What is Thalassemia?Thalassemia is a genetic blood disorder where the bone marrow fails to produce the required red blood cells for the body to thrive. People with thalassemia major require lifelong blood transfusions and other therapies to survive. People with thalassemia minor are carriers and lead normal lives without any clinical interventions. Thalassemia is the world's largest rare disease community with a prevalence of 288,000 with 60,000 live births each year. Thalassemia is most prevalent in the Mediterranean region (Italy, Greece) which is why it is also known as Mediterranean anemia.
Currently, there is no established curative except for bone marrow transplant, which carries risks of infection and rejection. Several therapies are in clinical trial phase and this is hope for the community.
World Thalassemia Day 2016World Thalassemia Day is celebrated each year on 8 May. This day is marked to show global solidarity and support for thalassemia patients and their families. Thalassemia presents a significant clinical and financial burden on patients and families especially in developing countries like India with poor public health systems and infrastructure. With little or no access to quality healthcare, the financial burden of treatment is entirely borne by individuals and families. This factor contributes to increased morbidity and mortality rates among patients.
‘Equal access to quality healthcare treatments and free or reimbursable drugs are vital for improving quality of life of people with thalassemia.’
AdvertisementThalassemia Day is a platform to raise awareness on this genetic disorder, mobilize resources and support and exchange information and updates on latest clinical research and therapeutics. Advocacy and activist organizations working for the betterment of the thalassemia community engage with a range of programs from patient activities, free health check-ups and tests, seminars, workshops and marathons. All these events put thalassemia into the public sphere and create a connect between the public and the patient community. Public engagement is crucial for the success of genetic counseling and prevention programs.
Thalassemia Day is also a day to share patient stories about their challenges, hurdles and successes in life. People with thalassemia in India and globally have successfully crossed all hurdles and entered various professions like medicine, engineering, academics and even public administration. Thalassemia Day celebrates past successes and encourages the community to move beyond the limitations of genes.
In a country like India with an estimated 35 lakh plus thalassemia majors, it is important to use every platform to inform, educate and make people aware about this disorder and its prevention. Patients' families receive genetic counseling for prenatal testing so that other children in the family do not carry this disorder. This is a day to dispel the myths surrounding Thalassemia and ensure inclusion for people with thalassemia in education, health, employment and other societal structures.
World Thalassemia Day is also a day to make the public aware of the importance of blood donation. On this occasion, several thalassemia societies across the globe arrange voluntary blood camps. One such mega camp this year is being organized by the Thalassemia Society of Mauritius. Blood is central to the survival of a person with thalassemia and this significance is highlighted during such blood camps.
In several parts of the globe, walkathons are organized to gather support and mobilize resources for Thalassemia. In Bangalore, the Rotary-TTK blood bank which provides free blood transfusions to people with Thalassemia and other blood disorders like hemophilia is participating in the TCS World 10K run to raise awareness of thalassemia and gather financial support for patients.
Theme: "Access to Safe and Effective Drugs for Thalassemia"This year, the theme for World Thalassemia Day has been developed by the Thalassemia International Federation (TIF), Cyprus, which is the largest patient advocacy organization globally. The theme is significant for India: "Access to Safe and Effective Drugs for Thalassemia." This theme also feeds into the objectives of the first two phases of clinical trials namely: safety and efficacy.
The theme is apt for India especially this year as it brings to light the need for policy changes to ensure health equity and justice. In India, several children and adults with thalassemia succumb due to lack of access to drugs and treatment. The lack of a national/state rare disease policy, which can enable free delivery of treatment and drugs, is totally missing in India.
At this point, India clearly needs immediate policy interventions to make available free drugs and treatment. In keeping with Universal Health Care for All, India needs to make this policy change to ensure that people with Thalassemia or any other rare disorder do not die for want of access to treatment. Access to free, safe and effective treatment and drugs are the key to survival and quality of life.
Hope on the HorizonClinical trials in the United States and Europe provide new hope for people with Thalassemia. The viral-vector mediated globin gene transfer LentiGlobin BB305 Drug by BlueBird Bio is currently in Phase 2 and being evaluated for efficacy.
Another drug product Luspatercept (ACE-536) by Acceleron and Celgene has moved into Phase 3. Luspatercept aims at reducing the blood transfusion requirements of patients with transfusion-dependent Thalassemia.
At the 57th American Society of Hematology (ASH) 2015 Annual Meeting in Orlando, BlueBird Bio, Acceleron and Celgene presented updates on the latest results from the clinical trials.
The research community, clinicians and the thalassemia community is hopeful that a therapeutic cure will soon be out to improve the lives of those living with thalassemia.
- About Thalassemia
- International Thalassemia Day 2016
- Study Evaluating the Safety and Efficacy of the LentiGlobin BB305 Drug Product in Beta Thalassemia Major Subjects
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