12605 Two Systematic Reviews Of Treatment Efficacy On Patient Important Outcomes In Children X-linked Hypophosphatemia
Notice bibliographique
Résumé
Abstract Disclosure: D.S. Ali: None. R. Mirza: None. S. Hussein: None. F. Alsarraf: None. R. Alexander: Consulting Fee; Self; Ultragenyx. Grant Recipient; Self; Ardylex Inc.. H. AbuAlrob: None. M. Brandi: Consulting Fee; Self; Aboca, Alexion, Amolyt, Bruno Farmaceutici, Calcilytix, Echolight, Kyowa Kirin, Personal Genomics,. Speaker; Self; Kyowa Kirin, Abiogen, Alexion, Amgen, Amolyt, Amorphical, Bruno Farmaceutici, Eli Lilly & Company. T.O. Carpenter: Advisory Board Member; Self; Kyowa Kirin, Ultragenyx. Consulting Fee; Self; Kyowa Kirin, Ultragenyx. Grant Recipient; Self; Ultragenyx. Other; Self; Assoc. Editor JBMR, President Ped Endo Soc, Author UpToDate (royalties), XLH-Network (Patient Advisory Org). K. Dandurand: Speaker; Self; Amgen Inc, Kyowa Kirin. G. Filler: Consulting Fee; Self; Kyowa Kirin, Ultragenyx, ProKidney. Speaker; Self; Ultragenyx, ProKidney. P.F. Florenzano: ; Institutional Research Grants: Ultragenyx. ; Advisory Boards: Ultragenyx, Kyowa Kirin. S. Fukumoto: Consulting Fee; Self; Kyowa Kirin. C. Grasemann: Speaker; Self; Kyowa Kirin. E.A. Imel: ; Ultragenyx (Research funding and consulting). ; Kyowa Kirin (research funding and consulting). ; Inozyme (consulting). ; Amgen (research funding). S.M. Jan De Beur: Consulting Fee; Self; Ultragenyx, Kyowa Kirin. Research Investigator; Self; Ultragenyx. E. Morgante: None. L.M. Ward: ; clinical trials with Ultragenyx. ; consultancy to Kyowa Kirin and Ultragenyx. ; unrestricted educational grants from Ultragenyx and Kyowa Kirin (with funds to Dr. Ward’s instituition). A.A. Khan: Advisory Board Member; Self; Amgen Inc, Ascendis, Alexion. Grant Recipient; Self; Takeda, Amolyt, Calcilytix. Speaker; Self; Amgen Inc, Ascendis, Alexion. G. Guyatt: None. Objective: We sought to examine the highest certainty evidence on managing X-linked hypophosphatemia (XLH) in children, aiming to inform treatment recommendations of XLH international guidelines. Data Sources: We searched Embase, MEDLINE, Web of Science, and Cochrane Central from inception to March 2023. We also reviewed reference lists of eligible studies and pertinent review articles. Study eligibility criteria: Eligible studies included randomized controlled trials (RCTs) and observational studies enrolling individuals younger than 18 years old with XLH diagnosed on clinical grounds or with a confirmed pathogenic variant in PHEX. Articles were selected according to specific criteria evaluating the effectiveness of burosumab compared to either no treatment or conventional therapy (phosphate salts and active vitamin D) or evaluating conventional therapy compared to no treatment. Methods: Two reviewers independently determined eligibility, conducted data extraction, and assessed the risk of bias (RoB) in eligible articles. We employed the GRADE methodology to evaluate the certainty of the evidence. Results: After removing duplicates from 7,043 citations, we screened 4,114 records and assessed 254 full texts, of which in the systematic review (SR) addressing burosumab one RCT and one post-Hoc study proved eligible. Being open-label design, the RoB was high, with certainty of evidence on individual outcomes ranging from moderate to very low. Burosumab, compared to conventional therapy, probably prevents lower limb deformity, and improves physical health quality of life (QoL) (moderate certainty). It might also increase height and possibly improve the burden of symptoms related to chronic hypophosphatemia (low certainty). Conversely, burosumab probably increases Treatment-Emergent adverse events after the first administration (moderate certainty), and it may increase dental abscesses (low certainty). In the second SR, one observational study assessing conventional therapy versus no treatment was at high RoB providing very low certainty of evidence regarding the impact of conventional therapy compared to no treatment on final height. Conclusion: Our review indicates that burosumab likely offers benefits in preventing lower limb deformity and improving physical health QoL, while potentially increasing height and improving the burden of symptoms related to chronic hypophosphatemia (low certainty). However, it may also increase adverse events, including dental abscesses. Additionally, our review found limited evidence regarding the impact of conventional therapy compared to no treatment on final height in children. These findings highlight the need for further research to better understand the long-term impact of conventional therapy and burosumab in children. Presentation: 6/2/2024
Récupéré en direct depuis OpenAlex et désinversé. Les résumés ne sont pas conservés dans cette base de données : les index inversés représentent 8,6 Go des 9,3 Go de texte de la base, et le serveur dispose de 13 Go libres.
Comment cette classification a été obtenuedéplier
Prédiction distillée sur la base complète
Imitation des enseignantsNi prévalence calibrée, ni vérité terrain. Validation humaine à venir. Apprise à partir de 10 348 étiquettes directes de Codex et de 10 348 étiquettes directes de Gemma. Le mode candidate est l'union des têtes enseignantes seuillées; le consensus est leur intersection. Ces sorties portent le statut machine_predicted_unvalidated et ne sont ni des étiquettes humaines ni des étiquettes directes de modèles de pointe.
Scores Codex et Gemma par catégorie
| Catégorie | Codex | Gemma |
|---|---|---|
| Métarecherche | 0,001 | 0,001 |
| Méta-épidémiologie (sens strict) | 0,000 | 0,000 |
| Méta-épidémiologie (sens large) | 0,001 | 0,001 |
| Bibliométrie | 0,000 | 0,000 |
| Études des sciences et des technologies | 0,000 | 0,000 |
| Communication savante | 0,000 | 0,000 |
| Science ouverte | 0,000 | 0,000 |
| Intégrité de la recherche | 0,000 | 0,000 |
| Charge utile insuffisante (le modèle a refusé de juger) | 0,000 | 0,000 |
Scores machine (provisoires)
Les deux têtes enseignantes du modèle étudiant, lues sur ce travail. Un score ordonne la base pour la relecture; il n'affirme jamais une catégorie, et le statut de validation accompagne chaque rangée tel quel.
Scores de référence d'un modèle non mature (critères de maturité non atteints, 7 itérations). Un score ordonne; il n'affirme jamais une catégorie.
score_only:v0-immature-baseline · tel quel depuis la passe de notation : score_only signifie que le nombre peut ordonner les travaux, et qu'aucune étiquette de catégorie n'en découleClassification
machine, non validéePrédiction automatique; un appel candidat d’une seule tête enseignante, pas un consensus.
Le détail, modèle par modèle et score par score, se trouve en fin de page sous « Comment cette classification a été obtenue ».