Ruxolitinib in Primary Myelofibrosis and Secondary to Essential Thrombocythemia or Polycythemia Vera

Recruiting

• Conditions: Myelofibrosis; Primary Myelofibrosis; Secondary Myelofibrosis

• Registration Number: NCT06516406
• Lead Sponsor: IRCCS Azienda Ospedaliero-Universitaria di Bologna

Brief Summary

The study is observational multicenter retrospective and prospective cohort study of patients with primary or secondary myelofibrosis who have initiated therapy with ruxolitinib, prescribed as part of the normal course of care and completely independent of study participation. The primary purpose is to determine the impact of clinical and laboratory characteristics of myelofibrosis on the prognosis of patients treated with ruxolitinib, understood as long-term survival.

Detailed Description

The study is observational multicenter retrospective and prospective cohort study of patients with primary or secondary myelofibrosis who have initiated therapy with ruxolitinib, prescribed as part of the normal course of care and completely independent of study participation. Laboratory tests and histological, cytogenetic, molecular, and radiological investigations performed by the patient and collected for study will be conducted in accordance with clinical practice, independent of the patient's participation in the study. In particular. data on systemic symptoms and splenomegaly will be collected at diagnosis and disease reassessments performed in the context of normal clinical practice. The minimum planned duration of individual patient observation is 3 months and the planned duration of the study is 10 years.

Study Timeline

• Study Start: 2022-05-06
• Study First Submitted: 2023-10-05
• Status Verified: 2024-02
• Study First Submitted QC: 2024-07-17
• Study First Posted: 2024-07-24
• Last Update Submitted: 2024-12-03
• Last Update Posted: 2024-12-05
• Primary Completion: 2032-05-31
• Study Completion: 2032-05-31

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 1055

Inclusion Criteria

• Age ≥ 18 years
• Patients diagnosed with Primary Myelofibrosis or secondary to Essential Thrombocythemia/Polycythemia vera who are being treated or have been treated with ruxolitinib therapy in accordance with normal clinical practice.
• Availability of data on clinical history prior to initiation of Ruxolitinib therapy
• Obtaining informed consent for data collection and processing

Exclusion Criteria

• None

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Long Term Survival	10 years	To determine the impact of clinical and laboratory characteristics of myelofibrosis on the prognosis of patients treated with ruxolitinib, in terms of long-term survival.

Secondary Outcome Measures

Name	Time	Method
Incidence of adverse events	10 years	To assess the incidence of adverse events in patients with myelofibrosis treated with ruxolitinib: thrombosis, haemorrhage, second neoplasms, blastic evolution.
Prognostic value of High Molecular Risk (HMR) mutations.	10 years	To assess the prognostic value of HMRs (via NGS) in MF patients treated with ruxolitinib and to develop a prognostic model that can be applied to patients before and during ruxolitinib therapy.
Significance of peripheral blasts	10 years	To assess correlations between amount of peripheral blasts and response to treatment, leukaemic transformation, drug withdrawal and survival.
Number of participants with treatment-related adverse events as assessed by CTCAE v4.0	10 years	To assess the toxicity of ruxolitinib therapy in the total cohort
To validate the use of the MTSS score	10 years	To validate the use of the MTSS score to assess post-ASCT survival of patients with MF

Trial Locations

Locations (26)

Azienda Ospedaliera Annunziata; 🇮🇹 Cosenza, Calabria, Italy

Grande Ospedale Metropolitano "Bianchi Melacrino Morelli"; 🇮🇹 Reggio Calabria, Calabria, Italy

Università degli Studi di Napoli Federico II U.O.C. di Ematologia e Trapianti di midollo; 🇮🇹 Napoli, Campania, Italy

IRCCS Policlinico Sant'Orsola; 🇮🇹 Bologna, Emilia Romagna, Italy

Università degli studi di Ferrara - Nuovo Polo Ospedaliero di Cona - A.O.U. Arcispedale S. Anna; 🇮🇹 Ferrara, Emilia-Romagna, Italy

Azienda Ospedaliero-Universitaria di Parma; 🇮🇹 Parma, Emilia-Romagna, Italy

AUSL di Piacenza - Palazzine Medicine Specialistiche; 🇮🇹 Piacenza, Emilia-Romagna, Italy

Dipartimento Oncoematologico - AUSL della Romagna; 🇮🇹 Ravenna, Emilia-Romagna, Italy

Ospedale Infermi di Rimini; 🇮🇹 Rimini, Emilia-Romagna, Italy

A.O.U. Integrata di Udine; 🇮🇹 Udine, Friuli-Venezia Giulia, Italy

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