Myeloproliferative neoplasms (MPNs) are rare cancers of the blood that result from genetic changes in the cells that make blood in the bone marrow. These changes cause too many red blood cells, white blood cells, or platelets to be produced, which can lead to various problems and symptoms.
MPNs were first identified in 1951, but the genetic causes were only evident once the JAK2 V617F mutation was found in 2005. This mutation is present in most people with polycythemia vera and about half with essential thrombocythemia and primary myelofibrosis. The JAK2 V617F mutation has helped researchers and doctors better understand and treat MPNs.
If you have an MPN, you may wonder about your disease and treatment options. You may also come across words and ideas that need to be explained. This space will present some basic facts about MPNs and the current research and clinical trials.
Would you like to participate in a clinical trial on myeloproliferative neoplasms (MPN)?
If so, you may wonder what a clinical trial entails, its benefits and risks, and what questions to ask before agreeing. In this article, we explain some fundamental aspects of clinical trials for MPN, a type of blood cancer affecting the production of red and white blood cells and platelets.
Clinical trials are scientific investigations that test the safety and efficacy of new treatments or interventions for a specific disease or condition. Clinical trials are necessary to advance medical knowledge and offer better therapeutic options to patients. Clinical trials of MPN seek to find new drugs or drug combinations that will improve disease control, quality of life, and survival in patients with essential thrombocythemia (ET), myelofibrosis (MF), or polycythemia vera (PV). There are also clinical trials investigating the causes and molecular mechanisms of MPNs, such as the discovery of JAK2, calreticulin, and other mutations.
Clinical trials are divided into different phases, depending on the objective and the number of participants:
Phase I: a new treatment is tested in a small group of healthy volunteers or patients to evaluate its safety, dose, and possible side effects.
Phase II: A new treatment is tested in a larger group of patients to assess its efficacy and safety compared to a standard treatment or placebo.
Phase III: A new treatment is tested in a large and diverse group of patients to confirm its efficacy and safety compared to a standard or placebo treatment. This phase is usually the last one before applying for regulatory approval of the treatment.
Phase IV: it is carried out after the treatment has been approved and marketed to monitor its long-term effects, safety in different populations, and usefulness in clinical practice.
If you are interested in participating in an MPN clinical trial, you should be well-informed about the trial's goals, procedures, benefits, and risks. You should also know your rights and responsibilities as a participant. Some questions you can ask the research team are:
What is the purpose of the clinical trial?
What treatment will be tried, and how is it administered?
What tests or visits are required during the clinical trial?
What benefits can I expect from the clinical trial?
What risks or side effects can I have from participating in the clinical trial?
What alternatives do I have if I do not participate in the clinical trial?
Who finances the clinical trial and supervises its ethical and scientific quality?
What rights do I have as a participant, and how can I withdraw from the clinical trial if I wish?
What follow-up will be done after the end of the clinical trial?
To participate in a clinical trial, you must give your informed consent in writing, which means that you have understood all the information about the clinical trial and voluntarily agree to participate. Informed consent is not a contract; you can withdraw it at any time without negative consequences for your health care.
Not all patients with MPN can participate in any clinical trial. Each clinical trial has eligibility criteria that define who may or may not participate, depending on factors such as the type and stage of the disease, previous treatment, age, and condition.