Resolution of CGD Related Colitis after Allogeneic Hematopoietic Stem Cell Transplantation in Patients with Chronic Granulomatous Disease—Early Results From the 6903 Study of the Primary Immune Deficiency Treatment Consortium (PIDTC)
Notice bibliographique
Résumé
Background: Phagocytes of patients with CGD have a deficient NADPH oxidase and respiratory burst, which limit production of reactive oxygen species. Allogeneic hematopoietic stem cell transplant (HSCT) offers a potential cure with resolution of life-threatening infections. Approximately 50% of CGD patients develop inflammatory bowel disease similar in pathology to Crohn's disease. CGD related colitis is often difficult to treat and increases disease associated morbidity. We sought to determine if CGD related colitis resolved after HSCT in CGD patients. Methods: Subjects were enrolled on PIDTC protocol 6903, a multi-center retrospective and prospective natural history study of patients treated for CGD since 1995. We evaluated survival, transplant strategy, and the incidence of colitis in CGD patients who received HSCT, at baseline and post-HSCT. The 2-year probability of survival among subjects with or without IBD at baseline was estimated by Kaplan-Meier analysis, and the groups were compared using the log-rank test. Results: 153 subjects underwent HSCT for treatment of CGD during 1/1/2005-6/31/2016 and had known baseline colitis. 53 (35%) had a history of colitis prior to HSCT, and, 17 out of 44 evaluable patients (34%) had disease that was not controlled with medications at the time of HSCT. Conditioning regimens included BuCy (27%), BuFlu (27%), Bu + TBI (19%), FluMel (13%), and others (13%). Of these, 32 (21%), 10 (7%), and 6 (4%) subjects received grafts from HLA-matched sibling donors, other HLA-matched related donors, or mismatched related donors, respectively, while 104 (68%) received grafts from unrelated donors. Graft sources included bone marrow (55%), peripheral blood stem cells (28%) or cord blood (17%). Most subjects (71%) received a calcineurin inhibitor for GVHD prophylaxis with or without 1 or more additional agent(s). The 2-year probability of overall survival of those with or without a history of colitis at baseline was 82% (95% CI 68-90%) vs. 87% (95% CI 78-92%); P = .77. Among the 153 subjects with a history of colitis at baseline and evaluated at 2 years post-HSCT, 90% had resolution of colitis, 5% still had colitis symptoms, and 5% had unknown colitis status. Conclusions: Allogeneic HSCT for CGD was associated with anoverall survival of > 80% at 2 years. For at least 90% of CGD patients with a history of colitis at baseline and evaluable at 2 years post-HSCT, resolution of colitis was observed, consistent with curative treatment for colitis related to CGD. Sponsored by: NIAID and ORDR, NCATS, NIH (U54-AI-082973).
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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) | 0,000 | 0,000 |
| Bibliométrie | 0,000 | 0,000 |
| Études des sciences et des technologies | 0,000 | 0,001 |
| 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 ».