Myelofibrosis, Primary Myelofibrosis, Secondary Myelofibrosis
Conditions
Keywords
Safety, Efficacy
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.
Interventions
None listed
Sponsors
IRCCS Azienda Ospedaliero-Universitaria di Bologna
Study design
Observational model
COHORT
Time perspective
CROSS_SECTIONAL
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| 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
| Measure | Time frame | Description |
|---|---|---|
| Significance of peripheral blasts | 10 years | To assess correlations between amount of peripheral blasts and response to treatment, leukaemic transformation, drug withdrawal and survival. |
| 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. |
| 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 |
| 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. |
| 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 |
Countries
Italy
Contacts
Primary ContactFrancesca Palandri, MD, PhD
Outcome results
None listed