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Article Summary on Bone Marrow Transplantation

The topic of thalassemia has gained attention in the medical sector, both practical and scholarly. This is centered on the condition being a common monogenic hematologic disorder globally. At least 60,000 children are born with the condition annually, and 80 million individuals carry B-thalassemia (Mohamed, 2017). The interesting aspect about the condition is the advancements in the last century. In the early twentieth century, all patients with the disorder died within six months after diagnosis. The field improvements were reflected between 1949-1957 where 9% of the diagnosed individuals lived up to six years. From the 1980s, medical discoveries, including transfusions, chelation, and transplantation, increased survival. This paper entails a summary of the article by Mohamed (2017) titled “Thalassemia Major: Transplantation or Transfusion and Chelation.”

Mohamed (2017) commences the study by describing the transformation in thalassemia, including how the disorder was a threat to children’s survival and the improvements. A key section in the article is how thalassemia individuals can live and what kills them. This information is the foundation of developing the treatment and other management strategies. Untreated thalassemia patients show chronic anemic symptoms, including poor concentration, headache, dizziness, fatigue, jaundice, and dysfunction of organs. These include heart, gonadal, endocrine, and liver failure. Other significant challenges include personal, family, financial, and career life. In most cases, sub-optimal patients lead unemployed life and succumb to iron overload with cirrhosis, hepatic fibrosis, and heart failure.

The article further evaluates the various treatment options and their weaknesses. The comparison demonstrates the importance and effectiveness of stem cell transplantation compared to the transfusion-chelation. Mohamed (2017) outlines the optimal comprehensive transfusion-chelation weaknesses, which include high costs, inadequate banking facilities, and human resources, and infections. There are also challenges with the poor compliance from patients and deteriorated quality of life for the individual and the family. The high cost affects individuals’ ability to access the treatment option, resulting in further deterioration of the individual’s quality of life and death. Notably, the effective management of the condition requires a combination of various resources, including financial, technical, and human resources. Also, there is a need for increased collaboration among stakeholders, including the patient, government, media, and patient support groups.

The article outlines some of the recent transplantation advancements and the advantages over the transfusion and chelation approach. As Mohamed (2017) described, transplantation remains the only clinically and realistic curative approach to treat thalassemia. Previous transplantations were limited to individuals at low risk and the young with few morbidities. Recent advancements have enhanced the technique and consider the alternate donors and other siblings instead of individuals that matched with varying conditioning intensities. Other transplantation developments have included haploidentical transplantation and graft engineering, significantly contributing to the transplantation success. These developments have enabled all individuals to receive the transplant, including those lacking unrelated donor and matched siblings. These developments’ impacts include a 90% and 80% transfusion-free survival (TFS) for classes 1 and 2 and 3, respectively.

The transplant based on the donor type varies in the outcomes. Among pediatrics, the HSCT from the sibling donors has been successful with remarkable outcomes. There has also been a decrease in graft rejection based on the advancements. For the young adults, the HSCT from unrelated sibling donors was characterized by necro-inflammatory liver abnormalities and chronic HBV or HCV abnormalities for individuals not chelated optimally. Recent advancements, including the introduction of regimens with reduced toxicity, replacing the cyclophosphamide, and changing the busulfan from oral to intravenous, have reduced comorbidities. Another advancement has been the unrelated donor transplantation (URD), where the conditioning regimens and adding the antithymocyte globulin (ATG) has enhanced the treatment and reduced graft rejection or failure. The options are available for children and adults, and young individuals have demonstrated better results.

A key point in the article is that although there is an increase in the transplantation outcome for thalassemia, essential in enhancing the number of transplants for the patients and reducing the mortality rate, the utilization is still inadequate and unsatisfactory. This is the challenge across all countries with a high number of cases. This implies that further developments are required to improve the transplantation process, reduce costs, and ensure more reduction of graft failure. Besides technology, other advancements should be centered on affordability and access by every individual in private and government institutions. The transplantation centers are also in the urban areas, which is a challenge for individuals in the rural areas. Another advancement area is applying the transplantation among the aged individuals, where the outcomes have been inferior. This is attributed to age and accumulated morbidities.

In summary, bone marrow transplantation has significantly been enhanced through technology, and discoveries have demonstrated the transplant’s success. Also, the advancements have seen individuals get the transplant regardless of whether they have a fully matched donor. This has significantly reduced the mortality and morbidity rates associated with thalassemia. Other advantages of transplantation include better quality of life for the individual and the family and a cure rate. However, further research is needed to ensure the transplantation is also successful among the aged. Also, the process should be made accessible and affordable to everyone. This can be achieved through collaboration among the stakeholders, including the government, patient-support groups, patients, and the community.