Assessment of Disease Burden in Hairy Cell Leukemia
Trial Snapshot
- Phase
- Not Applicable
- Status
- Recruiting
- Enrollment
- 45
- Locations
- 1
- Primary Endpoint
- Time to next treatment
Study Overview
Brief Summary
Drug-free, single-center, prospective observational pilot study in hairy Cell Leukemia patients
Detailed Description
The V600E gene lesion of B-raf, specific and almost always present in patients with hairy cell leukemia, correlates with the presence of neoplastic cells, therefore of active disease. The measurement of the fractional abundance of the mutated gene, by ddPCR, could therefore constitute a method of molecular assessment of the minimal residual disease. In addition, the values of fractional abundance (FA) of the mutated allele obtained can be integrated coherently in patients' clinical context, along with their PB counts and BM findings.
Primary objective Verify whether the absence of mutation at the end of treatment, indicative of a state of complete molecular response to therapy, can represent a predictor of long treatment-free survival.
Secondary objectives Verify the association between the absence of mutation and the duration of response in patients who do not need treatment for at least 5 years after only one treatment with purine analogues (cladribine and pentostatin) and judged in CR according to current criteria.
Study Design
- Study Type
- Interventional
- Allocation
- Na
- Intervention Model
- Single Group
- Primary Purpose
- Prevention
- Masking
- None
Eligibility Criteria
- Ages
- 18 Years to — (Adult, Older Adult)
- Sex
- All
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- •Histologically confirmed diagnosis of HCL patients:
- •newly diagnosed and candidates for first-line cytoreductive treatment with analogues purines or
- •in relapse after a previous line of treatment, with indication for rescue therapy (repetition of a purine analogue; use of targeted or innovative drugs), except splenectomy or
- •in CR for at least 5 years after a first line of treatment, in the absence of clinical alterations indicative of a state of hematological relapse, or in any case in the absence of an indication for a new line of cytoreductive therapy (time-to-next treatment exceeding 5 years).
- •Age ≥ 18 years at enrollment
- •Signature of written informed consent
Exclusion Criteria
- •Concomitant second malignancy.
Arms & Interventions
HCL, B-raf V600E-mutated patients
For each patient only pheripheral and medullary blood sample and medullary biospy will be collected
Intervention: Peripheral and BM blood sample (Other)
Outcomes
Primary Outcomes
Time to next treatment
Time Frame: through study completion, an average of 4 years
Time to next treatment
Progression Free Survival (PFS)
Time Frame: through study completion, an average of 4 years
Progression Free Survival (PFS)
Correlation between the share of mutated allele (fractional abundance) with the response to the treatment.Correlation between the share of mutated allele (fractional abundance) with the response to the treatment.
Time Frame: through study completion, an average of 4 years
Correlation between the share of mutated allele (fractional abundance) with the response
Secondary Outcomes
- mutational pattern of B-raf i(through study completion, an average of 4 years)