Clinical trial NCT05351216: The Effect of Sirolimus on Immunizations During the Treatment of Kaposiform Hemangioendothelioma
Official title: The Effect of Sirolimus on Time-sequentially Scheduled Immunizations During the Treatment of Kaposiform Hemangioendothelioma: a Case-control Study
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Kaposiform Hemangioendothelioma
Keywords: Following National Vaccination Program
Sirolimus
Lead sponsor: Children's Hospital of Fudan University
Sponsor class: OTHER
First posted: 2022-04-28
Enrollment: 174
Sex: ALL
Maximum age (years): 12
Standard ages: CHILD
Accepts healthy volunteers: yes
Observational model: CASE_CONTROL
Time perspective: PROSPECTIVE
Sampling method: PROBABILITY_SAMPLE
Study population: The calculation of the sample size came from the previous pre-experiment. Investigator take HBsAb protection rate as the main outcome indicator. The sample size of this study, calculated using a non-inferiority test, was 156 (78 per group). In addition, 10% was required to make up for loss to follow-up, withdrawal, etc. So a total of 174 subjects (87 per group) are anticipated.

Brief summary: To research and explore the antibody protection and immune memory after vaccination in children with KHE during sirolimus administration. To explore the feasibility (safety and efficacy) of vaccination in a timely manner during the administration of sirolimus in children with KHE. To search for back-up plans for vaccination regimens for KHE patients taking sirolimus in children who do not respond to primary vaccination.

Detailed description: Children with KHE have an early onset. KHE usually occurs in infants and young children less than 1 year old, of which neonates account for about 38.5%-60% of all cases. Due to the immunosuppressive effect of sirolimus, the vaccination was usually suspended after taking it, and children would be in a state of no immune protection. These children are at greatly increased risk of exposure to microorganisms and consequent infection. Therefore, it is necessary to vaccinate them against infectious diseases. However, vaccination with live vaccines has the potential to cause severe infections through reversion of the vaccine strain to a pathogenic form. Moreover, studies have also shown that protective antibodies are severely affected in transplant patients taking immunosuppressive drugs and in patients with solid tumors after chemotherapy. Loss of immune memory is very common, and marked deficits in B cell function and humoral immunity can persist even for years.

Eligibility criteria: Inclusion Criteria:

* Case groups:
* KHE patients treated with sirolimus.
* After immunoglobulin and flow cytometry assays, as well as outpatient evaluation and assessment, those participants will be vaccinated with live attenuated vaccines or inactivated vaccines in a timely order according to the advice.
* Control groups:
* Healthy children with no immune deficiencies.
* Participants are vaccinated according to the National Immunization Program in a timely manner.
* Participants are matched to the case group according to age.

Exclusion Criteria:

* HBsAg, HBeAg positive, or other active infectious diseases;
* History of immunodeficiency or low immunoglobulin levels;
* Autoimmune disease or fever during blood collection;
* Use of other medication or surgery;
* Suffering from other bleeding disorders;
* Suffering from other solid tumors or hematological tumors, etc.;
* Withdraw informed consent.

Primary outcomes:
measure: Titers of Hepatitis B virus surface antibody
timeFrame: Admission within 1 day
measure: Titers of Hepatitis B virus surface antibody
timeFrame: The 7th month after admission
measure: Titers of Hepatitis B virus surface antibody
timeFrame: The 12th month after admission
measure: Titers of Hepatitis B virus surface antibody
timeFrame: The 18th month after admission
Secondary outcomes:
measure: Levels of measles antibodies.
timeFrame: Admission within 1 day
measure: Levels of mumps antibodies
timeFrame: Admission within 1 day
measure: Levels of rubella antibodies.
timeFrame: Admission within 1 day
measure: Levels of measles antibodies.
timeFrame: The 7th month after admission
measure: Levels of mumps antibodies.
timeFrame: The 7th month after admission
measure: Levels of rubella antibodies.
timeFrame: The 7th month after admission
measure: Levels of measles antibodies.
timeFrame: The 12th month after admission
measure: Levels of mumps antibodies.
timeFrame: The 12th month after admission
measure: Levels of rubella antibodies.
timeFrame: The 12th month after admission
measure: Levels of measles antibodies.
timeFrame: The 18th month after admission
measure: Levels of mumps antibodies.
timeFrame: The 18th month after admission
measure: Levels of rubella antibodies.
timeFrame: The 18th month after admission
measure: Level of Japanese encephalitis antibody.
timeFrame: Admission within 1 day
measure: Level of Japanese encephalitis antibody.
timeFrame: The 7th month after admission
measure: Level of Japanese encephalitis antibody.
timeFrame: The 12th month after admission
measure: Level of Japanese encephalitis antibody.
timeFrame: The 18th month after admission
measure: Level of varicella antibody
timeFrame: Admission within 1 day
measure: Level of varicella antibody.
timeFrame: The 7th month after admission
measure: Level of varicella antibody.
timeFrame: The 12th month after admission
measure: Level of varicella antibody.
timeFrame: The 18th month after admission
measure: Level of COVID-19 antibody.
timeFrame: Admission within 1 day
measure: Level of COVID-19 antibody.
timeFrame: The 7th month after admission
measure: Level of COVID-19 antibody.
timeFrame: The 12th month after admission
measure: Level of COVID-19 antibody.
timeFrame: The 18th month after admission
Other outcomes:
Locations:
city: Shanghai
country: China
facility: Children's Hospital of Fudan University
state: Shanghai
Study officials:
affiliation: Children's Hospital of Fudan University
name: Kai Li, PhD
role: PRINCIPAL_INVESTIGATOR
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