|Year : 2018 | Volume
| Issue : 1 | Page : 5-10
Health-related quality of life in adult patients with hemophilia, Riyadh, Saudi Arabia
Maha Abdullah Allhaidan1, Arwa Mohammad Almashaan1, Atheer Ali Alduaij1, Hessa Abdulaziz Altuwaijri1, Lama Abdullah Alotaibi1, Abdulkareem M Almomen2
1 Intern, College of Medicine, King Saud University, Riyadh, Saudi Arabia
2 Department of Medicine, Division of Hematology and Oncology, King Khalid University Hospital, King Saud University, Riyadh, Saudi Arabia
|Date of Web Publication||22-Mar-2018|
Dr. Maha Abdullah Allhaidan
Saad Ibn Amir Alansari Street, P. O. Box 270166, Riyadh 11352
Source of Support: None, Conflict of Interest: None
Background: Despite a reduction in mortality rates, hemophilia patients continue to suffer the consequences of repeated bleeding episodes. Assessment of health-related quality of life (HRQoL) attempts to determine how different life aspects are affected and helps guide future decision-making.
AIMS: The aim of the study was to assess HRQoL in adult patients with hemophilia managed at King Khalid University Hospital (KKUH), Riyadh and to identify factors affecting HRQoL.
Settings and Design: An observational cross-sectional study was conducted at KKUH in Riyadh from September 2014 to April 2015.
Subjects and Methods: Sixty-one patients followed up and treated at KKUH from 2000 to 2015 were enrolled in the study. Patients were asked to complete the Arabic version of the Hemophilia-Specific QoL (HAEMO-QoL-A) Questionnaire.
Results: Forty-Nine hemophilic patients completed the HAEMO-QoL-A questionnaire. Of these, 81.6% had hemophilia A, with a mean age of 29.84 years. The most common target joint was the knee (89.7%). The majority used on-demand treatment (55.1%). The HAEMO-QoL-A mean total score was 99.89. “Treatment concerns” were the most impaired dimension, whereas the “work/school, family, and social life” dimension was least affected. Patients receiving on-demand treatment had the lowest average bleeding events (8.22, P = 0.007).
Conclusion: Frequent bleeding events, prophylactic treatment, and presence of clotting factor inhibitors negatively impact the HRQoL of patients with hemophilia.
Keywords: Adult patients, hemophilia, health-related quality of life, Saudi Arabia
|How to cite this article:|
Allhaidan MA, Almashaan AM, Alduaij AA, Altuwaijri HA, Alotaibi LA, Almomen AM. Health-related quality of life in adult patients with hemophilia, Riyadh, Saudi Arabia. J Appl Hematol 2018;9:5-10
|How to cite this URL:|
Allhaidan MA, Almashaan AM, Alduaij AA, Altuwaijri HA, Alotaibi LA, Almomen AM. Health-related quality of life in adult patients with hemophilia, Riyadh, Saudi Arabia. J Appl Hematol [serial online] 2018 [cited 2022 Sep 27];9:5-10. Available from: https://www.jahjournal.org/text.asp?2018/9/1/5/228330
| Introduction|| |
Hemophilia is a life-long X-linked disorder characterized by recurrent bleeding episodes, predominantly into joint spaces.,, According to the World Federation of Hemophilia Annual Global Survey 2015, hemophilia accounts for 54.79% of all bleeding disorders in Saudi Arabia. Despite the fact that mortality rates have been successfully reduced by advanced treatment strategies, patients with hemophilia continue to suffer from irreversible damage to their joints as a consequence of repeated bleeding episodes. Subsequently, patients experience pain, stiffness, and arthropathies, which lead to limitations of their movements and, eventually, potential disabilities., Not only does hemophilia restrict patients from their physical activities, but also it impacts many aspects of these patients' lives such as emotional, psychological, and social wellbeing.
