Neoplasms

Blood and Lymph Conditions

All Conditions

Rare Diseases

Polycythemia Vera

Polycythemia

Hematologic Diseases

Bone Marrow Neoplasms

Hematologic Neoplasms

Bone Marrow Diseases

Myeloproliferative Disorders

Chronic Myeloproliferative Disorders

Patients may self-administer daily ruxolitinib tablets qd or bid orally, without regard to food, in accordance with specified dosing schedule provided by the investigator.

Ruxolitinib

Novartis Pharmaceuticals

The purpose of this Cohort Treatment Plan is to allow access to ruxolitinib for eligible patients diagnosed with Polycythemia Vera. The patient's Treating Physician should follow the suggested treatment guidelines and comply with all local health authority regulations.

The requesting Treating Physician must submit a request for access to drug (often referred to as Compassionate Use) to Novartis which will review and approve by the medical team experienced with the drug and indication.

MAP to Provide Access to Ruxolitinib, for Patients With Polycythemia Vera

Treatment Plan for Managed Access Program (MAP) to Provide Access to Ruxolitinib, for Patients With Polycythemia Vera (PV)

All Drugs and Chemicals

Janus Kinase Inhibitors

JAK inhibitor pre-treated patients (Jakavi® or any other JAK inhibitor for ≥3 months)

Prospective observational study. There is no treatment allocation. Patients administered Jakavi by prescription and administered according to the SmPC.

Jakavi

Jakavi® therapy for polycythemia vera (PV) has so far been studied exclusively in clinical trials and at selected clinical trial centres. This observational study is intended to document the therapy of PV in daily practice with a broad patient population and a geographically representative selection of German centres (both hospitals and practices). The prospective mapping of daily practice reality is thus the main goal of this project.

To achieve meaningful results in accordance with the study objective and to obtain long-term data from daily clinical practice in a real-world setting, the observation period under Jakavi® therapy is specified as 36 months.

A Prospective, Non-interventional Study of JAKAVI® (Ruxolitinib) Treatment in Patients With Polycythemia Vera

A Prospective, Non-interventional Study of JAKAVI® (Ruxolitinib) Treatment in Patients With Polycythemia Vera (PAVE)

Number of patients by initial dosing and number of patients with dose modifications will be provided

Number of patients with treatment interruptions

Overall survival for JAK inhibitor naive and pretreated patients

Number of patients with changes in different blood count values over time (hematocrit, erythrocytes, thrombocytes, leukocytes)

The Myeloproliferative Neoplasm (MPN) Symptom Assessment Form Total Symptom Score (MPN-SAF TSS; MPN-10) questionnaire contains the ten most clinically relevant symptoms reported by patients with MPNs.

It includes disease related symptoms each scored from 0 (absent) to 10 (worst imaginable). Total Scores range from 0-100, with higher scores indicating a greater number of symptoms and severity.

Number of patients with thromboembolic events.

The ECOG performance status is a scale used to assess how a patient's disease is progressing, assess how the disease affects the daily living abilities of the patient, and determine appropriate treatment and prognosis.

The grade ranges from 0 (fully active, able to carry on all pre-disease performance without restriction) to 5 (dead)

Number of patients with change in constitutional symptoms

The Short Form-36 questionnaire consists of questions measuring physical function, physical role limitation, pain, general health, vitality, social function, emotional role limitations, and mental health status. The scores that can be obtained from the scale vary between 0 and 100 and the increase in the scores indicates that the quality of life is high.

