Hydroxyurea-Based Therapy in Patients With Sickle Cell Disease

Introduction

The purpose of this project is to examine medication adherence related to hydroxyurea-based (HU) therapy in patients with sickle cell disease (SCD). The focus of this project is on determining the outcomes of using pill compliance packaging on HU therapy for SCD patients in the context when non-compliance packaging approaches proved to be ineffective. As a result, it is important to examine how the application of pill packaging systems can contribute to improving medication adherence, decreasing patients’ stay at hospitals, and improving their health outcomes. The methodology described in this chapter is proposed to address the question of how HU therapy combined with applying pill packaging systems can affect medication adherence and associated pain levels and hospitalization rates, as well as hospital stays, in patients with SCD. In this chapter, the research problem, the clinical question, the appropriate methodology, and design, as well as the population and sample, are described in detail. Furthermore, the chapter presents the applied instrument with a focus on its validity and reliability and the description of data collection and analysis procedures, ethical considerations, and limitations.

Statement of the Problem

The problem is that there is a lack of evidence to state whether HU therapy combined with the use of a pill packaging system and patient education can have a positive impact on medication adherence and health outcomes in patients with SCD. According to researchers, the application of HU can potentially prevent the development of complications associated with SCD, but the research on the role of this therapy in reducing hospital stays is still limited (Badawy et al., 2017; Wang, 2016). Taking hydroxyurea is discussed as one of the most effective therapies in patients with SCD, but there is still a high occurrence of non-adherence to this medication and the lack of research on the problem.

Additionally, the specific problem studied in this project is that, although HU is actively used to treat SCD, healthcare providers had a difficult time finding alternatives to increase medication adherence in patients. The use of pill packaging systems in SCD patients requires more detailed investigation because only limited aspects of pill intake management were researched previously (Telen, 2016). A lack of data on applying pill packaging systems in combination with HU therapy illustrates the necessity of more research in the field to examine all potential outcomes of using HU and pill packaging systems for SCD patients in terms of their medication adherence, a hospital stay, and the quality of life.

Clinical Question

The clinical question appropriate for this project is formulated to address the problem and purpose that were determined for this project. In this chapter, the described methodology will work to provide answers to the following question: How does the intervention of pill packaging systems related to HU therapy and combined with patient education allow for increased medication adherence in patients with SCD in comparison to using HU therapy with no pill packaging over one month? In this question, independent variables are HU therapy based on using or not pill packaging systems and patient education that will be measured with reference to the presence or absence of the intervention. Dependent variables are changes in medication adherence and clinical outcomes in patients with SCD, the data on which will be collected with the help of medical records and measured referring to the provided figures. The focus on the relationship between these variables accentuates the necessity of selecting the quantitative methodology for this project.

Project Methodology

A quantitative methodology is viewed as the most appropriate option for conducting this project in comparison to a qualitative methodology and mixed methods. The reason is that a quantitative methodology provides a set of effective tools for finding the relationships between variables with reference to collecting numerical data and applying statistical tests (Trochim, Donnelly, & Arora, 2015). A qualitative methodology helps a clinician understand specific details regarding the phenomenon, concentrate on subjects’ visions, and formulate the theory referring to collected data (Trochim et al., 2015). This approach will not lead to addressing the set clinical question in this project. On the contrary, a quantitative method can be viewed as appropriate to apply the selected theory to explain the studied relationship and test hypotheses.

For the purpose of this project, a quantitative methodology covers the questions asked with reference to the purpose of this study, and the application of mixed methods can seem unrequired. Applying the tools proposed in the context of quantitative methods, it will be possible to collect data regarding the use of compliance pill packaging in patients with SCD and outcomes in the numerical form and analyze them to further conclusions about the effectiveness of the combined intervention to include patient education for individuals with SCD. Referring to the existing literature on the problem where a quantitative method was applied, it is possible to predict positive outcomes of using the intervention in persons with SCD (Badawy et al., 2017; Lê et al., 2015). From this perspective, a quantitative methodology is selected as appropriate to examine the efficacy of pill packaging systems for the improvement of medication adherence and the promotion of well-being among patients with SCD.

Project Design

The research design selected for this project is a retrospective chart review (RCR) that is based on the application of a quantitative methodology. The reason for choosing this research design in the context of this project is that it is possible to answer the set clinical question only with reference to a large amount of data on many patients with SCD and the outcomes of HU combined with using pill packaging and patient education for them. The data can be collected only retrospectively, and the application of medical records containing the information on the progress of patients with SCD, as well as the application of data on medication adherence and the length of stay, is required in this project.

The use of an RCR allows for examining the existing data on patients with SCD, and all information regarding the variables will be obtained from medical records. Researchers actively use RCRs for their quantitative studies because this research design allows for accessing a lot of information on specific conditions with the opportunity to make the sample size large and representative (Wickson-Griffiths, Kaasalainen, Ploeg, & McAiney, 2014). Therefore, the data collection tools associated with RCRs are medical protocols and records provided by hospital authorities.

