainty regarding burosumab versus conventional therapy. Two observational studies on conventional therapy versus no treatment had high RoB and very low certainty regarding the impact of conventional therapy on patient-important outcomes.CONCLUSION: No formal comparisons between burosumab and conventional therapy in adults exist. Evidence for conventional therapy versus no treatment is very uncertain. Our review highlights the need for more data on the long-term effects of burosumab and conventional therapy on patient-important outcomes in adult patients with XLH.",

author = "Ali, {Dalal S} and Mirza, {Reza D} and Farah Alsarraf and Salma Hussein and {Abu Alrob}, Hajar and Appelman-Dijkstra, {Natasha M} and Beck-Nielsen, {Signe Sparre} and Martin Biosse-Duplan and Brandi, {Maria Luisa} and Carpenter, {Thomas O} and Catherine Chaussain and Martine Cohen-Solal and Crowley, {Rachel K} and Karel Dandurand and Pablo Florenzano and Seiji Fukumoto and Claudia Gagnon and Paul Goodyer and Corinna Grasemann and Imel, {Erik A} and {Jan de Beur}, {Suzanne M} and Anna Lehman and Lewiecki, {E Michael} and Emmett Morgante and Ward, {Leanne M} and Khan, {Aliya A} and Gordon Guyatt",

note = "{\textcopyright} The Author(s) 2024. Published by Oxford University Press on behalf of the Endocrine Society.",

year = "2024",

month = dec,

day = "23",

doi = "10.1210/clinem/dgae890",

language = "English",

journal = "The Journal of clinical endocrinology and metabolism",

issn = "0021-972X",

publisher = "Endocrine Society",

}

Ali, DS, Mirza, RD, Alsarraf, F, Hussein, S, Abu Alrob, H, Appelman-Dijkstra, NM, Beck-Nielsen, SS, Biosse-Duplan, M, Brandi, ML, Carpenter, TO, Chaussain, C, Cohen-Solal, M, Crowley, RK, Dandurand, K, Florenzano, P, Fukumoto, S, Gagnon, C, Goodyer, P, Grasemann, C, Imel, EA, Jan de Beur, SM, Lehman, A, Lewiecki, EM, Morgante, E, Ward, LM, Khan, AA & Guyatt, G 2024, 'Systematic Review: Efficacy of Medical Therapy on Outcomes Important to Adult Patients with X-Linked Hypophosphatemia', The Journal of clinical endocrinology and metabolism. https://doi.org/10.1210/clinem/dgae890

TY - JOUR

T1 - Systematic Review

T2 - Efficacy of Medical Therapy on Outcomes Important to Adult Patients with X-Linked Hypophosphatemia

AU - Ali, Dalal S

AU - Mirza, Reza D

AU - Alsarraf, Farah

AU - Hussein, Salma

AU - Abu Alrob, Hajar

AU - Appelman-Dijkstra, Natasha M

AU - Beck-Nielsen, Signe Sparre

AU - Biosse-Duplan, Martin

AU - Brandi, Maria Luisa

AU - Carpenter, Thomas O

AU - Chaussain, Catherine

AU - Cohen-Solal, Martine

AU - Crowley, Rachel K

AU - Dandurand, Karel

AU - Florenzano, Pablo

AU - Fukumoto, Seiji

AU - Gagnon, Claudia

AU - Goodyer, Paul

AU - Grasemann, Corinna

AU - Imel, Erik A

AU - Jan de Beur, Suzanne M

AU - Lehman, Anna

AU - Lewiecki, E Michael

AU - Morgante, Emmett

AU - Ward, Leanne M

AU - Khan, Aliya A

AU - Guyatt, Gordon

PY - 2024/12/23

Y1 - 2024/12/23

N2 - OBJECTIVE: To examine the highest certainty evidence addressing the management of X-linked hypophosphatemia (XLH) in adults to inform treatment recommendations.METHODS: We searched Embase, MEDLINE, Web of Science, and Cochrane Central up to May 2023. Eligible studies included RCTs and observational studies of individuals 18+ with clinically or genetically confirmed XLH. Manuscripts comparing burosumab to no treatment or conventional therapy (phosphate and active vitamin D) and conventional therapy to no treatment were included. Two reviewers independently determined eligibility, extracted data, and assessed risk of bias (RoB). GRADE methodology was used to assess evidence certainty.RESULTS: We screened 4,114 records, after removing duplicates, and assessed 254 full texts. One RCT and two observational studies were eligible. The RCT of burosumab versus no treatment had low RoB. Burosumab probably improves pain from fracture/pseudofracture healing (moderate certainty) but has little or no impact on direct pain measures (moderate certainty). Burosumab may reduce the need for parathyroidectomy (low certainty) but has little or no impact on fatigue (high certainty), stiffness (moderate certainty), and mobility (low certainty) over 24 weeks. Burosumab may increase dental abscess risk (low certainty). Indirect evidence comparing burosumab to conventional therapy provided low certainty regarding burosumab versus conventional therapy. Two observational studies on conventional therapy versus no treatment had high RoB and very low certainty regarding the impact of conventional therapy on patient-important outcomes.CONCLUSION: No formal comparisons between burosumab and conventional therapy in adults exist. Evidence for conventional therapy versus no treatment is very uncertain. Our review highlights the need for more data on the long-term effects of burosumab and conventional therapy on patient-important outcomes in adult patients with XLH.

AB - OBJECTIVE: To examine the highest certainty evidence addressing the management of X-linked hypophosphatemia (XLH) in adults to inform treatment recommendations.METHODS: We searched Embase, MEDLINE, Web of Science, and Cochrane Central up to May 2023. Eligible studies included RCTs and observational studies of individuals 18+ with clinically or genetically confirmed XLH. Manuscripts comparing burosumab to no treatment or conventional therapy (phosphate and active vitamin D) and conventional therapy to no treatment were included. Two reviewers independently determined eligibility, extracted data, and assessed risk of bias (RoB). GRADE methodology was used to assess evidence certainty.RESULTS: We screened 4,114 records, after removing duplicates, and assessed 254 full texts. One RCT and two observational studies were eligible. The RCT of burosumab versus no treatment had low RoB. Burosumab probably improves pain from fracture/pseudofracture healing (moderate certainty) but has little or no impact on direct pain measures (moderate certainty). Burosumab may reduce the need for parathyroidectomy (low certainty) but has little or no impact on fatigue (high certainty), stiffness (moderate certainty), and mobility (low certainty) over 24 weeks. Burosumab may increase dental abscess risk (low certainty). Indirect evidence comparing burosumab to conventional therapy provided low certainty regarding burosumab versus conventional therapy. Two observational studies on conventional therapy versus no treatment had high RoB and very low certainty regarding the impact of conventional therapy on patient-important outcomes.CONCLUSION: No formal comparisons between burosumab and conventional therapy in adults exist. Evidence for conventional therapy versus no treatment is very uncertain. Our review highlights the need for more data on the long-term effects of burosumab and conventional therapy on patient-important outcomes in adult patients with XLH.

U2 - 10.1210/clinem/dgae890

DO - 10.1210/clinem/dgae890

M3 - Review

C2 - 39715351

SN - 0021-972X

JO - The Journal of clinical endocrinology and metabolism

JF - The Journal of clinical endocrinology and metabolism

ER -

Systematic Review: Efficacy of Medical Therapy on Outcomes Important to Adult Patients with X-Linked Hypophosphatemia

Abstract

Adgang til dokumentet

Fingeraftryk

Citationsformater