Longitudinal Cohort of Pediatric Primary Immune Thrombocytopenia (ITP)

Condition(s) studied

Investigational drug(s) / intervention(s)

data collection: The study will collect basic information, diagnostic and treatment information from medical records and use questionnaire to measure the exposure of patients, and prospectively follow-up to collect the prognosis information.

Study summary

Immune thrombocytopenic purpura (ITP) is a kind of rare childhood disease that involve autoimmune destruction of platelets.The current Pediatric ITP cohorts are mostly based on single-center or multi-center cases, or cohorts with limited sample size in China. There is a lack of comprehensive and large-scale prospective cohort studies in pediatric ITP. The purpose of this study is to analyze the clinical characteristics of Pediatric ITP, the treatment methods, prognosis and prognostic model of these patients in China.

Eligibility

Primary outcome measure(s)

• Overall response rate — 3 years
  Overall response rate defined as proportion of subjects with a platelet count ≥ 30 × 10\^9/L and at least 2-fold from baseline without bleeding
• Time to onset response — 3 years
  Time to onset response defined as the time needed for subjects to have a platelet count ≥ 30 × 10\^9/L and at least 2-fold from baseline without bleeding
• Duration of response — 3 years
  Duration of response defined as the longest duration for which the subject sustained a platelet count ≥ 30 × 10\^9/L and at least 2-fold from baseline without bleeding
• Sustained response rate — 3 years
  Sustained response rate defined as proportion of subjects who keep a platelet count ≥ 30 × 10\^9/L and at least 2-fold from baseline without bleeding at 6, 12, 24, 36 months after initial administration of certain treatment in absence of rescue therapy
• Emergency treatment — 3 years
  Percentage of subjects who received emergency treatment after initial administration of certain treatment
• Number of subjects with clinically significant bleeding as assessed using the bleeding scale for pediatric patients with ITP after initial administration of certain treatment — 3 years
  Changes of the subjects' numbers in bleeding score after administration of certain treatment according to the reported bleeding scale for pediatric patients with ITP. The bleeding scale for pediatric patients with ITP is a measure of bleeding severity with the following grades: Grade 1 (minor) Minor bleeding, few petechiae (≤100 total) and/or ≤5 small bruises (≤3 cm in diameter), no mucosal bleeding；Grade 2 (mild) Mild bleeding, many petechiae (\>100 total) and/or \>5 large bruises (\>3 cm in diameter), no mucosal bleeding；Grade 3 (moderate) Moderate bleeding, overt mucosal bleeding, troublesome lifestyle；Grade 4 (severe) Severe bleeding, mucosal bleeding leading to decrease in Hb\>2 g/dL or suspected internal hemorrhage；
• Number of subjects with clinically significant bleeding as assessed using the world health organization (WHO) bleeding scale after initial administration of certain treatment — 3 years
  Changes of the subjects' numbers in WHO bleeding score after administration of certain treatment according to the reported World Health Organization's Bleeding Scale. The WHO Bleeding Scale is a measure of bleeding severity with the following grades: grade 0 = no bleeding, grade 1= petechiae, grade 2= mild blood loss, grade 3 = gross blood loss, and grade 4 = debilitating blood loss.
• Recurrence-free survival rate — 3 years
  Time from the start of treatment to the occurrence of a relapse or death event

Trial sites (1)

More Institute of Hematology & Blood Diseases Hospital, China trials in China

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