Clinical trials on Myelodysplastic Syndromes

Understanding Myelodysplastic Syndromes

Myelodysplastic Syndromes (MDS) represent a group of diverse bone marrow disorders characterized by the bone marrow’s inability to produce adequate healthy blood cells. This condition is primarily seen in individuals over the age of 60, but it can occur at any age. The etiology of MDS remains complex, involving genetic mutations and environmental factors, such as exposure to certain chemicals or radiation. The hallmark of MDS is the presence of dysplasia in one or more types of blood cells (red cells, white cells, and platelets), leading to a high risk of developing into acute myeloid leukemia (AML) over time.

Signs, Symptoms, and Diagnosis

The symptoms of MDS are often subtle and can include fatigue, weakness, shortness of breath, and frequent infections, which result from anemia, neutropenia, and thrombocytopenia respectively. Due to the nonspecific nature of these symptoms, MDS can be challenging to diagnose without detailed blood tests and bone marrow examination. The diagnosis involves a complete blood count (CBC) to check for abnormal levels of blood cells, followed by a bone marrow biopsy to examine the morphology of marrow cells and to assess the percentage of blast cells, which are immature blood cells indicative of MDS.

Treatment and Management

Treatment for MDS varies widely depending on the specific type of syndrome, the severity of symptoms, and the patient’s overall health. Options may include supportive care, such as blood transfusions and medication to stimulate blood cell production, or more aggressive treatments like chemotherapy and bone marrow transplantation. The goal of treatment is to manage symptoms, improve quality of life, and prevent progression to AML. Advances in understanding the genetic basis of MDS have led to the development of targeted therapies, offering hope for more effective and personalized treatment strategies in the future.

Prognosis for Myelodysplastic Syndromes

Myelodysplastic Syndromes (MDS) are a group of diverse bone marrow disorders in which the bone marrow does not produce enough healthy blood cells. The prognosis for individuals with MDS varies significantly based on several factors, including the specific type of MDS, age, and overall health. Some may experience a slow progression of the disease with few symptoms for several years, while others may have a more aggressive form that quickly leads to life-threatening blood shortages. The progression of MDS can also lead to acute myeloid leukemia (AML) in some cases, which can further impact the long-term outlook. Prognostic scoring systems, such as the International Prognostic Scoring System (IPSS), are commonly used to help predict the course of the disease and potential outcomes. Overall, the long-term prospects for individuals with MDS are highly individualized, and regular monitoring by healthcare professionals is essential to manage the condition effectively.

Complications in Myelodysplastic Syndromes

Myelodysplastic Syndromes (MDS) can lead to several complications that impact health and daily life. One common issue is anemia, where there is not enough red blood cells in the body, causing persistent fatigue and weakness. There may also be frequent infections due to a lack of healthy white blood cells, which are crucial for fighting off germs. Bleeding and bruising easily is another complication, resulting from a reduced number of platelets that help with blood clotting. These complications can necessitate regular blood transfusions, which can be time-consuming and may affect one’s lifestyle. Additionally, there is a risk that MDS can evolve into acute myeloid leukemia (AML), a more aggressive type of cancer. These health challenges can significantly affect quality of life, leading to physical limitations and emotional stress.

Treatment Methods for Myelodysplastic Syndromes

For the management of Myelodysplastic Syndromes, the incorporation of lifestyle adjustments is crucial. A balanced diet rich in fruits, vegetables, lean proteins, and whole grains supports overall health. Regular physical activity, tailored to the abilities of the individual, helps maintain physical function and well-being.

  • Pharmacotherapy options, not in clinical trials, may include medications to address specific symptoms or blood count irregularities.
  • Vitamins or supplements are often recommended to correct deficiencies.
  • Modern technology contributes through mobile apps and wearable devices that track health metrics, allowing for personalized management of daily activities and medication reminders.

Consultation with healthcare professionals is important before initiating any new treatment or making lifestyle changes.

  • CT-EU-00084459

    Venetoclax in recurrence of blood cancer after previous stem cell transplantation

    This study aims to investigate the effectiveness and safety of a novel treatment approach, combining the drugs venetoclax and azacitidine, along with donor lymphocyte infusion (DLI) in patients experiencing a relapse of one of two of blood-related disorders: myelodysplastic syndrome (condition where blood cells do not develop properly) or acute myeloid leukemia (bone marrow cells become abnormal and begin to accumulate in excess), following stem cell transplantation. The study involves a group of patients for whom standard treatments have not yielded satisfactory results. The objective is to understand whether the combination of new drugs and donor lymphocyte infusion can effectively control these blood disorders, especially after stem cell transplantation. Researchers are monitoring side effects, assessing disease progression, and determining the safety of the procedure. The results may provide valuable insights into effective therapeutic strategies for these challenging blood-related disorders.
    • azacitidine
    • venetoclax
  • Assessment of the effect of tamibarotene on patients with myelodysplastic syndrome

    This is a study on a new treatment for a type of blood disorder called high-risk myelodysplastic syndrome (HR-MDS). This condition often occurs when certain genes, like RARA, are overactive. First, a blood test will be done to see if participants have RARA. Participants will be given a new medicine named Tamibarotene along with another drug called Azacitidine. Results will be compared to those people who got a placebo pill (a pill without medicine) with Azacitidine. The main goal is to see if Tamibarotene with Azacitidine gets better results compared to the placebo with Azacitidine.

