First-line treatment for patients with polycythemia vera, essential thrombocythemia, and pre-myelofibrosis is based on hydroxyurea or pegylated interferon. The objective of treatment is to prevent thrombotic complications and leukemic transformation. Despite overall good response rates, some patients do not respond to treatment and others lose their response over time. Both situations are associated with worse survival and there are to date no clear predictive factors for response although the existence of additional mutations seems unfavorable. In this exploratory study, we hypothesize that biological factors at diagnosis are associated with hematological response at 12 months. We will more specifically study the association between mutational profile, assessed by next-generation sequencing, and cytokine profile with hematological response. This study will help in identifying patients who will not respond to hydroxyurea or pegylated interferon and give the opportunity to try other treatments upfront, in the perspective of precision medicine. On the basic science side, this study will help in understanding the molecular and immunological factors involved in resistance to treatment.
Study Type
OBSERVATIONAL
Enrollment
120
Next-generation sequencing and cytokine profile will be established in all patients before the start of treatment.
Chu Angers
Angers, France
RECRUITINGChu Brest
Brest, France
RECRUITINGChu Nantes
Nantes, France
RECRUITINGChu Poitiers
Poitiers, France
RECRUITINGChu Rennes
Rennes, France
RECRUITINGChu Tours
Tours, France
RECRUITINGComplete hematological response
ELN-2013 criteria by meeting all of the following: * Durable resolution of disease-related signs including palpable hepatosplenomegaly, large symptoms improvement, AND * Durable peripheral blood count remission, defined as: platelet count ≤400 ×109/L, WBC count \<10 × 109/L, Ht lower than 45% without phlebotomies (for PV patients), absence of leukoerythroblastosis, AND * Without signs of progressive disease, and absence of any hemorrhagic or thrombotic events.
Time frame: 12 months
Complete hematological response
ELN-2013 criteria by meeting all of the following: * Durable resolution of disease-related signs including palpable hepatosplenomegaly, large symptoms improvement, AND * Durable peripheral blood count remission, defined as: platelet count ≤400 ×109/L, WBC count \<10 × 109/L, Ht lower than 45% without phlebotomies (for PV patients), absence of leukoerythroblastosis, AND * Without signs of progressive disease, and absence of any hemorrhagic or thrombotic events.
Time frame: 24, 36, 48, and 60 months
Molecular response
ELN-2013 criteria: Complete response is defined as eradication of a preexisting abnormality (CALR, JAK2, or MPL mutations) by quantitative PCR. Partial response applies only to patients with at least 20% mutant allele burden at baseline. Partial response is defined as ≥50% decrease in allele burden by quantitative PCR.
Time frame: 12 and 24 months
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