Number of performed assessments of JAK2V617F mutation per visist

Heart and Blood Diseases

Primary Myelofibrosis

Thrombocytosis

Thrombocythemia, Essential

Blood Platelet Disorders

Hemostatic Disorders

Blood Coagulation Disorders

Hemorrhagic Disorders

Essential Thrombocythemia

JAK-inhibitor-naive patients, treatment start with ruxolitinib less than 14 days prior to the baseline visit

Patients pretreated with a JAK-inhibitor for more than 14 days prior to the baseline visit

This was a prospective, two-arm, non-interventional study of JAKAVI® (Ruxolitinib) in patients with myelofibrosis

The purpose of this NIS was to gather data from the daily clinical practice of the Jakavi®-treatment in a broad patient population. In order to evaluate the direct effect of Jakavi®, only JAK inhibitor naive patients were documented in the first study arm; patients pretreated with JAK inhibitors were documented in the second study arm to evaluate the long-term efficacy of Jakavi® in this subpopulation.

The documentation of all patients was carried out prospectively and began after the baseline visit. The medical decision on which therapeutic and diagnostic measures to take was made solely by the responsible physician. The observational period per patient was 36 months. The visit schedule after the baseline visit was set by the responsible physician according to standard clinical care, the clinical condition of the respective patients and the SmPC.

A Prospective, Two-arm, Non-interventional Study of JAKAVI® (Ruxolitinib) in Patients With Myelofibrosis

A Prospective, Two-arm, Non Interventional Study of JAKAVI® (Ruxolitinib) in Patients With Myelofibrosis

Evaluation of all occurring adverse events, serious adverse events and serious and non-serious adverse drug reactions

Spleen size (or volume) reduction was measured by palpation

The ECOG performance status is a scale used to assess how a patient's disease is progressing, assess how the disease affects the daily living abilities of the patient, and determine appropriate treatment and prognosis.

The grade ranges from 0 (fully active, able to carry on all pre-disease performance without restriction) to 5 (dead).

Number of patients with change in constitutional symptoms was collected

The MPN-SAF questionnaire contains important questions that cover MF-specific symptoms whose analysis is part of the standard of care. It includes disease related symptoms each scored from 0 (absent) to 10 (worst imaginable). Total Scores range from 0-100, with higher scores indicating a greater number of symptoms and severity.

This questionnaire consists of questions measuring physical function, physical role limitation, pain, general health, vitality, social function, emotional role limitations, and mental health status. The scores that can be obtained from the scale vary between 0 and 100 and the increase in the scores indicates that the quality of life is high.

Ruxolitinib start and end dose was collected

Number of participants with therapy discontinuation and dose adjustments was collected

Number of patients with co-morbidities was collected

Number of patients with blood transfusion dependency was collected

Number of patients with concomitant medications prescribed for myelofibrosis therapy and for the management of side effects was collected

Study title	NCT ID	Conditions	Interventions	Eligibility	Organization
MAP to Provide Access to Ruxolitinib, for Patients With Polycythemia Vera	NCT05269771	Polycythemia Ve...	Ruxolitinib	12 Years - 99 Years	Novartis
A Prospective, Non-interventional Study of JAKAVI® (Ruxolitinib) Treatment in Patients With Polycythemia Vera	NCT05853445	Polycythemia Ve...	Jakavi	18 Years - 120 Years	Novartis
A Prospective, Two-arm, Non-interventional Study of JAKAVI® (Ruxolitinib) in Patients With Myelofibrosis	NCT05044026	Primary Myelofi... Post-polycythem... Post-essential ...	Jakavi	18 Years - 120 Years	Novartis

Spots Global Cancer Trial Database for jakavi

Every month we try and update this database with for jakavi cancer trials from around the world to help patients and their families find trials that might be right for them.
We offer this 100% free of charge and do not endorse any of the trials listed here, we hope it helps you or a loved one.

Spots Global Cancer Trial Database for jakavi

Every month we try and update this database with for jakavi cancer trials from around the world to help patients and their families find trials that might be right for them. We offer this 100% free of charge and do not endorse any of the trials listed here, we hope it helps you or a loved one.

Every month we try and update this database with for jakavi cancer trials from around the world to help patients and their families find trials that might be right for them.
We offer this 100% free of charge and do not endorse any of the trials listed here, we hope it helps you or a loved one.