Population and Sample Selection

For the purpose of this study, the setting is extended to include a medical center and a pharmacy located in Charlotte, North Carolina, the authorities of which have agreed to provide the information on patients with SCD who received treatment based on HU supported by using or not pill packaging systems. The population will be attained with the help of Premier Pharmacy, and Wellness Center data based on the fact patients receive medications according to Hydroxyurea prescriptions there. The total population for this project covers all individuals with SCD treated by HU in Charlotte, and the project population covers those patients having SCD who received medications in Premier Pharmacy and Wellness Center.

The project sample (out of more than 1,000 people with SCD) is 200 participants. This project sample size is determined after completing the power analysis where the power value was set as 0.8 and the type I error rate was set as 5%. To receive access to records on patients with SCD and taken medications, the authorities of the selected pharmacies will be contacted, and their approval and supervision are required to gather all required data for analysis in this project. The clinician will not participate in selecting medical records for analysis, and such an approach allows for the randomization of data.

Instrumentation or Sources of Data

A retrospective chart review as an approach to collecting and analyzing data selected for this project requires gathering records on SCD patients’ prescription fill history and pill pack counts from Premier Pharmacy in Charlotte, as well as medical records from Wellness Center. These sources of data include information on patient demographics, the related sickle cell genotype, HU prescriptions, the use of pill packaging systems (if any), refills, the number of hospitalizations for one patient and the overall rate for a center, comorbidities developed in a patient, and the type of therapy received (the focus is on HU as an inclusion criterion). The RCR in Wellness Center provides information regarding medication adherence for SCD patients, their hospital stay, and the presence or absence of patient education. The RCR allows for collecting data on 21-a 65-year-old male and female patients diagnosed with SCD, and this information is related to the period of 2014-2018. Similar records for RCRs were also used by Wickson-Griffiths et al. (2014) and Van Melle et al. (2018) in their studies.

Validity

RCRs are actively applied in nursing and medical studies, but data provided in medical records are collected not for the purpose of a certain study, which leads to the validity issue. As a result, these data are not structured appropriately by researchers to address their needs. From this perspective, the accuracy of study results, as well as their validity, depends on a researcher’s approach to retrieving the most efficient data to be used to measure variables in research (Van Melle et al., 2018). Still, for records and RCRs, Cronbach’s alpha reported in other studies is usually high: 0.79-0.89. To increase the validity of using medical records in the context of an RCR, researchers are expected to pay much attention to formulating relevant inclusion and exclusion criteria, according to which the data will be retrieved from available medical protocols and charts.

Reliability

The high-level reliability is typical for medical records used in the context of RCRs as sources of data because the provided information cannot change if several researchers use the same data set and similar inclusion and exclusion criteria. It is important to note that reliability related to RCRs is calculated with reference to Cohen’s kappa. For RCRs, Cohen’s kappa is usually higher than 0.6 based on the application of medical records as sources of data (Van Melle et al., 2018; Wickson-Griffiths et al., 2014). This Cohen’s kappa demonstrates the coefficient of agreement between coders regarding the quality of data in used sets of medical records or other types of charts, and it is appropriate to guarantee the conducted project presents reliable results.

Data Collection Procedures

During the first stage, the authorities of Premier Pharmacy and Wellness Center are to be contacted to receive access to their databases, including information on patients with SCD receiving HU-based treatment and pill packaging. After obtaining the approval of authorities, the issue of receiving consent from patients should be addressed (Trochim et al., 2015). At the second stage, after accessing medical records and protocols, the researcher will select 200 records, including all the data on patients’ SCD, their prescription fill history, as well as pill pack counts got before and after one month of taking the medication. Those SCD patients of Wellness Center who have not prescribed any type of HU are regarded as not meeting inclusion criteria for this project, and the related data will not be collected.

At this stage, the information on the applied HU therapy and the presence or absence of patient education will be collected, along with the information on changes in patients’ adherence to the medication. In addition, the data on the number of hospitalizations and average lengths of hospital stays will be monitored. Additionally, demographics data related to patients will also be retrieved, and they will include individuals’ age, gender, race, as well as the presence of comorbidities and the information on deaths. All the data gathered for this project will be stored using the web cloud application and protected by passwords. It is important to note that the retrieved files will be destroyed after completing this quantitative project.

Data Analysis Procedures

While using medical records, both categorical and numerical data were retrieved for further analysis using statistical tests. For the purpose of this research, both descriptive and inferential statistics data should be collected. Descriptive data are important to determine the averages for the number of pills taken per day, hospitalizations, hospital stays, and positive outcomes in pain management associated with HU therapy and changes in these figures in different years and for different groups of patients.

To focus on statistically significant differences in collected data and address the research question, it is necessary to conduct a series of t-tests to accentuate the changes in medication adherence and hospital stays depending on the presence of HU therapy with or without pill packaging systems supported by patient education. The focus is on finding a statistically significant difference in the number of days of hospital stays depending on the application of HU, referring to p <0.05. The focus should also be on finding a statistically significant difference in the level of medication adherence reported depending on the application of HU (p <0.05). Thus, a t-test is selected for this project as a tool applied in a quantitative methodology (Trochim et al., 2015). The reason is that its results will allow for concluding about a potential relationship between the application of HU along with patient education and possible reduction in levels of pain, as well as decreasing hospitalization rates and a hospital stays in patients with SCD. SPSS software will be used for conducting a series of t-tests to evaluate the assumptions formulated for this research.

