Clinical trial NCT03752372: Microbiome Alterations in IL10RA-deficient Patients After HSCT
Official title: Longitudinal Development of Intestinal Microbiome in IL10RA-deficient Patients After Hematopoietic Stem Cell Transplantation
Study type: OBSERVATIONAL
Overall status: TERMINATED
Conditions: Inflammatory Bowel Diseases
Microbiota
Keywords: interleukin 10 receptor alpha gene
Lead sponsor: Children's Hospital of Fudan University
Sponsor class: OTHER
First posted: 2018-11-26
Enrollment: 13
Sex: ALL
Minimum age (years): 1
Maximum age (years): 6
Standard ages: CHILD
Accepts healthy volunteers: yes
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Patients who were diagnosed with IL10RA deficiency and who received the hematopoietic stem cell transplantation in a tertiary hospital were enrolled.

Brief summary: To elucidate the longitudinal development of intestinal microbiota in patients with IL10RA deficiency after hematopoietic stem cell transplantation (HSCT). The investigators planned to collect fecal samples from IL10RA-deficient patients who received HSCT. Samples were collected more than once every three days after engraftment in lamina flow ward and at least once a week before discharge. Microbial DNA was extracted from the fecal samples. And all analysis was based on the next generation sequencing data.

Detailed description: Hematopoietic stem cell transplantation is considered the only curative therapy for patients with interleukin-10 receptor-A(IL10RA) deficiency. The investigators aimed to collect the fecal samples of these patients throughout the conditioning, transplantation until discharge, providing a dense insight into the longitudinal development of intestinal microbiota.

Eligibility criteria: Inclusion Criteria:

* IL10RA gene mutations;
* Eligible for hematopoietic stem cell transplantation.

Exclusion Criteria:

* Unwilling to participate.

Primary outcomes:
measure: Shannon indices
timeFrame: during the hospitalization of each eligible patient, an average of 90 days per patient
measure: Simpson indices
timeFrame: during the hospitalization of each eligible patient, an average of 90 days per patient
measure: relative abundance of Proteobacteria
timeFrame: during the hospitalization of each eligible patient, an average of 90 days per patient
measure: relative abundance of Firmicutes
timeFrame: during the hospitalization of each eligible patient, an average of 90 days per patient
measure: Clostridium sensus_stricto_1
timeFrame: during the hospitalization of each eligible patient, an average of 90 days per patient
measure: Escherichia - Shigella
timeFrame: during the hospitalization of each eligible patient, an average of 90 days per patient
Secondary outcomes:
measure: chimerism
timeFrame: during the hospitalization of each eligible patient, an average of 90 days per patient
measure: manifestation of graft-versus-host disease
timeFrame: during the hospitalization of each eligible patient, an average of 90 days per patient
measure: Sobs indices
timeFrame: during the hospitalization of each eligible patient, an average of 90 days per patient
measure: Chao1 estimator
timeFrame: during the hospitalization of each eligible patient, an average of 90 days per patient
measure: neutrophil cell count
timeFrame: during the hospitalization of each eligible patient, an average of 90 days per patient
measure: platelet count
timeFrame: during the hospitalization of each eligible patient, an average of 90 days per patient
Other outcomes:
measure: relative abundance of Clostridiales
timeFrame: during the hospitalization of each eligible patient, an average of 90 days per patient
measure: Shannoneven indices
timeFrame: during the hospitalization of each eligible patient, an average of 90 days per patient
measure: Simpsoneven indices
timeFrame: during the hospitalization of each eligible patient, an average of 90 days per patient
Locations:
city: Shanghai
country: China
facility: Children's hospital of Fudan university
state: Shanghai
Study officials:
affiliation: Children's Hospital of Fudan University
name: Ying Huang, MD
role: PRINCIPAL_INVESTIGATOR
References:
citation: Peng K, Qian X, Huang Z, Lu J, Wang Y, Zhou Y, Wang H, Wu B, Wang Y, Chen L, Zhai X, Huang Y. Umbilical Cord Blood Transplantation Corrects Very Early-Onset Inflammatory Bowel Disease in Chinese Patients With IL10RA-Associated Immune Deficiency. Inflamm Bowel Dis. 2018 Jun 8;24(7):1416-1427. doi: 10.1093/ibd/izy028.