    • Tamibarotene
    • Azacitidine
  • Study on luspatercept treatment for Myelodysplastic Syndrome

    This study is looking into a medication named Luspatercept and how safe and effective it is for people who have a blood condition known as lower-risk Myelodysplastic Syndrome, or LR-MDS for short. These individuals often need transfusions or have their blood refreshed with new, healthy red blood cells. The drug will be given at its highest approved dose to see what effect it has. This is being done in a controlled and careful way to make sure everything is safe for the participants in the study. People who are taking part in this study have been identified as having a very low to medium risk according to the International Prognostic Scoring System (IPSS-R). This means their disease isnt considered high-risk, which makes them suitable candidates for this research. The study is open-label, meaning everybody knows what is in the drug they are getting.

    • Luspatercept
  • Study on effective dose of new potential drug for Leukemia or myelodysplastic syndrome treatment

    In this study, patients with a type of blood cancer, either Acute Myeloid Leukemia or Myelodysplastic Syndrome that has recurred or not responded to previous treatment, will be administered a new drug named CYAD-02. The initial phase of the study aims to determine the safe dosage of CYAD-02 for patients. Before receiving CYAD-02, patients will undergo a milder form of chemotherapy termed non-myeloablative preconditioning.

    The testing process is divided into three stages, each involving different doses of CYAD-02. The first CYAD-02 treatment will be administered after three consecutive days of chemotherapy. For patients showing stability, additional CYAD-02 treatments may be provided without preceding light chemotherapy. All patients who have received at least one CYAD-02 treatment will be monitored for approximately 15 years.

    • CYAD-02
    • Fludara
  • Treatment of anemia in patients with myelodysplastic syndrome with canakinumab

    This study involves a new treatment for patients suffering from anemia linked to lower-risk myelodysplastic syndrome (LR-MDS) or myelodysplastic/myeloproliferative neoplasm (MDS/MPN). The treatment hinges on the use of canakinumab, a medication given through subcutaneous (under the skin) injections every three weeks. The goal is to assess whether canakinumab can effectively treat symptomatic anemia in patients who have either had no response to or are ineligible for erythropoiesis stimulating agents (ESAs). Patients need to be 18 or older and provide informed consent to participate. The study excludes patients with specific health conditions like severe neutropenia, thrombocytopenia, and certain liver ailments. Those with a history of malignancies apart from MDS are excluded unless they have been disease-free for at least five years.

    • Canakinumab
  • Treating anemia in patients due to myelodysplastic syndromes: luspatercept vs epoetin alfa

    This clinical trial compares the effectiveness and safety of two treatments, luspatercept and epoetin alfa, for anemia in adults with myelodysplastic syndromes (MDS). Targeting individuals with low to intermediate-risk MDS who haven’t received blood transfusions, the study involves 360 participants. It is designed to understand whether luspatercept, a biological medication, is more effective than epoetin alfa, another biological treatment. The study focuses on various outcomes, including the progression to blood transfusion dependence, improvements in hemoglobin levels, and overall health-related quality of life.

    • Epoetin Alfa
    • Luspatercept
  • Long-term safety evaluation of luspatercept treatment

    The study focuses on patients with myeloproliferative neoplasms. It tests the drug Luspatercept (ACE-536), evaluating its safety and efficacy over a long term compared to a placebo. This study assessing the prolonged safety of luspatercept in individuals previously enrolled in luspatercept studies. The trial involves multiple phases, including an initial assessment and a follow-up period, where the effects of Luspatercept on the disease’s progression and symptoms are closely monitored. This helps determine if the drug can significantly improve the condition or quality of life for those affected by these neoplasms.

    • Luspatercept
  • Study on the safety and effectiveness of luspatercept in myelodysplastic syndromes patients

    The aim of this study is to test a drug called Luspatercept in people with lower-risk myelodysplastic syndrome (MDS) characterized by ring sideroblasts (MDS-RS), a condition in which the body does not produce enough healthy blood cells. The main aim of the study is to see if Luspatercept can help patients who require regular blood transfusions due to anemia. The study will also look at how long it takes for the medicine to work, how long it lasts, and changes in the need for blood transfusions and levels of hemoglobin (an important protein in red blood cells). The study will also measure changes in certain substances in the blood and the progression of their MDS. Finally, the study will use questionnaires to understand how patients’ lives and physical abilities have changed over time.

    • Luspatercept
  • Testing the effectiveness of oral azacitidine in treating Myelodysplasia Syndromes

    This is a medical research study about a disease of the blood and bone marrow, called low or intermediate-risk myelodysplastic syndrome. It involves a medicine known as oral azacitidine. In this study, some patients will receive the oral azacitidine plus supportive care (like medicines to control symptoms, blood transfusions, etc.), while others will get a placebo (a substance with no medical effect) plus supportive care. Researchers will look at how each patient is responding to better understand if the medicine is helping and if it’s safe.

    • Azacitidine