Bone Marrow Transplantation, Cure for Sickle Cell Disease

Bone Marrow Transplantation, Cure for Sickle Cell Disease

Abstract

Research methods are strategies and processes employed during the data collection for analysis to discover new treatment approaches and increase knowledge about the topic being investigated. The selected research topic is “Bone marrow Transplant, a cure for sickle celled anemia.” The study on health issues attracted interest due to the high prevalence observed within the global populations. Also, the investigation will focus on how effective bone marrow transplant is a potential solution for widespread cases of inherited red blood cell disorder-Sickle cell anemia. Taking cognizance study on bone marrow is a clinical investigation; it is worth noting that the research method ideal for the selected topic will require specific considerations to ensure the entire process generate accurate and reliable information.   

Introduction

In the last couple of years, the mortality of people for Sickle Cell disease increased. By definition, sickle cell disease is a hereditary disease in the blood cell in which the cells lack oxygen and have an abnormal shape. The abnormal shape of the red blood cell is a result of the hemoglobins, the red proteins responsible for transporting oxygen in the blood, becoming distorted. As a result, the affected red blood cell is not able to transport oxygen leading to clotting of the blood taking place in the blood veins. The abnormality of the red blood cells causes an adverse condition that affects nearly every tissue in the body. It is one of the human diseases known to cause significant morbidity. The disease is also known to result in a markedly to reduce life expectancy. The treatment of this disease is based mainly on genetic engineering through the use of chemotherapy. However, this method of treatment has not been effective since many complications, and side effects accompany it. It is in this sense that borne marrow transplant has been suggested as one of the best ways for treating this disease two decades ago.

Research Question:

1. How can the Bone Marrow Transplant cure a hereditary disease like Sickle Cell?
2. What are the side effects of the Bone Marrow Transplant and post-transplant in patients?
3. How can Bone Marrow Transplant modify the sickle-cell of the patients for new normal cells?

Hypothesis:

H0:  kids and adults will not be dying if Borne Marrow Transplant is implemented.

H1: Kids and adults will be dying if the Bone Marrow Transplant is not implemented

Thesis Statement:

A Bone Marrow Transplant will save lives since it provides a total cure for sickle cell disease in kids and young adults.

Literature Review

In modern medicine, bone marrow transplant means a 90% survival rate for sickle cell patients. Although the survival rate is high, this treatment has many obstacles to become more universally able to apply. One of the major problems is the lack of donors (Thomas). Considering barriers for this treatment, the problem can be solved, and the only way for a curative approach is a bone marrow transplant (Ballas SK).

Sickle cell anemia is a hereditary disorder that results in the formation of the sickle hemoglobin (Hb) in the red blood cell. The bone marrow transplantations provide the cures but are only possible for about 1% of the affected population that fills this criterion (Gore L). The abnormality shape of the red blood cells caused by the distorted hemoglobin (Hb), causes vascular occlusion.  Vascular occlusion is the first cause of complications that result in death for the patients. It is believed the bone marrow transplant provides a high rate of good life quality (Hebbel RP). The bone marrow transplant is having a good impact on patients and their families, stopping the high risk of mortality for a crisis and organ failure.

While conducting the effectiveness of bone marrow transplant for an adult with sickle cell disease, Krishnamurti, (et al., 2019) found that due to higher graft-versus-host disease (GVHD), the risk of death with the treatment for adults is high. In this sense, Krishnamurti, (et al., 2019) posits that this risk can be reduced by involving non-myeloablative preparation conducting bone marrow transplant. This, according to Devadasan et al., 2018), helps in establishing a stable donor-host hematopoietic chimerism after transplantation. This is essential in providing significant clinical benefits to adult patients.

Methodology

The research mainly focused on the survey research design using the quantitative approach. Owing to this, structured interviews were utilized in the study to obtain first-hand information from the respondents. The qualitative research design was best suited to examine the effectiveness of bone marrow transplant in the treatment of sickle cell disease. The qualitative approach will also aid in understanding the research question by interacting with the research respondents by way of gathering empirical data from the patients. This approach is also due to its benefits of economies of time and resources as compared to other research designs. Lastly, we can conclude and say that the quantitative research design is chosen due to its compatibility with the research problem with the research questions.

Result

One of the first participants qualified for a Bone Marrow Transplant, Brenda Alfaro (37 Years old) with a sickle cell, and the result was a success, case 100% cured. The clinical trials should consider both inclusion and exclusion criteria before enrolling study participants. The research method should ensure that selected study participants meet set criteria that include factors such as health condition, age, and treatment history. A clinical trial should plan for monitoring of the patient after bone marrow transplant.