Clinical assessment of disease status in hemophilia patients can be performed either by physical and radiological examination or by monitoring the recurrence and severity of joint bleeds. Although it is important to assess patients' clinical condition, these approaches do not accurately reflect patients' perception of the disease and its impact on their health-related quality of life (HRQoL).
Many studies showed that prophylactic treatment is better than on-demand treatment in the long-term, as it has a major impact on the patient's HRQoL in terms of improving the physical functioning and reducing the severity of joint deformities, pain, hospital admissions, and the need for joint replacement surgeries.,,,
HRQoL is defined as the patient's perception of a disease and its treatment impact on different dimensions of life., Assessment of HRQoL represents an attempt to determine how different life aspects are affected by the patient's condition. For life-long, incurable diseases such as hemophilia, it is important to assess HRQoL to guide future decision-making related to disease management and the improvement of overall QoL in those patients.,,
In Saudi Arabia, there is a scarcity in demonstrating how hemophilia affects the HRQoL of those patients. Our present study aimed to assess HRQoL in adult patients with hemophilia, managed at King Khalid University Hospital (KKUH), Riyadh, Saudi Arabia, using the Arabic version of the Hemophilia-Specific QoL (HAEMO-QoL-A) questionnaire. We also aimed to identify the factors affecting HRQoL in those patients.
| Subjects and Methods|| |
This was an observational cross-sectional study conducted in a tertiary hospital, KKUH, in Riyadh, Saudi Arabia. Sixty-one patients followed up and treated at KKUH from 2000 to 2015 were identified through medical records and enrolled in the study. Study participants were 18 years and older with an established diagnosis of hemophilia. Patients under 18 years of age, females, and those with acquired hemophilia were excluded from the study.
Volunteered medical students who underwent training and interview simulation collected the patients' data. Due to the difficulty in reaching each patient in person as some of them live outside Riyadh and others do not show up for their regular follow-up at the clinic, it was more feasible to interview them by phone. Patients were asked to complete a translated version of the predesigned Hemophilia-Specific QoL Questionnaire for Adults (HAEMO-QoL-A), and permission was obtained from the original authors of the questionnaire. Two independent native Arabic speakers who are fluent in English performed a forward translation of the questionnaire from English to Arabic language, and a hematology consultant evaluated it. A backward translation was also done to ensure efficient translation.
The HAEMO-QoL-A questionnaire contained 41 questions, each with six options, distributed into four sets of domains that evaluated the effect of hemophilia on “day-to-day activates,” “mood and feelings,” “work or school life, family life, and social life,” and patient's experience with “hemophilia treatment,” containing 11, 11, 14, and 5 questions, respectively. Considering the cultural variety and the sensitivity toward asking about intimate relationships, the following question “hemophilia interferes with my ability to have an intimate relationship with another person” was removed. Patients were asked to take into consideration the past 4 weeks while answering the questions. For HRQoL scoring, items included in each domain were summed across the questionnaire. Owing to the variety in the way of writing the questions, as some questions were written in a negative manner and others in a positive manner, reversal of the scores of negative questions was done so that higher scores indicate better HRQoL. Majority of questions were positives except the following negative items (3, 6, 7, 9, 12, 16, 19, 32, and 34), and for those negative items were reversed as follows: 0 = 5, 1 = 4, 2 = 3, 3 = 2, 4 = 1, and 5 = 0.
Demographic and clinical data were obtained from the patients themselves including age, residency, educational level, type of hemophilia, inhibitors status, previous episodes of bleeding into joints, previous episodes of bleeding elsewhere, number of bleeding events in the past year, previous joint replacement surgery, type of hemophilia treatment, use of pain medications, and comorbidities (diabetes, hypertension, asthma, heart disease, and cancer).
A pilot study was done involving ten hemophilia patients who met the inclusion criteria. The aims of the pilot study were to ensure patients' understanding of the translated version of the HAEMO-QoL-A questionnaire and to verify the time estimated to complete the interview. No items were identified for further modification, and 10 to 15 min were allocated for each patient.
