Adult-onset leukoencephalopathy with axonal spheroids and pigmented glia hereditary white matter disorder
Conditions
Interventions
None listed
Sponsors
Vrije Universiteit Medisch Centrum
Eligibility
Age
18 Years to 99 Years
Inclusion criteria
Inclusion criteria: - Diagnosis of ALSP confirmed by a pathogenic CSF1R mutation - Aged 18 years or older - Capable of giving informed consent
Exclusion criteria
Exclusion criteria: - No informed consent given by the patient - Cognitive capabilities are too low at inclusion of the study to give informed consent
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| The primary outcomes of objective 1 are cytokine profiles in blood before HCT, expressed in Normalized Protein eXpression (NPX) in Log2 scale, and cytokine profiles in blood over time. | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary endpoints will be the association of cytokine profiles with clinical outcomes after treatment. | — |
Countries
Netherlands
Outcome results
None listed