SUN-741 Bone Mineral Density in Adults With Hypophosphatasia With or Without History of Fractures
Notice bibliographique
Résumé
Abstract Disclosure: L. Seefried: Alexion, AstraZeneca Rare Disease. P.S. Kishnani: Alexion, AstraZeneca Rare Disease. K.M. Dahir: Alexion, AstraZeneca Rare Disease. C. Rockman-Greenberg: Alexion, AstraZeneca Rare Disease. K. Ozono: Alexion, AstraZeneca Rare Disease. G.A. Martos Moreno: Alexion, AstraZeneca Rare Disease. W. Högler: Alexion, AstraZeneca Rare Disease. A. Linglart: Alexion, AstraZeneca Rare Disease. A. Petryk: Alexion, AstraZeneca Rare Disease. S. Fang: Alexion, AstraZeneca Rare Disease. P.R. Ebeling: Alexion, AstraZeneca Rare Disease. Rationale: To describe prevalence of patients with bone mineral density (BMD) T-scores ≤−2.5 by age in adults with hypophosphatasia (HPP) with and without history of fracture (HoF). Methods: Fractures occurring before asfotase alfa treatment (or at any time in untreated patients) were analyzed in younger (aged 18-≤50 y) and older (aged >50 y) adults from the Global HPP Registry (data cut: June 2023). Dual X-ray absorptiometry (DXA) results within 12 months of fracture (or most recent DXA for patients without HoF) were deemed evaluable. Sites analyzed included lumbar spine, hip neck, and total hip. T-scores <−2.5 were interpreted as osteoporosis. BMD T-scores (median [min, max; n), difference in medians (DM), and 95% confidence intervals (CIs) are presented. Results: The study included 278 younger adults (106 with HoF) and 249 older adults (142 with HoF). In younger adults, 17 with HoF and 86 without HoF had an evaluable DXA. Median T-scores were within a normal range in younger adults, although they were lower for those with HoF vs those without HoF at the lumbar spine (−1.2 [−2.8, 1.2; n=13] vs 0 [−1.9, 2.3; n=75]; DM: −1.1; CI: −1.9, −0.4), hip neck (−1.4 [−2.6, 0.6; n=12] vs −0.4 [−2.4, 1.4; n=61]; DM: −0.8; CI: −1.4, −0.1), and total hip (−1.3 [−2.5, 0.4; n=11] vs −0.3 [−2.2, 1.7; n=53]; DM: −0.9; CI: −1.5, −0.3). Few younger adults with HoF had T-scores ≤−2.5 (lumbar spine: 1/13 [8%]; hip neck: 2/12 [17%]; total hip: 1/11 [9%]). No younger adults without HoF had T-scores ≤−2.5. Median age at DXA was 33.9 y in those with HoF and 37.9 y in those without HoF. In older adults, 16 with HoF and 56 without HoF had an evaluable DXA. Median T-scores were within a normal range in older adults and were similar between those with HoF vs those without HoF at the lumbar spine (−0.8 [−4.1, 3.8; n=16] vs −0.8 [−3.8, 5.4; n=51]; DM: −0.3; CI: −1.4, 0.9), hip neck (−1.5 [−3.7, −0.3; n=16] vs −1.4 [−3.5, 0.7; n=44]; DM: 0; CI: −0.6, 0.7), and total hip (−1.6 [−2.9, 0.5; n=13] vs −1.1 [−3.4, 1.7; n=38]; DM: −0.8; CI:−1.5, 0.1). The percent of patients with T-scores ≤−2.5 was similar between those with vs without HoF (lumbar spine: 4/16 [25%] vs 11/51 [22%]; hip neck: 3/16 [19%] vs 13/44 [30%]; total hip: 3/13 [23%] vs 3/38 [8%]). Median age at DXA was similar in those with vs without HoF (60.6 y vs 60.8 y). Metatarsal/femoral fractures characteristic of HPP were present in 10/17 (59%) younger adults and 9/16 (56%) older adults; fractures characteristic of osteoporosis/low BMD (eg, vertebra, hip, wrist, humerus) were present in 5/17 (29%) younger adults and 7/16 (44%) older adults. Conclusions: Median BMD T-scores were within a normal range in adults regardless of fracture history. Some patients may have concomitant osteoporosis in addition to the underlying HPP-related osteomalacia. Further analysis to distinguish fractures due to osteoporosis vs compromised mineralization is warranted. Presentation: Sunday, July 13, 2025
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 | 0,000 | 0,000 |
| Méta-épidémiologie (sens strict) | 0,000 | 0,000 |
| Méta-épidémiologie (sens large) | 0,001 | 0,000 |
| 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 | 0,001 |
| 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 ».