Data were analyzed using Statistical Package for the Social Sciences (SPSS) software version 21.0, (IBM Corp., Armonk, NY). Descriptive statistics (frequencies, percentages, mean, and standard deviation [SD]) were used to describe categorical and quantitative data and outcome variables. The Pearson Chi-square test was performed to test the association between categorical data and outcome variable. P<0.05 was considered statistically significant. Student's t-test and one-way analysis of variance were used to compare the mean values. Spearman's rank correlation was used to observe the correlation between quantitative variables. Reliability analysis was applied to determine the internal consistency of the HAEMO-QoL-A questionnaire with a total Cronbach's alpha coefficient of 0.83 (P< 0.0001).
Informed consent was provided by all patients. This included a brief explanation of the study aim and stated that the patients' participation is voluntary, without any reward and with the full right to withdraw at any time. Patients were assured that their information will be kept confidential and will only be used for research purposes.
| Results|| |
A total of 61 patients enrolled in the study; one refused to participate in the study and 11 were out of reach due to incorrect phone numbers. Thus, 49 patients (80.3% response rate) completed the HAEMO-QoL-A questionnaire.
Of those participants, 40 (81.6%) had hemophilia A and 9 (18.4%) had hemophilia B, with a mean age of 29.84 years (SD 8.34). The most commonly reported target joints were the knee (89.7%), ankle (69.2%), and elbow (56.4%). The presence of clotting factor inhibitor was detected in six patients (12.2%). The majority of subjects used on-demand treatment (55.1%). The patients' clinical and demographical characteristics are described in [Table 1].
|Table 1: Clinical and demographic characteristics of hemophilia patients (n=49)|
Click here to view
The average HAEMO-QoL-A total score was 99.89, ranging from 44 to 146. The “treatment concerns” dimension had the lowest average score (13.69), which indicates a worse QoL, while the “work/school, family, and social life” dimension was the least affected among the participants (mean 39.32), as shown in [Table 2].
One of the four HAEMO-QoL-A subscales (day-to-day activities) had a significant correlation with the number of bleeding events in the past year (P = 0.012); patients with more number of bleeding events in the past year had lower scores in that dimension. While the other HAEMO-QoL-A subscales (mood and feelings; work/school, family, and social life; and treatment concerns) and the total score had no correlations with the number of bleeding events in the past year (P = 0.426, 0.437, 0.472, and 0.166, respectively).
Significant correlations were detected between clinical characteristics and HAEMO-QoL-A mean scores [Table 3]. When comparing patients who were receiving on-demand treatment to those who were receiving prophylactic treatment, patients receiving on-demand treatment were shown to have higher (better) mean scores in “day-to-day activities” and “work/school, family, and social life” subscales and mean total score (P = 0.010, 0.012, and 0.010, respectively). The presence of clotting factor inhibitors was associated with lower (worse) mean score in the “treatment concerns” subscale and mean total score than those without inhibitors (P = 0.042 and 0.030, respectively). Patients who reported to have target joints, when compared to those who do not, exhibited lower (worse) mean total and subscale scores. HAEMO-QoL-A mean scores were able to distinguish between subjects who underwent joint replacement surgery and those who did not; patients without joint replacement surgery showed significantly higher (better) mean total and subscale scores, except in the “day-to-day activities” dimension [Table 3]. The use of pain medications was associated with lower (worse) mean total and “work/school, family, and social life” scores (P = 0.012 and 0.015, respectively). No significant difference in HAEMO-QoL-A scores was detected between patients with comorbidities and those without (97.66 vs. 100.88; P = 0.714). Those demographic characteristics that did not show a significant correlation with HAEMO-QoL-A scores are also presented in [Table 3].
