Immunosuppressive Therapy and Future Response to Androgens or Survival After Hematopoietic Stem Cell Transplantation in Fanconi Anemia.
Notice bibliographique
Résumé
Abstract Abstract 1082 Poster Board I-104 Introduction A large proportion of patients with Fanconi anemia (FA) develop aplastic anemia (AA), which is unlikely to respond to immune suppressive therapy (IST). It is not known whether exposure to IST might have a negative effect on patients' response to other therapeutic interventions or their cancer risk. We therefore surveyed FA patients in the United States and Canada to determine whether treatment of AA with IST (perhaps inadvertently prior to the diagnosis of FA) jeopardized subsequent response to anabolic steroids or survival following hematopoietic stem cell transplantation (HSCT), or increased the risk of a malignancy. Methods With the help of Fanconi Anemia Research Fund (FARF) and Fanconi Canada (FC), we sent surveys to 905 adults including patients and their parents or other care providers. A single primary respondent was identified for each patient, using the following priority order: mother, father, adult patient or other care provider; each patient was thus accounted for only once. The survey ascertained age, gender, reason for diagnosis of FA, vital status, treatment, HSCT outcome and development of cancer or leukemia. IST was defined as treatment with cyclosporine A, antithymocyte globulin, or intravenous immunoglobulin. The frequencies of androgen responders, HSCT survivors, and malignancy among FA patients who received IST were compared with those who did not receive IST. Results We received responses representing 224 FA patients. The median age (range) of the patients was 17.3 years (2.6-54.4), the male:female ratio was 114:110, and the overall survival was 144/224 (64%). Single, double or pancytopenia led to the diagnosis of FA in 138 of the 224 patients (62%). Fifty-eight of the 224 patients received IST (26%), 74 received androgens (33%), 121 underwent HSCT (54%), and 23 developed a malignancy (10%). Fifteen patients had solid tumors and 9 had leukemia. One patient developed both a solid tumor and leukemia. Twenty-three of 30 (77%) who received androgens after IST reported a response, compared with 51 of 58 (88%) androgen recipients who had not received IST (p = 0.2). Thirty-two of 53 (60%) patients who had prior IST survived HSCT, while 45 of 68 (66%) without IST for AA also survived HSCT (p = 0.6). Three of 58 (5%) patients developed a malignancy after IST compared with 20 of 166 (12%) without IST (p = 0.2). Conclusion Treatment of AA in a patient who is subsequently diagnosed with FA does not appear to have negative or positive effects on later response to androgens, survival following HSCT, or the risk of malignancy. The limitations of this study are its retrospective nature, the inclusion of multiple family members as the respondents, the inability to ascertain the timing of IST, and its dependence on self-report. The apparent lack of not only harmful but beneficial effects of IST raises questions about its utility for the treatment of AA in FA patients, and suggests that the use of IST may lead to delay in appropriate management. Disclosures No relevant conflicts of interest to declare.
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 | 0,000 | 0,002 |
| Méta-épidémiologie (sens strict) | 0,000 | 0,000 |
| Méta-épidémiologie (sens large) | 0,000 | 0,000 |
| Bibliométrie | 0,000 | 0,001 |
| 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) | 0,002 | 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 ».