Bone Marrow Transplantation From HLA-Identical Sibling for Thalassemia.
Notice bibliographique
Résumé
Abstract Abstract 3361 Poster Board III-249 Allogeneic bone marrow transplantation (BMT) is curative treatment for β-Thalassemia major. The most extensive patient series have been reported by transplant teams in Italy. Outside these large groups, smaller, more limited series have reported variable results. Here we report the current experience of BMT for thalassemia outside Italy, and describe the characteristics and transplant outcomes in children after HLA-identical sibling BMT for thalassemia. Patients aged ≤20 years at BMT who received a myeloablative HLA-matched sibling BMT for thalassemia between 1995 and 2001 at 19 centers were eligible for the survey (n=179). Recipients of mobilized peripheral blood or umbilical cord blood stem cells, and reduced intensity transplant preparation were excluded as there were too few patients in each category for analysis. Also excluded were patients previously reported by the Italian centers. The median age at BMT was 7 years and the median follow-up of survivors was 6 years. Most recipients had high-risk features at BMT. The distribution of Lucarelli risk class I, II and III categories was 2%, 42%; and 36%, respectively. Data required to assign a risk category was not available for 20% of the study population. Before BMT, 85% had received ≥ 20 red blood cell transfusions. Of 87% who had a pre-BMT liver biopsy, 59% had portal fibrosis. There was hepatomegaly in 52% of patients and 94% had inadequate iron chelation therapy. All patients received busulfan and cyclophosphamide with or without anti-thymocyte globulin as the transplant conditioning regimen and almost all received cyclosporine-containing GVHD prophylaxis. The overall survival (OS) probabilities at 100 days and at 5 years were 85% and 80%, respectively. Seventeen patients (9.5%) had graft failure that was fatal in 11 cases. Six of 9 patients with graft failure survive after a successful second transplantation. Thirty-five patients (20%) died after BMT and the leading cause of death was interstitial pneumonitis in the current analysis. The probability of acute GVHD (grade II-IV) was 38% and the 5-year probability of chronic GVHD was 13%. Veno-occlusive disease (VOD) occurred frequently after BMT (n=58; 32% of patients) and 21 of the 35 (57%) patients who died after BMT had had a history VOD. A multivariate analysis showed that age at BMT ≥10 years (RR 2.16, 95% CI 1.1 – 4.23, p=0.025) and baseline hepatomegaly (RR 5.57, 95% CI 2.14 – 14.49, p<0.001) were associated with higher risk of mortality. The probabilities of disease-free survival (DFS) and OS are shown in the Table below. Younger patients without hepatomegaly fared the best and, older patients with hepatomegaly, the worst. This series confirms reports from large single center series that the results after HLA-matched sibling BMT for thalassemia major are optimized when performed in young children before the development of risk-factors for poor outcome. Pulmonary and hepatic complications were the leading cause of death in high-risk patients. 5-year OS 5-year DFS Age < 10 years without hepatomegaly 96% 93% Age < 10 years with hepatomegaly 73% 69% Age ≥ 10 years without hepatomegaly 86% 81% Age ≥ 10 years with hepatomegaly 53% 53% 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 | 0,000 |
| Méta-épidémiologie (sens strict) | 0,000 | 0,000 |
| Méta-épidémiologie (sens large) | 0,000 | 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 | 0,000 |
| Charge utile insuffisante (le modèle a refusé de juger) | 0,003 | 0,001 |
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 ».