Ethical Considerations

The potential ethical issues associated with this project are related to retrieving medical records on patients with SCD that include protected health information according to the Health Insurance Portability and Accountability Act (HIPAA) and the protection of received data. To guarantee that all the ethical issues are addressed, it is necessary to ask for and receive the permission of the authorities regarding access to clinical data for the purpose of this project (Trochim et al., 2015). One more important issue to address is the possibility to use collected data without the consent of patients of the selected hospitals as potential participants in this project. Any personal data that are presented in medical records and protocols are labeled to guarantee the protection of patients’ confidentiality and anonymity. According to the Belmont Report, beneficence, respect, and justice will be guaranteed by protecting patients’ anonymity when disclosing project results. The approval for conducting such type of research should be received from the Institutional Review Board.

Limitations

One of the major limitations is the necessity to limit the project scope to the settings located in Charlotte, North Carolina, which makes the project more specific. The other key limitations in this study are associated with the use of the quantitative methodology and an RCR research design in particular. Depending on the specifics of RCRs, one of the limitations is the lack of access to available and accurate records presenting all the required information. It is important to note that if the documentation is incomplete, it is almost impossible to receive statistically significant results when conducting certain analyses to test hypotheses and assumptions. In the project, this limitation will be addressed with a focus on selecting only those patients, medical records for whom include all the required data.

In addition, the accuracy and validity of the study results can also be limited by inappropriate inclusion and exclusion criteria. This limitation is addressed in the study with a focus on formulating effective criteria depending on the research question, the purpose, and the identified variables. Another limitation associated with the application of a quantitative method is the impossibility of receiving detailed examinations of trends in the sphere of treating patients with SCD with reference to figures gathered for this quantitative study (Trochim et al., 2015). Therefore, further research on the topic is recommended to be based on a qualitative methodology to examine patient outcomes after receiving HU therapy in detail to support the findings of this study.

Summary

This chapter has presented detailed and comprehensive information on the methodology that was selected for examining how the application of HU therapy combined with patient education and pill packaging in individuals with SCD can predict changes in medication adherence, hospitalization rates, and lengths of hospital stays. It is important to note that, for this project, a quantitative method has been selected as more suitable to address the purpose of research than a qualitative method. A retrospective chart review has been proposed as a specific research design that allows for conducting analysis with reference to retrospective data on a certain condition to examine outcomes for patients.

Data collection and analysis procedures typical for RCRs have also been described in this chapter with much detail to present the steps to be made by the researcher to realize the purpose of this study. In addition, the chapter also includes information on the validity and reliability of using medical records as the sources of data in the context of RCRs. Finally, the ethical issues and limitations associated with this research have also been described in this chapter to inform the reader about all the details related to conducting this quantitative study. The results associated with applying an RCR to address the clinical question set for this project will be presented in Chapter 4.

References

Badawy, S. M., Thompson, A. A., Lai, J. S., Penedo, F. J., Rychlik, K., & Liem, R. I. (2017). Adherence to hydroxyurea, health-related quality of life domains, and patients’ perceptions of sickle cell disease and hydroxyurea: A cross-sectional study in adolescents and young adults. Health and Quality of Life Outcomes, 15(1), 136-145. Web.

Lê, P. Q., Gulbis, B., Dedeken, L., Dupont, S., Vanderfaeillie, A., Heijmans, C.,… Rozen, L. (2015). Survival among children and adults with sickle cell disease in Belgium: Benefit from hydroxyurea treatment. Pediatric Blood & Cancer, 62(11), 1956-1961. Web.

Telen, M. J. (2016). Beyond hydroxyurea: New and old drugs in the pipeline for sickle cell disease. Blood, 127(7), 810-819. Web.

Trochim, W. M., Donnelly, J. P., & Arora, K. (2015). Research methods: The essential knowledge base (2nd ed.). New York, NY: Cengage Learning.

Van Melle, M. A., Zwart, D. L., Poldervaart, J. M., Verkerk, O. J., Langelaan, M., Van Stel, H. F., & De Wit, N. J. (2018). Validity and reliability of a medical record review method identifying transitional patient safety incidents in merged primary and secondary care patients’ records. BMJ Open, 8(8), e018576. Web.

Wang, W. C. (2016). Minireview: Prognostic factors and the response to hydroxurea treatment in sickle cell disease. Experimental Biology and Medicine, 241(7), 730-736. Web.

Wickson-Griffiths, A., Kaasalainen, S., Ploeg, J., & McAiney, C. (2014). Revisiting retrospective chart review: An evaluation of nursing home palliative and end-of-life care research. Palliative Medicine & Care, 1(2): 8-12. Web.

I, Shawanda L Spencer, verify that I have completed 15 clock hours in association with the goals and objectives for this assignment. I have also tracked said practice hours in the Typhon Student Tracking System for verification purposes and will be sure that all approvals are in place from my faculty and practice mentor.