The mainstay of care for patients with thalassemia is regular blood transfusions, which improve the anemia and suppress the complications of thalassemia such as serious growth, skeletal, and neurological complications. However, such methods increase iron concentrations in the body leading to detrimental consequences.
Novartis launched an injectable iron chelator in 1968. Although it was the first solution provided to patients with iron overload and had helped many suffering from iron overload, its inconvenient mode of administration may have been a cause of suboptimal chelation of iron overload in patients associated with a shorter life expectancy and increased morbidity. With oral medications, emerging better compliance and adherence rates were seen reflecting lower iron concentration in patients, although there were some inconveniences caused by the dispersible tablet formulation where patients were experiencing palatability issues, inconvenient preparation methods and side effects.
With the newest launch of the film-coated tablet, Novartis is providing patients not only with the efficacy to avoid iron overload complications, but also the convenience of administration and less bothersome adverse events. This will certainly help in increasing patients’ compliance to their medication translating into a higher efficacy.
Although the switch to oral therapy significantly increased adherence, other factors should be considered such as the healthcare system where individualizing therapy - routinely assessing adherence- and establishment of a multidisciplinary team are essential to assure a smooth patient journey. Moreover, addressing the psychosocial problems such as attitudes and beliefs about thalassemia, lack of perception of severity of iron overload, and expectations from treatment are other important areas to approach thalassemia and iron overload in a holistic manner.
Important initiatives taken by the Ministry of Health - Oman such as promoting pre-marital screening to check for carrier state is crucial in raising awareness on thalassemia and its consequences as well as preparing parents of children with thalassemia on the management of the disorder.
In Oman, patients with blood disorders have many options to receive medical care from highly qualified doctors and medical experts.
Today, a diagnosis of thalassemia does not mean the end of patients’ normal lives. With proper management and optimal chelation patients are able to avoid the consequences of their blood disorders. Focusing on improving patients’ quality of life and taking a holistic approach to the management of thalassemia will result in a positive outcome for patients with thalassemia.
Novartis is reimagining medicine to improve and extend people’s lives. As a leading global medicines company, we use innovative science and digital technologies to create transformative treatments in areas of great medical need. In our quest to find new medicines, we consistently rank among the world’s top companies investing in research and development. Novartis products reach nearly 1 billion people globally and we are finding innovative ways to expand access to our latest treatments. About 125 000 people of more than 140 nationalities work at Novartis around the world.
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