Pediatric Primary Immunodeficiency Disease (PID) in China

Clinical Characteristics and Prognosis of Primary Immunodeficiency Disease (PID) in Chinese Children

This study is a prospective cohort study of children diagnosed with primary immunodeficiency disease (PID). The aim is to investigate the clinical characteristics and prognosis of PID in Chinese children.

Study Overview

• Status: Unknown
• Conditions: Primary Immunodeficiency Disease

Detailed Description

Primary immunodeficiency disease (PID) is a group of rare and fatal disease. However, the research about clinical characteristics, treatment, management and prognosis of Chinese children with PID is still not perfect and there is no basic and large database. Therefore, this study is conducted, in order to set up a complete database and long term follow up of Chinese children with PID, and establish foundations for basic research and precise medicine.

This study is divided into two parts. In part one, pediatric patients with PID in the past 10 years will be collected retrospectively by review medical records from these centers, and investigate the clinical features and prognosis. In part two, all new cases with suspected PID will be collected. Clinical symptoms, physical examination, blood tests, and humoral and cellular immune functions will be detected. Gene tests would be done when necessary to confirm the diagnosis. Then follow up at 6-moth, 1-year, 2-year, 3-year, 4-year and 5-year respectively. The trial will be completed in 60 months.

• Study Type: Observational
• Enrollment (Anticipated): 400

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 1 day to 18 years (ADULT, CHILD)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

• Sampling Method: Probability Sample

Study Population

Children who was confirmed diagnosis as PID at the certain hospitals

Description

Inclusion Criteria:

1. Age: from neonates to 18 years old

2. Fulfilling the one of the followings:

   1. Severe infection after vaccination (BCG, polio arthritis pill)
   2. Chronic/recurrent ear infection, sinus infections and respiratory tract infection
   3. Recurrent, deep skin or deep-seated infections
   4. Boys with early period and refractory thrombocytopenia
   5. Infants with obviously decreased absolute number of lymphocytes
   6. Severe allergy with infection
   7. Infant diabetes with severe diarrhea
   8. Boys with severe Epstein-Barr virus infection
   9. Infants with hepatosplenomegaly and generalized lymphadenopathy
   10. Infantile hemophagocytic syndrome
   11. Failure of an infant to gain weight or grow normally
   12. Persistent thrush in mouth or fungalinfection on skin
   13. Family history of primary immunodeficiency
   14. Invasive pneumococcal infection
   15. Erythrodermic psoriasis
3. Gene test confirmed PID pediatric patients

Exclusion Criteria:

Subject will be excluded if she or he would not enroll into this study

Study Plan

How is the study designed?

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Frequencies of severe infection of children with PID	Times of severe infection attack will be recorded every year from baseline to 60 months, assessed by questionnaire	60 months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Life quality of children with PID	Life Quality Questionnaire, once for every year	60 months
Complications of children with PID		60 months
Long-term changes on lung function of children with PID		60 months

Collaborators and Investigators

• Sponsor: Beijing Children's Hospital
• Collaborators: Shenzhen Children's Hospital; Capital Institute of Pediatrics, China
• Investigators: Study Director: kunling shen, MD,PhD, Beijing Children's Hospital, Capital Medical University, China; Principal Investigator: jianxin he, MD,PhD, Beijing Children's Hospital, Capital Medical University, China

Study record dates

Study Major Dates

• Study Start (ANTICIPATED): January 1, 2018
• Primary Completion (ANTICIPATED): August 1, 2022
• Study Completion (ANTICIPATED): August 1, 2022

Study Registration Dates

• First Submitted: August 15, 2017
• First Submitted That Met QC Criteria: August 15, 2017
• First Posted (ACTUAL): August 17, 2017

Study Record Updates

• Last Update Posted (ACTUAL): January 3, 2018
• Last Update Submitted That Met QC Criteria: January 1, 2018
• Last Verified: May 1, 2017

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Immune System Diseases; Genetic Diseases, Inborn; Immunologic Deficiency Syndromes; Primary Immunodeficiency Diseases
• Other Study ID Numbers: BCHlung006

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No