Human Plasminogen Replacement Therapy Vs. Best Supportive Care in Patients with Congenital Plasminogen Deficiency: A Comparison of Health Care Resource Use over a 48-Week Period
Notice bibliographique
Résumé
Abstract Background: Congenital plasminogen deficiency (CPD), is an ultra-rare autosomal recessive disorder that causes severe and debilitating clinical sequelae. Clinical signs are characterized by ligneous conjunctivitis, gingivitis, otitis media, impaired wound healing and even hydrocephalus. Current disease management includes surgery and systemic/topical therapies, but successful clinical responses are few and recurrences are typically rapid. Intravenous (IV) human plasminogen is a new treatment for CPD currently undergoing regulatory review in the United States. A recent clinical trial demonstrated that regular infusions of plasminogen IV (every 2 to 4 days) induced near complete and long term durable responses in affected patients. The current study compared overall health care resource use required to manage clinical manifestations of CPD for patients treated with human plasminogen in the clinical trial relative to a historical control group. Methods: The registration trial database was used to extract health care resource data in CPD patients (n=14) who received human plasminogen IV therapy throughout a 48-week period. Total health care resource use was compared to a control sample consisting of CPD patients (n=16) who did not receive human plasminogen IV and were managed with only best supportive care (BSC) over a one year period from the date of diagnosis. Health care resource extraction consisted of topical and systemic therapies such as fresh frozen plasma (FFP), clinic visits, surgical interventions and hospital admissions related to CPD. Results: A total of 16 patients who received BSC in either the United States (n=3), Spain (n=3) and Turkey (n=10) were identified and formed the control group. Over a 12-month assessment period, there were 304 clinic visits and 65 days of hospitalization for CPD related complications. IV FFP was given to 12 of 16 (75%) patients, which translated to 246 individual FFP doses administered (dose range = 5 to 15 mg/kg) over 12 months. Furthermore, there were 54 eye surgeries performed on the 16 control patients to remove ligneous membranes. In contrast, none of the 14 patients treated with plasminogen IV required surgical interventions, hospital admissions or FFP infusions over the 48-week treatment period (p < 0.001). There were also significant reductions in all types of supportive therapy in the human plasminogen treated patients relative to their utilization at baseline (p < 0.05). Conclusions: Human plasminogen replacement therapy was able to significantly offset health care resource use compared to patients receiving BSC only. Patients in the trial were also able to incorporate IV human plasminogen therapy into their weekly routine, with administrations often performed at home. Disclosures Dranitsaris: Prometic Life Sciences Inc.: Consultancy. Dorward:Prometic Life Sciences Inc.: Employment.
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 machine sur la base complète
Imitation des enseignantsNi prévalence calibrée, ni vérité terrain. Validation humaine à venir. Le volet Gemma est une étiquette directe du modèle pour chaque travail de la base, lue sur la notice réduite au titre. Le volet Codex est un classifieur appris des 10 348 étiquettes directes de Codex et calibré sur les taux pondérés de l'échantillon; les champs sans appui suffisant ne portent aucun appel Codex. Le mode candidate est l'union des deux volets; le consensus est leur intersection. Ces sorties portent le statut machine_predicted_unvalidated et ne sont pas des étiquettes humaines.
Scores du classifieur distillé par catégorie (deux têtes)
| Catégorie | Codex | Gemma |
|---|---|---|
| Métarecherche | 0,002 | 0,004 |
| Méta-épidémiologie (sens strict) | 0,000 | 0,000 |
| Méta-épidémiologie (sens large) | 0,001 | 0,002 |
| Bibliométrie | 0,001 | 0,002 |
| Études des sciences et des technologies | 0,000 | 0,000 |
| Communication savante | 0,001 | 0,001 |
| Science ouverte | 0,000 | 0,000 |
| Intégrité de la recherche | 0,001 | 0,001 |
| Charge utile insuffisante (le modèle a refusé de juger) | 0,002 | 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 source (Gemma direct ou Codex distillé), 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 ».