Primary immunosuppressive TNI-based conditioning regimens in pediatric patients treated with haploidentical hematopoietic cell transplantation.
Strahlenther Onkol
; 198(1): 66-72, 2022 Jan.
Article
em En
| MEDLINE
| ID: mdl-34476532
ABSTRACT
PURPOSE:
This retrospective analysis aims to address the toxicity and efficacy of a modified total nodal irradiation (TNI)-based conditioning regimen before haploidentical hematopoietic cell transplantation (HCT) in pediatric patients. MATERIALS ANDMETHODS:
Patient data including long-term follow-up were evaluated of 7 pediatric patients with malignant (nâ¯= 2) and non-malignant diseases (nâ¯= 5) who were treated by a primary TNI-based conditioning regimen. TNI was performed using anterior/posterior opposing fields. All patients received 7â¯Gy single-dose TNI combined with systemic agents followed by an infusion of peripheral blood stem cells (nâ¯= 7). All children had haploidentical family donors.RESULTS:
Engraftment was reached in 6/7 children after a median time of 9.5 days; 1 child had primary graft failure but was successfully reconditioned shortly thereafter. After an average follow-up time of 103.5 months (range 8.8-138.5 months), event-free (EFS) and overall survival (OS) rates were 71.4% and 85.7%, respectively. One child with a non-malignant disease died 8.8 months after transplantation due to a relapse and a multiple organ failure. Follow-up data was available for 5/6 long-term survivors with a median follow-up (FU) of 106.2 months (range 54.5-138.5 months). Hypothyroidism and deficiency of sexual hormones was present in 3/5 patients each. Mean forced expiratory volume in 1â¯s (FEV1) after TNI was 71%; mean vital capacity (VC) was 78%. Growth failure (<â¯10th percentile) occurred in 2/5 patients (height) and 1/5 patient (weight). No secondary malignancies were reported.CONCLUSION:
In this group of patients, a primary single-dose 7â¯Gy TNI-based conditioning regimen before HCT in pediatric patients allowed sustained engraftment combined with a tolerable toxicity profile leading to long-term OS/EFS. Late toxicity after a median FU of over 9 years includes growth failure, manageable hormonal deficiencies, and acceptable decrease in lung function.Palavras-chave
Texto completo:
1
Coleções:
01-internacional
Base de dados:
MEDLINE
Assunto principal:
Transplante de Células-Tronco Hematopoéticas
/
Doença Enxerto-Hospedeiro
Tipo de estudo:
Etiology_studies
/
Observational_studies
Limite:
Child
/
Humans
Idioma:
En
Revista:
Strahlenther Onkol
Ano de publicação:
2022
Tipo de documento:
Article