Journal of Applied Hematology

: 2022  |  Volume : 13  |  Issue : 4  |  Page : 263--267

Patient activation measure among transfusion-Dependent patients with hemoglobinopathies

Maha A Badawi1, Sultan M Alkhamesi2, Dana S Alsulaimani3, Aseel Khodary3, Khulud A Almutairi2, Roaa S Alsolaimani2, Salwa A Al-Najjar1,  
1 Department of Hematology; King Fahd Medical Research Center, Hematology Research Unit, King Abdulaziz University, Jeddah, Saudi Arabia
2 Department of Medicine and Faculty of Medicine, King Abdulaziz University, Jeddah, Saudi Arabia
3 Department of Clinical Nutrition, Faculty of Applied Medical Sciences, King Abdulaziz University, Jeddah, Saudi Arabia

Correspondence Address:
Dr. Maha A Badawi
Department of Hematology, Faculty of Medicine, King Abdulaziz University, Jeddah
Saudi Arabia


CONTEXT: Patient's sense of control over their disease management (patient activation) is reported to be associated with better disease outcomes in many chronic conditions. AIMS: The aim of this study is to assess patient activation levels among adult transfusion-dependent patients with hemoglobinopathies. The secondary aims were to determine whether the level of patient activation is associated with different disease outcomes and whether an association exists between higher levels of activation and specific patient characteristics. SETTINGS AND DESIGN: Participants were recruited from the day-care unit in the period between November 2019 and April 2021. Patients who agreed to participate were asked to provide demographic and socioeconomic data and complete the patient activation measure (PAM-10) in Arabic. Hospital records were reviewed for the retrieval of clinical data. RESULTS: The total number of participants was 84. The mean PAM score was 52.90 ± 10. Of all the participants, 21 (25%) had very low levels of activation (Level 1), 38 (45.2%) were categorized as Level 2, 21 (25%) were categorized as Level 3, while 4 (4.8%) were categorized as Level 4 (highest activation level). Disease outcomes were not different among patients by level of patient activation. CONCLUSIONS: Our results show that transfusion-dependent patients with hemoglobinopathies have low levels of activation. No association was found between patient activation and clinical disease outcomes.

How to cite this article:
Badawi MA, Alkhamesi SM, Alsulaimani DS, Khodary A, Almutairi KA, Alsolaimani RS, Al-Najjar SA. Patient activation measure among transfusion-Dependent patients with hemoglobinopathies.J Appl Hematol 2022;13:263-267

How to cite this URL:
Badawi MA, Alkhamesi SM, Alsulaimani DS, Khodary A, Almutairi KA, Alsolaimani RS, Al-Najjar SA. Patient activation measure among transfusion-Dependent patients with hemoglobinopathies. J Appl Hematol [serial online] 2022 [cited 2023 Feb 7 ];13:263-267
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Full Text


Sickle cell disease (SCD) and transfusion-dependent thalassemia (TDT) are highly prevalent in Saudi Arabia, and both are associated with a high risk of mortality and morbidity.[1],[2] By the fifth decade of life, up to half of surviving SCD patients would have developed end-organ dysfunction,[3] while patients with TDT rarely live beyond their forties secondary to disease complications.[4]

Some patients with SCD require chronic red cell transfusions or exchange transfusions. The most common indication for chronic transfusion in SCD is secondary prophylaxis for stroke, based on the recommendation of the guidelines of the American Society of Hematology.[5] In addition, SCD patients may require transfusions for the management of recurrent painful crises, priapism, and acute chest syndrome.[5]

Both SCD patients on chronic transfusion regimens and patients with TDT are at risk of transfusion iron overload. Compliance and adherence with iron-chelation therapy are pivotal for the prevention of iron-related organ damage, a leading cause of mortality and morbidity in these patients.[6],[7]

Moreover, compliance with pharmacological therapy may be influenced by several factors. One of these factors[8] is the level of patient activation. Patient activation is defined as the individual's capacity to take on an active role in self-managing their own health and health care with knowledge and confidence.[9] It is expected that high levels of patient engagement and skill promote patients' abilities to participate in behaviors associated with better disease outcomes.[10] The patient activation measure (PAM) is a validated instrument that can be utilized to measure the degree to which patients are activated to manage their own health care. Higher patient activation is associated with overall better health outcomes.[11],[12],[13]

The goal of the study was to evaluate the mean level of activation among transfusion-dependent patients with SCD and TDT in our institution and to assess the association between higher levels of activation and specific patient characteristics. In addition, we investigated whether high levels of patient activation are associated with lower levels of ferritin as a measure of iron overload.

 Subjects and Methods

Participant selection and study design

This cross-sectional study was conducted at a tertiary academic hospital. Recruitment of patients took place in the day-care unit (day admission unit) where patients receive their regular transfusions between November 2019 and April 2021. Patients were invited to participate in the study if they met the following inclusion criteria: age 17 years or older, have a diagnosis of SCD or thalassemia, and are on chronic blood transfusion in the Daycare Unit at King Abdulaziz University Hospital. Chronic blood transfusion was defined as a simple transfusion or exchange transfusion that occurred every 4–8 weeks. All patients who met these criteria were approached consecutively, and no sampling was attempted.

Patients who agreed to participate signed informed consent and were asked to complete the PAM-10 questionnaire in Arabic in the presence of an investigator. Patients were also asked to answer written questions about demographics and socioeconomic data including social status, number of children, educational level, and employment status. No compensations or rewards were offered to patients. Illiterate patients were assisted by the investigator.

Electronic medical records of patients were reviewed, and the following data were also collected on the date of the interview: mean of the last three measurements of ferritin levels, number of clinic visits over the previous 12 months, number of emergency room (ER) visits over the previous 12 months, and number of admissions to the day-care unit admissions over the previous 12 months.

