Outcomes in pediatrics patients diagnosed with bone marrow failure disorders treated in a tertiary care center. / Desenlace de los pacientes pediátricos con falla medular tratados en un centro de alta complejidad.
Rev Chil Pediatr
; 91(4): 545-552, 2020 Aug.
Article
in En, Es
| MEDLINE
| ID: mdl-33399731
ABSTRACT
INTRODUCTION:
Bone marrow failure (BMF) syndromes are rare disorders with an annual incidence of 2-4 cases per million. Treatment options include immunosuppressive therapy (IST) and hematopoietic stem cell transplantation (HSCT).OBJECTIVE:
To analyze the outcomes of pediatric patients diagnosed with BMF treated in a tertiary care center. PATIENTS ANDMETHOD:
Retrospective study of pediatric patients diagnosed with BMF who consulted at Fundación Valle de Lili, Cali. Descriptive statistical analysis was performed according to Acquired BMF (ABMF) and Inherited BMF (IBMF). The outcomes include treatment, complications, overall survival (OS) in transplant patients, calculated using the Kaplan Meier method.RESULTS:
We included 24 patients with BMF, average age 6.5 ± 4 years, and 50% were women. 58% presented IBMF, 9 with Fanconi anemia (FA), 2 dyskeratosis congenita, 2 congenital amegakaryocytic thrombocytopenia, and 1 presented Diamond-Blackfan anemia. 12 patients treated with HSCT had a 5-year OS of 83%. ABMF represented 42%. 6 patients received IST-HSCT, 3 received IST, and 1 received HSCT. The OS of the IST-HSCT group was 86%. Six patients died, four of them related to infection.CONCLUSIONS:
In this series, there was a higher number of cases with IBMF. The OS of patients treated with HSCT is similar to that reported in recent studies. The most frequent cause of death was of infectious origin which has also been previously reported. The treatment esta blished in the patients showed favorable results in a Latin American tertiary care center.
Full text:
1
Collection:
01-internacional
Database:
MEDLINE
Main subject:
Hematopoietic Stem Cell Transplantation
/
Bone Marrow Failure Disorders
/
Immunosuppressive Agents
Type of study:
Diagnostic_studies
/
Observational_studies
/
Risk_factors_studies
Limits:
Adolescent
/
Child
/
Child, preschool
/
Female
/
Humans
/
Infant
/
Male
/
Newborn
Country/Region as subject:
America do sul
/
Colombia
Language:
En
/
Es
Journal:
Rev Chil Pediatr
Year:
2020
Document type:
Article
Affiliation country:
Colombia
Publication country:
CHILE
/
CL