Clinical trial NCT02910258: Interferon-pegyle α2a Efficiency and Tolerance in Myelofibrosis
Official title: Etude de l'efficacité et de la tolérance de l'interféron-pégylé Dans Les myélofibroses
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Myelofibrosis
Keywords: myelofibrosis
pegylated interferon
Lead sponsor: University Hospital, Brest
Sponsor class: OTHER
First posted: 2016-09-22
Enrollment: 62
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: PROBABILITY_SAMPLE
Study population: Patients diagnosed for PMF or SMF and treated with pegykated interferon α2a

Brief summary: Patients who carried primary or secondary myelofibrosis from Philadelphia negative MPNs (PMF/SMF) and who are treated or are about to be treated with pegylated-interferon (mostly α2a) are eligible to this prospective study. Biological and clinical parameters will be collected from the beginning of the drug use until last news.

A non-opposition consent form need to be signed before entering this study.

Detailed description: In all centres interested, patients who were diagnosed for PMF/SMF can be treated with oral or sub-cutaneous drugs in order to reduce clinical symptoms and biological abnormalities belonging to these diseases.

Pegylated-interferon is one of the better weapons that can be used in these cases. These treatments are well known and used from many years in french hematological centres.

The investigators to collect clinical and biological data from patients treated with pegylated-interferon α2a (the most use) in case of PMF/SMF in order to assess efficacity and tolerance to this drug. The prescription of the product and the dose are under the responsibility of each practician.

Clinical data collected: disease and patients characteristics at the time of diagnosis and at the beginning of the drug use, spleen and liver size, presence of constitutive symptoms, transfusion needs.

Biological data collected: complete hemogram, CD34+ cells count, allele burden of JAK2V617F and calreticulin clones if DNA samples are collected.

These data will be collected avery 3 months during the two first years and every 6 months after.

The investigators also recorded adverse side effects if significative, the concomitant use of hematological drugs (cytoreductive treatments, ASE...), modalities of prescription of the drugs and the reason for stopping the drug and etiology of death if happened.

Eligibility criteria: Inclusion Criteria:

* Primary or secondary Myelofibrosis
* Prescription of pegylated interferon α2a
* Age \> 18 years-old

Exclusion Criteria:

* Other MPNs treated with pegylated interferon α2a
* Patients treated for PMF or SMF but without pegylated interferon

Primary outcomes:
measure: Efficiency of the interferon in Myelofibrosis
timeFrame: Evaluation at 1 year
Secondary outcomes:
measure: Rate of survival of patients with Myelofibrosis under interferon
timeFrame: Evaluation at 5 years
measure: Tolerance of Interferon
timeFrame: Evaluation at 1 year
Other outcomes:
Locations:
city: Angers
country: France
facility: CHU d'Angers
city: Annecy
country: France
facility: Hopital du genevois
city: Brest
country: France
facility: CHRU de Brest
city: LE Mans
country: France
facility: Centre Hospitalier du Mans
city: Lens
country: France
facility: Centre Hospitalier de Lens
city: Marseille
country: France
facility: Institut Paoli Calmette
city: Morlaix
country: France
facility: CH des pays de Morlaix
city: Nice
country: France
facility: Centre Hospitalier de Nice
city: Paris
country: France
facility: AP-HP Hôpital Saint Louis
city: Quimper
country: France
facility: CHIC de Cornouaille
city: Tours
country: France
facility: Centre Hospitalier de Tours
city: Vandoeuvre-les-Nancy
country: France
facility: CHU de Nancy
Study officials:
affiliation: CHRU de Brest
name: Jean-Christophe Ianotto, MD
role: STUDY_DIRECTOR
References:
citation: Ianotto JC, Chauveau A, Boyer-Perrard F, Gyan E, Laribi K, Cony-Makhoul P, Demory JL, de Renzis B, Dosquet C, Rey J, Roy L, Dupriez B, Knoops L, Legros L, Malou M, Hutin P, Ranta D, Benbrahim O, Ugo V, Lippert E, Kiladjian JJ. Benefits and pitfalls of pegylated interferon-alpha2a therapy in patients with myeloproliferative neoplasm-associated myelofibrosis: a French Intergroup of Myeloproliferative neoplasms (FIM) study. Haematologica. 2018 Mar;103(3):438-446. doi: 10.3324/haematol.2017.181297. Epub 2017 Dec 7.