Cartilage-hair Hypoplasia
Conditions
Brief summary
The investigatoris will carefully select cartilage-hair hypoplasia patients unexposed to varicella (VZV) and immunize patients in a controlled manner with VZV vaccine. Patients will be selected on the basis of disease severity and the degree of immunodeficiency (including CD4+ T-cell counts). Any potential complication of VZV immunization, such as rash, will be discussed with the patients/caregivers beforehand and acyclovir will be used to treat any VZV related symptoms, consistent with the current practices. The investigators will verify the development of immune response to vaccination by testing for VZV antibodies and cell-mediated immunity.
Interventions
BIOLOGICALVarilrix
Vaccination against varicella in selected cartilage-hair hypoplasia patients
Sponsors
Helsinki University Central Hospital
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
PREVENTION
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
12 Months to No maximum
Healthy volunteers
No
Inclusion criteria
* genetically confirmed cartilage-hair hypoplasia diagnosis * age \>12 months * no history of chickenpox * informed concent of the patient/caregiver.
Exclusion criteria
* positive serum IgG for varicella zoster virus * low CD4+ cell counts (\<15% or \<200 cells/mm3) * clinical or laboratory signs of severe immunodeficiency * ongoing intravenous or subcutaneous immunoglobulin treatment
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Humoral response to vaccination | 4-6 weeks post-vaccination |
| Cell-mediated response to vaccination | 4-6 wks post-vaccination |
| Number and severity of adverse events to vaccination | 0-60 days |
Outcome results
None listed