Assessment of Disease Burden in Hairy Cell Leukemia (BRAF)

Scientific Institute for Research Hospitalization and Healthcare (IRCCS)

Status: Enrolling

Conditions: Hairy Cell Leukemia

Treatments: Other: Peripheral and BM blood sample

Study type: Interventional

Funder types: Other

Identifiers

NCT06781515

BRAF

Details and patient eligibility

About

Drug-free, single-center, prospective observational pilot study in hairy Cell Leukemia patients

Full 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.

Enrollment: 45 estimated patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

1. Histologically confirmed diagnosis of HCL patients:

   1. newly diagnosed and candidates for first-line cytoreductive treatment with analogues purines or
   2. 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
   3. 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).

2. Age ≥ 18 years at enrollment

3. Signature of written informed consent

Exclusion criteria

1. Concomitant second malignancy.

Trial design

Primary purpose: Prevention

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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45 participants in 1 patient group

HCL, B-raf V600E-mutated patients

Other group

Description:

For each patient only pheripheral and medullary blood sample and medullary biospy will be collected

Treatment:

Other: Peripheral and BM blood sample