Thalassemia Treatment Advancements: All That Patients And Caregivers Need To Know

Like many other conditions, home healthcare has become a precious option for managing thalassemia patients requiring frequent medical attention.

India’s diverse demography poses significant health challenges, including susceptibility to genetic disorders such as thalassemia. India has the most important number of children with Thalassemia major about 1 to 1.5 lakhs and almost 42 million carriers of (beta) thalassemia trait. In a rare genetic blood disorder, thalassemia, the body makes an abnormal form of haemoglobin. The condition also leads to excessive destruction of red blood cells causing anaemia. On World Thalassemia Day, there is a need to raise awareness about the disease and understand the advancements in treating the condition. It is also essential to discuss the role of home care and caregivers in treating and managing this condition. Dr Vishal Sehgal, President of Portea Medical, ensures early detection of thalassemia and helps make informed decisions.

Types Of Thalassemia And Symptoms

Depending on which part of the haemoglobin molecule is underproduced, there are two types of thalassemia. The most common condition is beta thalassemia, in which low haemoglobin levels lead to a lack of oxygen. The second type is alpha thalassemia, in which one or more genes controlling the production of alpha haemoglobin are either defective or completely absent. One parent with traits of thalassemia minor can increase the chances of the child being born with thalassemia minor by about 50%. This becomes 25% when both parents are carriers of thalassemia minor. It is imperative to test newborns at birth because the symptoms do not appear until the child is six months old. It is essential to watch out for symptoms like fatigue, pale skin, cold hands and feet, shortness of breath, and headaches. People with alpha thalassemia can experience chest pain, drowsiness, and dizziness. In beta thalassemia, there is delayed growth (in kids), skeletal deformities, excessive iron, increased infections, and delayed puberty.

Conventional Treatment Processes

There are two kinds of traditional treatment plans for thalassemia.

• Transfusion and chelation therapy Regular transfusion and chelation therapy have helped improve patients’ survival and quality of life with thalassemia. They have also ensured that a previously fatal disease with early death is now a chronic disease with which people can survive longer.
• Allogeneic hematopoietic stem cell transplantation is a natural and clinically rational curative modality for treating thalassemia major. Recent developments have advanced the technique from siblings and alternate donors other than fully matched siblings with different conditioning and graft engineering intensities for global use.

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Recent Advancements In Treatment

There have been many recent developments in the treatment of thalassemia. This includes a gene therapy used by researchers in treating severely affected beta-thalassemia patients. In gene therapy, the fully functional good beta chain genes are added to the patient’s body after clearing the abnormal hematopoietic stem cells. The latter carry defective genes. In the follow-up of these trial patients over many years now, it has been found that their average haemoglobin production is stable. This could indicate that gene therapy can become a viable cure for beta-thalassemia. Another alternative approach is being explored to fix the defective gene using CRISPR. This is a technique used to edit the gene. Despite the immense promise they hold for treating thalassemia, there are challenges like high cost and lack of necessary infrastructure, which limit their availability at the global level.

Role Of Home Healthcare And Caregivers

Like many other conditions, home healthcare has become a precious option for managing thalassemia patients requiring frequent medical attention. By providing care at home, healthcare professionals can improve patient’s quality of life and ensure their treatment plan is tailored to their needs.