|Table 3: Demographic and clinical variables for hemophilia patients and its association with Hemophilia-Specific Quality of life A mean scores and their statistical significance|
Click here to view
There is a statistically significant association between the type of treatment received and the reported mean number of bleeding events in the past year. Patients treated with both on-demand and prophylaxis had significantly higher average bleeding events (33.50) compared to those who reported only prophylaxis (13.36) or on-demand alone (8.22) as their treatment strategies (P = 0.007) [Figure 1].
|Figure 1: Relation between type of hemophilia treatment and mean number of bleeding in the past year with a (P = 0.007)|
Click here to view
| Discussion|| |
HRQoL refers to the subjective evaluation of the impact of a certain disease on different life aspects including physical, social, and emotional well-being., For patients with chronic diseases, such as hemophilia, for which there is no cure and treatment is lifelong, HAEMO-QoL-A is considered an important outcome measure that should be assessed to determine the aspects that are most affected by the disease so that necessary actions are taken into consideration to improve the overall QoL in those patients.,,
The mean total score of the HAEMO-QoL-A in this study was 99.89. However, there is no cutoff value that indicates whether the patient is having impaired QoL or not. Many studies conducted in different countries have assessed the QoL in adult patients with hemophilia using different instruments.,,,,, To allow comparison between our study and those conducted in other centuries, we transformed the mean total HRQoL scores of each country on scales ranging from 0 to 100, with higher scores indicating better QoL [Table 4]. Upon comparison of the HRQoL scores, an international study conducted by Rentz et al. had the highest mean total score (73.12). On the other hand, our score, which is similar to the score of a study conducted in Iran by Dolatkhah et al., was relatively low when compared to other countries.
|Table 4: Mean total scores of hemophilia patients' quality of life in different countries using different instruments|
Click here to view
Furthermore, it is noteworthy that the “treatment concern” is the most impaired dimension in our study, which could be a result of the invasive and inconvenient factor infusion. In addition, the majority of the patients in this study have emphasized the fear of being treated by health-care providers who do not know how to manage their condition. Adding to that, there is only one specialized center for the treatment of hemophilia in Saudi Arabia, which as assumed by Alzubaidy et al. to be insufficient compared to the Saudi population and the country's financial recourses.
Prophylactic treatment was shown to worsen HRQoL, and this finding could be attributed to the clinical practice in Saudi Arabia; as prophylaxis is mainly given to severe cases. However, patients with more severe disease tend to require prophylactic treatment; hence, it is the severity of the disease that leads to poor QoL rather than being on prophylaxis. Similar results were reported by Rentz et al., who observed that 90% of patients who were on prophylactic treatment had severe disease, whereas 43% of those receiving on-demand treatment had severe disease. In addition, these authors stated that patients who are currently on prophylactic treatment might be doing so because of an acute deterioration of their disease, which could bias HRQoL outcomes. Nevertheless, it is noteworthy to state that this finding can be attributed to data collection method that was done through phone interview.
Joint replacement surgery was negatively correlated with patients' HRQoL, which is in contrast to the findings reported in a previous study. These findings might be due to the few subjects (14.3%) who underwent joint replacement surgery. Moreover, we believe that it may be because those patients who underwent joint replacement surgery already had severe underlying joint injury and arthritis, which worsened their HRQoL. However, as we mentioned earlier it could be also attributed to the data collection method used in this study. Further studies with more accurate data collection method are needed to better clarify this discrepancy.
This study has limitations, one of which is that it was conducted in one tertiary hospital in Riyadh, which restricts the generalization of the results. In addition, the severity of the disease was not recorded. The small sample size is also a drawback, which limits the power of estimating the impact of variables on HRQoL.
| Conclusion|| |
Despite the above-mentioned limitations, this study provides an initial step in addressing the HRQoL in hemophilia patients in Saudi Arabia. Future studies in other regions of the country are now needed to be able to generalize the results. Furthermore, educational programs targeting health-care providers are recommended to improve the health services delivered to hemophilia patients, in addition to workshops aiming to rise patients' education about self-treatment. This study provided an insight into the patients' experiences with hemophilia treatment, which was the most impaired QoL dimension. For this reason, physicians should take those factors into consideration in their clinical practice to yield better QoL outcomes.
