Introduction
Acute lymphoblastic leukemia (ALL) is a rare disease, which affects thousands of people annually nonetheless. Although both children and adults can get ALL, it primarily develops in small children, teenagers, and young adults. Despite medical advances, acute lymphoblastic leukemia often leads to the worst possible outcome, leaving patients with a poor prognosis. The purpose of this paper is to present the first part of the case study by describing the disease’s pathophysiology, treatment options, prevalence, and incidence. In addition, the essay will partially touch on the subjects of the FDA regulatory process, health care economics, as well as family involvement in decision-making.
Description of the Disease, Its Prevalence, and Incidence
Acute lymphoblastic leukemia is a rare type of cancer, which damages white blood cells in a person’s bone marrow. The disease develops from not yet mature lymphocytes and progresses quickly, which is why it has the term “acute” in the title. According to Terwilliger and Abdul-Hay (2017), the pathogenesis of this condition “involves the abnormal proliferation and differentiation of a global population of lymphoid cells” (para. 3). ALL demonstrates the breakages in lymphoid development caused by genetic mutations. Thus, certain groups are predisposed to the diseases, including those with Down and Bloom syndromes, ataxia telangiectasia, and other conditions (Terwilliger & Abdul-Hay, 2017).
Additionally, there is a range of other predisposing factors such as pesticide and radiation exposure, as well as contact with certain viruses. As for symptoms, the most common are weight loss, high body temperature, excessive sweating, fatigue, and frequent bruising. Some cases lead to the spread of affected white cells into the central nervous system. This can result in more serious symptoms, including seizures, dizziness, and headaches.
Apart from ALL’s pathophysiology and symptoms, it is crucial to discuss the risk factors. Although there is no definite answer as to what the primary reason for mutations is, known risk factors are smoking, obesity, a weak immune system, as well as previous rounds of chemotherapy. As mentioned earlier, a number of ALL cases in children are related to specific genetic disorders. Although 80% of ALL occurs in the younger population comprised of predominantly children, the disease affects adults as well. Terwilliger and Abdul-Hay (2017) state that the incidence of acute lymphoblastic leukemia is 1.6 per 100,000 population in the United States alone.
The researchers note that in the year 2016, doctors diagnosed 6590 cases of the disorder and detected 1400 deaths (Terwilliger & Abdul-Hay, 2017). As the most common childhood type of cancer, ALL is one of the leading causes of morbidity and mortality in kids (Iacobucci & Mullighan, 2017). The incidence of the disease follows a system of bimodal distribution with two peaks: one in childhood and another one in a person’s 50s. Although the innovative treatment strategies significantly help pediatric patients suffering from ALL, the prognosis for the elderly is often extremely negative as this population has simply no energy to endure the painful and time-consuming cycle of treatments.
It is important to recognize the urgency a patient with ALL experiences in getting proper treatment. After all, acute lymphoblastic leukemia is an aggressive disease, which developes quickly and requires treatment immediately after diagnosis. The treatment plan usually consists of three primary stages, including remission induction, consolidation, and maintenance. The first stage is aimed at minimizing possible symptoms by destroying leukemia cells in the bone marrow.
The second part furthers the progress, while the last one implies the administration of chemotherapy medicine to prevent a relapse. Terwilliger and Abdul-Hay (2017) raise concern regarding the efficiency of modern-day ALL treatment as “despite a high rate of response to induction chemotherapy, only 30-40% of adult patients with ALL will achieve long-term remission” (para. 2). Therefore, even the most appropriate treatment regimen may not lead to any positive health outcomes for ALL patients, particularly adults.
Discussion of Possible Laboratory Testing
A crucial factor, which determines whether a physician manages to assess a patient’s prognosis accurately, is laboratory testing. In the case of acute lymphoblastic leukemia, a doctor might run a number of tests, including blood, bone marrow, and imaging tests. Blood tests may include a complete blood count to determine exactly what number of each blood cell type a patient has, as well as a peripheral blood smear to detect changes in the appearance and migration of blood cells. Bone marrow tests can help the medical team to move forward with the diagnosis by checking for signs of leukemia in a bone marrow sample.
Lastly, imaging tests such as an X-ray, CT scans, and ultrasounds are essential to demonstrate how far cancer has spread. In addition, a physician can express the need for a lumbar puncture if they have to be sure ALL has not reached a patient’s brain or spinal cord. Only after all these tests, a doctor can start to create the most appropriate treatment regimen and consider the prospects of offering a patient an experimental treatment.
