Assessment of Disease Burden in Hairy Cell Leukemia
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: Peripheral and BM blood sample.
- Who it may be relevant to
- Registry conditions: Hairy Cell Leukemia. Basic parameters: from 18 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- Italy
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Overview
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.
Interventions
- Other Peripheral and BM blood sample
Peripheral and BM blood samples will be analyzed with the ddPCR method
Primary outcome measures
- Progression Free Survival (PFS) [Time frame: through study completion, an average of 4 years]
- Time to next treatment [Time frame: through study completion, an average of 4 years]
- 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]
Secondary outcome measures (1)
- mutational pattern of B-raf i [Time frame: through study completion, an average of 4 years]
Eligibility criteria
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
1\. Concomitant second malignancy.
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- N/A
- Model
- Single group
- Masking
- Open label
- Primary purpose
- Prevention
Study locations
Italy · 1 center
- IRCCS Azienda Ospedaliero - Universitaria di Bologna — Bologna
Identifiers
NCT: NCT06781515 · BRAF