The authors would like to express their deep gratitude to all patients who participated in this study, and also would like to thank the data collectors for their help.
Financial support and sponsorship
Conflicts of interest
There are no conflicts of interest.
| References|| |
Mercan A, Sarper N, Inanir M, Mercan HI, Zengin E, Kiliç SÇ, et al.
Hemophilia-specific quality of life index (Haemo-qoL and haem-A-qoL questionnaires) of children and adults: Result of a single center from Turkey. Pediatr Hematol Oncol 2010;27:449-61.
Flood E, Pocoski J, Michaels LA, Bell JA, Valluri S, Sasanè R, et al.
Illustrating the impact of mild/moderate and severe haemophilia on health-related quality of life: Hypothesised conceptual models. Eur J Haematol 2014;93 Suppl 75:9-18.
Gringeri A, Ewenstein B, Reininger A. The burden of bleeding in haemophilia: Is one bleed too many? Haemophilia 2014;20:459-63.
Ferreira AA, Leite IC, Bustamante-Teixeira MT, Corrêa CS, da Cruz DT, Rodrigues Dde O, et al.
Health-related quality of life in hemophilia: Results of the hemophilia-specific quality of life index (Haem-a-qol) at a Brazilian blood center. Rev Bras Hematol Hemoter 2013;35:314-8.
Dolatkhah R, Fakhari A, Pezeshki MZ, Shabanlouei R, Tavassoli N, Gholchin M, et al.
Social determinants and health-related dimensions of quality of life in adult patients with haemophilia. Haemophilia 2014;20:376-81.
Rentz A, Flood E, Altisent C, Bullinger M, Klamroth R, Garrido RP, et al.
Cross-cultural development and psychometric evaluation of a patient-reported health-related quality of life questionnaire for adults with haemophilia. Haemophilia 2008;14:1023-34.
Remor E. Quality of life in hemophilia. Curr Future Issues Hemophilia Care 2011:49-52.
Lindvall K, Von Mackensen S, Berntorp E. Quality of life in adult patients with haemophilia – a single centre experience from Sweden. Haemophilia 2012;18:527-31.
Kodra Y, Cavazza M, Schieppati A, De Santis M, Armeni P, Arcieri R, et al.
The social burden and quality of life of patients with haemophilia in Italy. Blood Transfus 2014;12 Suppl 3:s567-75.
Varaklioti A, Kontodimopoulos N, Katsarou O, Niakas D. Psychometric properties of the greek haem-A-qoL for measuring quality of life in Greek haemophilia patients. Biomed Res Int 2014;2014:968081.
AL-Zubaidy A. Descriptive study of hemophilia in Al-Ramadi city. Diyala J Med 2014;6:55-9.
Zaher G, Adam S. Outcomes of congenital bleeding disorders. Bahrain Med Bull 2012;34:78-81.
[Table 1], [Table 2], [Table 3], [Table 4]
|This article has been cited by|
||The effect of emicizumab prophylaxis on long-term, self-reported physical health in persons with haemophilia A without factor VIII inhibitors in the HAVEN 3 and HAVEN 4 studies
| ||Mark W. Skinner,Claude Négrier,Ido Paz-Priel,Sammy Chebon,Victor Jiménez-Yuste,Michael U. Callaghan,Michaela Lehle,Markus Niggli,Johnny Mahlangu,Amy Shapiro,Midori Shima,Avrita Campinha-Bacote,Gallia G. Levy,Johannes Oldenburg,Sylvia Mackensen,Steven W. Pipe |
| ||Haemophilia. 2021; |
|[Pubmed] | [DOI]|