Thalassemia: Pharmacotherapy for Hematologic Disorders Essay

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Introduction

Many genetic disorders develop in childhood and affect patients’ entire lives. Thalassemia is a hematologic condition that is defined by low levels of hemoglobin and erythrocytes in one’s blood (Arcangelo, Peterson, Wilbur, & Reinhold, 2017).

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This disorder is often confused with iron deficiency anemia due to the similarity of symptoms (Arcangelo et al., 2017). There exist many forms of thalassemia, where mild conditions such as alpha-thalassemia and beta-thalassemia minor do not require treatment. However, such severe forms as beta-thalassemia major need blood transfusions, as well as supporting supplements and drugs to regulate the level of iron in the blood. (Arcangelo et al., 2017) The disorder affects children, whose response to transfusions may manifest in a variety of complications, including heart problems (Gode, Prabhu, & Venkatesh, 2017). Furthermore, some children have difficulties with adhering to regular procedures, increasing the risk of adverse outcomes (Kannan & Singh, 2017).

To treat children with thalassemia, healthcare providers should use blood transfusions and iron-controlling drugs as well as follow an engaging program to increase the rate of therapy compliance.

Hematologic Disorder: Treatment

The main types of thalassemia include alpha-thalassemia and beta-thalassemia, which are further divided into minor and major forms. Alpha-thalassemia is characterized by mild symptoms of fatigue and weakness, and beta-thalassemia minor also presents with symptoms that do not alter affected persons’ life significantly (Arcangelo et al., 2017). On the other hand, beta-thalassemia major and rare cases of other severe subtypes of the disorder are defined by patients with moderate or severe symptoms that substantially affect their daily acidities. These individuals require medical treatment in the form of blood transfusions (Arcangelo et al., 2017).

They should also receive supplements to control the level of folate and undergo iron chelation therapy to reduce the adverse effects of excessive iron transfusion. The latter procedure is performed with the help of deferoxamine mesylate (Arcangelo et al., 2017). Iqbal et al. (2018) also suggest utilizing hydroxyurea to treat children with beta-thalassemia. Nevertheless, the combination of blood transfusion and iron chelation remains the basis of the therapy plan for people with severe thalassemia.

Patient Factor

Age is one of the factors that affect the results of therapy. Although the described procedures have an impact on all patients, children may suffer from serious complications that can escalate because many problems are not visible. According to Gode et al. (2017), cardiovascular issues such as tachycardia are silent in children with this hematologic disorder. This issue can be overcome with timely diagnostics and a controlled treatment plan.

Another problem is the lack of adherence to the procedure of blood transfusion by children. Kannan and Singh (2017) state that the monotonous regimen of the therapy leads to children losing interest in treatment. The lack of regularity in transfusions and iron chelation may result in growth retardation, tissue damage, and other severe outcomes (Kannan & Singh, 2017). In order to resolve this issue, healthcare providers should monitor children’s compliance levels and implement a scoring initiative to keep young patients motivated to maintain their schedule.

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Conclusion

Thalassemia is a genetic disorder that presents itself in various types and ranging from mild to severe forms. Some of them do not require therapy, but severe manifestations can be treated with blood transfusions and iron chelation to control the level of iron in one’s blood and avoid complications. Children respond to monotonous therapy with low adherence which may lead to serious health-related problems. A low rate of compliance can be overcome with a scoring system that motivates children to participate.

References

Arcangelo, V. P., Peterson, A. M., Wilbur, V., & Reinhold, J. A. (Eds.). (2017). Pharmacotherapeutics for advanced practice: A practical approach (4th ed.). Ambler, PA: Lippincott Williams & Wilkins.

Gode, V. K., Prabhu, S. S., & Venkatesh, S. (2017). Cardiac abnormalities in transfusion dependent beta thalassemia children. Global Journal for Research Analysis, 6(9), 146-149.

Iqbal, A., Ansari, S. H., Parveen, S., Khan, I. A., Siddiqui, A. J., & Musharraf, S. G. (2018). Hydroxyurea treated β-thalassemia children demonstrate a shift in metabolism towards healthy pattern. Scientific Reports, 8(15152), 1-9.

Kannan, S., & Singh, A. (2017). Compliance score as a monitoring tool to promote treatment adherence in children with thalassemia major for improved physical growth. Asian Journal of Transfusion Science, 11(2), 108-114.

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IvyPanda. (2021, July 20). Thalassemia: Pharmacotherapy for Hematologic Disorders. https://ivypanda.com/essays/thalassemia-pharmacotherapy-for-hematologic-disorders/

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"Thalassemia: Pharmacotherapy for Hematologic Disorders." IvyPanda, 20 July 2021, ivypanda.com/essays/thalassemia-pharmacotherapy-for-hematologic-disorders/.

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IvyPanda. (2021) 'Thalassemia: Pharmacotherapy for Hematologic Disorders'. 20 July.

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IvyPanda. 2021. "Thalassemia: Pharmacotherapy for Hematologic Disorders." July 20, 2021. https://ivypanda.com/essays/thalassemia-pharmacotherapy-for-hematologic-disorders/.

1. IvyPanda. "Thalassemia: Pharmacotherapy for Hematologic Disorders." July 20, 2021. https://ivypanda.com/essays/thalassemia-pharmacotherapy-for-hematologic-disorders/.


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IvyPanda. "Thalassemia: Pharmacotherapy for Hematologic Disorders." July 20, 2021. https://ivypanda.com/essays/thalassemia-pharmacotherapy-for-hematologic-disorders/.

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