Guidelines and Reasons behind the FDA Regulations for Introducing New Pharmaceutical Agents (Policy)
The United States’ pharmaceutical industry operates under the constraints of the country’s highly intricate regulatory framework for drug approval. The primary challenge for the U.S. Food and Drug Administration (FDA) in charge of the aforementioned process is to maintain a balance between rigorous, standardized testing of new pharmaceutical agents and the need of the health care sector for timely drugs, which can save countless lives.
The drug development plan includes basic steps, which include initial research, pre-clinical development, clinical development in three phases, FDA review and approval, as well as, finally, post-approval practices, including promotion and marketing. The primary reasons behind the potential approval may include the fact that a chosen drug is safe and efficient according to its proposed use, the medication’s benefits far outweigh possible risks, the pharmaceutical’s labeling is appropriate and fits the pre-determined FDA standards, and so on. Furthermore, FDA reviewers have to make sure that the methods utilized by the drug’s manufacturers are efficient and strategic enough to ensure its long-term consistency in access and quality (Darrow et al., 2020).
After the first stage of target condition and existing treatments examination, FDA can move on to evaluating the drug’s benefits and risks based on at least two Phase III clinical trials provided by the manufacturer (Van Norman, 2016). Thus, it is evident that the reviewers from the organization do not test the drug themselves but rely on the manufacturer to provide true results of clinical trials. Examining the label proposed by the manufacturer is a part of the FDA’s overall strategy for managing risks, which all drugs inevitably have.
The Role of Money and Grants in Scientific Advances: The Economics of Health Care (Capitalism)
Scientific research is a business akin to farming or banking, which means that the industry has to produce innovations and generate an array of new discoveries in order to make profits. Scholars and scientists are rightfully concerned with the threat research funding can pose. Indeed, sponsors who pay for laboratory equipment, scientists’ salaries, and intellectual property can have a significant impact on the researcher’s adherence to ethical norms, including honesty, impartiality, and social responsibility (Okagaki & Dean, 2016).
They can manipulate study designs or frameworks for the interpretation of results in order to generate seemingly objective outcomes, which unfairly favor their interests in reality. In addition, these individuals and organizations can shake the very foundation of scientific integrity by banning scientists on their payroll from publishing certain findings.
The Role of Family Involvement in the Health Care Decision
Adult patients often do not have the slightest idea as to what decision they should make in regards to their diagnosis or treatment, which leads to decisions conflict. As acute lymphoblastic leukemia is a disease predominantly affecting children, it is that much more important for physicians to involve caregivers/family to make medical decisions. The study by Boland et al. (2017) demonstrates that it is exceptionally important to involve parents in the decision-making process to reduce the risk of decisional conflict and to provide the much-needed support system for the patient. These findings extend beyond pediatrics and can apply to patients of different age groups. However, it is also crucial to acknowledge how emotionally draining it must be for clinicians to create and keep communication channels with families efficient, as well as to share grim prognoses with them.
Conclusion
In conclusion, it is evident that acute lymphoblastic leukemia is a serious condition, which affects thousands of people in the United States. The primary target is children who are forced to go through various stages of treatment in order to get better and prevent cancer from coming back. Patients suffering from ALL undoubtedly need the support of their families as their involvement in the decision-making process can assist in making an informed decision.
References
Boland, L., Kryworuchko, J., Saarimaki, A. & Lawson , M. L. (2017). Parental decision making involvement and decisional conflict: a descriptive study. BMC Pediatrics, 17(1). Web.
Darrow, J. J., Avorn, J., & Kesselheim, A. S. (2020). FDA approval and regulation of pharmaceuticals, 1983-2018. JAMA, 323(2). Web.
Iacobucci, I., & Mullighan, C. G. (2017). Genetic basis of acute lymphoblastic leukemia. Journal of Clinical Oncology, 35(9), 975–983. Web.
Okagaki, L. H., & Dean, R. A. (2016). The influence of funding sources on the scientific method. Molecular Plant Pathology, 17(5), 651–653. Web.
Terwilliger, T, & Abdul-Hay, M. (2017). Acute lymphoblastic leukemia: a comprehensive review and 2017 update. Blood Cancer Journal, 7(6), e577. Web.
Van Norman, G. A. (2016). Drugs, devices, and the FDA: Part 1. JACC: Basic to Translational Science, 1(3), 170–179. Web.