Discussion

Sickle cell anemia is a disease that is affecting the populations years and years without positive results. Most of the patients die in their early life because of organ damage or several crises.However, two years ago, the cure was found complementing chemotherapy with a bone marrow transplant. As a trial is difficult to believe that the cure was found even with a good result. The reason that makes hardest the process of this procedure is the match for the patient and the high risk of dying during the process.

Nowadays, doctors still discussing the way to perform the bone marrow transplant for young adult patients keeping in mind the highest risk of organ failure during the chemo process even when they know that this kind of processed cured this disease.

Limitations and Implications

“Bone marrow Transplant, a cure for sickle celled anemia,” The study on health issues attracted interest due to the high prevalence observed within the global populations. Also, the investigation will focus on how effective bone marrow transplant is a potential solution for widespread cases of inherited red blood cell disorder “sickle cell anemia.” Taking cognizance study on bone marrow is a clinical investigation; it’s worth noting that the research ideal for the selected topic will require specific considerations to ensure the entire process generate accurate and reliable information.

Furthermore, it critical for clinical research to consider elements that should be met during the actual experiment to make sure approaches applied are safe and effective for human subjects. Research method ideal for studying cure for sickle cell anemia should comply with laws and regulations of ethical committee governing clinical studies. According to the nature of the study, the strategy will need a plan of how the research will be conducted. As such, elements such as research subjects, objectives of the study, review of the literature, research questions, study design, and measures will be employed during the investigation on the effectiveness of bone marrow transplant in treating sickle-celled anemia.

Firstly, the research subject will demonstrate its practical interests concerning bone marrow transplant. The research method will focus on the main objective, suggesting what the study intended to achieve. For instance, the investigation of bone marrow transplant aims to find out the effectiveness of the treatment for sickle cell anemia. The literature review is a vital element of the research method that will point out inconsistencies and gaps, which may add more interest to the study topic (Pautasso, 2015). Additionally, literature from a credible and reliable source will assist researchers in identifying the conceptual models and theoretical models that will be used to conduct empirical research.

Conclusion

In conclusion, we have that the Bone Marrow Transplant is currently the only cure for sickle cell disease, and only a small number of people who have sickle cell disease can have the transplant. There are effective treatments that can reduce symptoms and prolong life. Early diagnosis and regular medical care to prevent complications also contribute to improving well-being. Sickle cell disease is a lifelong illness, and the severity of the disease varies widely.

References

Charache, S., Barton, F. B., Moore, R. D., Terrin, M. L., Steinberg, M. H., Dover, G. J., … &        Orringer, E. P. (1996). Hydroxyurea and sickle cell anemia. Clinical utility of a       myelosuppressive” switching” agent. The Multicenter Study of Hydroxyurea in Sickle             Cell Anemia. Medicine, 75(6), 300-326.

Devadasan, D., Sun, C. W., Westin, E. R., Wu, L. C., Pawlik, K. M., Townes, T. M., &    Goldman, F. D. (2018). Bone Marrow Transplantation after Nonmyeloablative         Treosulfan Conditioning Is Curative in a Murine Model of Sickle Cell Disease. Biology of Blood and Marrow Transplantation, 24(8), 1554-1562.

Gore, L., Lane, P. A., Quinones, R. R., & Giller, R. H. (2000). Successful cord blood        transplantation for sickle cell anemia from a sibling who is human leukocyte antigen-  identical: implications for comprehensive care. Journal of pediatric hematology/oncology, 22(5), 437-440.

Hammarberg, K., Kirkman, M., & de Lacey, S. (2016). Qualitative research methods: when to      use them and how to judge them. Human Reproduction, 31(3), 498-501.

Hebbel, R. P., Boogaerts, M. A., Eaton, J. W., & Steinberg, M. H. (1980). Erythrocyte adherence to endothelium in sickle-cell anemia: a possible determinant of disease severity. New   England Journal of Medicine, 302(18), 992-995.

Krishnamurti, L., Neuberg, D. S., Sullivan, K. M., Kamani, N. R., Abraham, A., Campigotto, F., … & Bakshi, N. (2019). Bone marrow transplantation for adolescents and young adults       with sickle cell disease: Results of a prospective multicenter pilot study. American journal of hematology, 94(4), 446-454.

Nietert, P. J., Silverstein, M. D., & Abboud, M. R. (2002). Sickle cell anemia. Pharmacoeconomics, 20(6), 357-366.

Noyes, J., Booth, A., Moore, G., Flemming, K., Tunçalp, Ö., & Shakibazadeh, E. (2019). Synthesizing quantitative and qualitative evidence to inform guidelines on complex    interventions: clarifying the purposes, designs, and outlining some methods. BMJ global health, 4(Suppl 1), e000893.

Pautasso, Marco. “Ten simple rules for writing a literature review.” (2013): e1003149.