Variables and measures

PAM-10 questionnaire was licensed from Insignia Health[12] for the purpose of this research study. The PAM is a patient-reported outcome measure containing 10 or 13 items related to the patient's knowledge, skills, and beliefs to manage their own health issues, collaborate with the medical team, and maintain healthy lifestyles.[12] Results of the PAM instrument are reported as an activation score from 0 to 100, and patients are divided according to their score into four categories: Level 1 (passive and overwhelmed); Level 2 (lack of knowledge and self-trust); Level 3 (taking action but lack of confidence and skills); and Level 4 (adopting relevant behaviors).

The PAM instrument has been shown to be a valid and reliable measure with strong psychometric measures.[14] It is available in many languages from its original developer. The Arabic version was used in this study.

Data analysis

Data were analyzed using IBM SPSS version 23 (IBM Corp., Armonk, N.Y., USA) and visually presented by using GraphPad Prism version 8 (GraphPad Software Inc., San Diego, CA, USA). A simple descriptive statistics was used to define the characteristics of the study variables. To establish a relationship between categorical variables, this study used Chi-square test. While comparing two group means and more than two groups, an independent t-test and one-way ANOVA, respectively were used. These tests were done with the assumption of normal distribution. Otherwise, Welch's t-test for two group means was used as an alternative test. To correlate variables that are both represented by means, a Pearson's correlation coefficient was used. Finally, a conventional P < 0.05 was the criteria to reject the null hypothesis.


Eighty-five patients were invited to participate in the study, and 84 agreed to participate. These included 62 patients with TDT and 22 patients with SCD. Both genders were represented equally. The highest level of education for most patients was intermediate or high school (53.6%), with a minority of patients enrolled in postsecondary education (17.9%). Mean serum ferritin was 3475 ng/ml. [Table 1] summarizes the characteristics of participants.{Table 1}

The mean PAM score was 52.90 ± 10. Most patients (70.2%) had low activation levels. The summary of scores is presented in [Table 2]. Details of responses to each statement are presented in [Table S1] Supplementary Material.{Table 2}[INLINE:1]

Statistical analysis revealed no correlation between PAM scores and age, mean ferritin, or the number of health-care visits including inpatient, emergency room, or clinic visits. There was also no statistically significant difference among mean PAM scores of patients by gender, level of education, employment, marital status, or diagnosis (TDT vs. SCD). Data are presented in [Table S2] and [Table S3] Supplementary Material.[INLINE:2][INLINE:3]

In addition, patient characteristics were not significantly different among patients with low and high activation levels [Table 3].{Table 3}


Patient activation plays an important role in predicting overall patient health, health-care utilization, and anticipating possible complications.[9] In addition, increasing patient participation in their health-care plan is more likely to improve overall outcomes.[9] The literature also suggests that high patient activation is associated with improved quality of life (QOL) among patients with various chronic conditions.[15] Several approaches were successfully utilized to improve patient activation including telephone calls, text messaging, and social media.[16],[17],[18],[19] Low levels of activation may be improved by educating patients about the importance of their active participation in their management plan and the effect this may play on their disease course. At high levels of activation, outcomes may be improved further by providing resources for enhancing the knowledge and skills of patients for self-management and boosting their confidence to practice these skills.[16],[17] Studies including patients with other diseases, such as diabetes, chronic kidney disease, and heart disease, have shown that patient activation is associated with age, sex, comorbidities, and disease progression.[18] These observations were not replicated in our study.

We identified two studies on patient activation in patients with SCD. Cronin et al.[20] showed that only a minority of participants with SCD had high activation scores. Moreover, a higher patient activation level was associated with less frequent ER visits and hospitalizations. In a study from Cincinnati,[21] investigators used PAM-13 in 22 young adults with SCD. The cohort had a mean activation score of 69.75 (±13.17) at baseline. Participants were then enrolled in the Stanford Chronic Disease Self-Management Program, a 6-week group therapy plan. The intervention resulted in a significant improvement in self-efficacy, with a medium effect size on PAM.

We could not identify any studies of PAM in patients with thalassemia. However, multiple studies investigated tools for the evaluation of QOL, revealing variable results. QOL was good among adult TDT patients in Sardinia and not different from control participants.[22] Similar good results scores were seen in an Indian group for physical functioning, emotional functioning, and social functioning.[23] However, results were not as good in cohorts of TDT patients from Saudi Arabia,[24] Oman,[25] Jordan,[26] and various other countries.[27],[28]

In this study, we did not find an association between the mean ferritin level and the level of patient activation. This may be a result of the small number of participants. It can also be due to a multitude of factors affecting iron overload, including frequency of transfusions, availability of iron-chelation therapy, and access to optimal monitoring. Options for iron-chelation therapy are limited. Difficulty in adherence to subcutaneous deferoxamine infusion is a well-recognized hurdle. Gastrointestinal and other complications commonly affect patient compliance to oral deferiprone and deferasirox.

Our study has a number of limitations. Captured visits were limited to those in our institution. Visits to other hospitals were not captured. Mean ferritin was used as a surrogate marker of iron overload, but compliance with iron-chelation therapy was not directly evaluated. In addition, not all patients had access to magnetic resonance imaging (MRI T2*) to assess cardiac and hepatic iron loading, so that was not included as an outcome measure. This would be a more specific indicator of tissue iron overload status in comparison to ferritin. Details were not collected about current regimens of iron-chelation therapy, which may affect results.


Transfusion-dependent patients with thalassemia and SCD have low levels of activation. Future studies are required to evaluate various interventions to improve patient activation and its effect on health outcomes.

Financial support and sponsorship


Conflicts of interest

There are no conflicts of interest.


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