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 Table of Contents  
ORIGINAL ARTICLE
Year : 2020  |  Volume : 18  |  Issue : 2  |  Page : 146-151

Clinical audit on management of β-thalassemia major in Minia General Hospital


Department of Pediatric, Faculty of Medicine, Al-Azhar University, Assiut, Egypt

Date of Submission15-Jun-2019
Date of Decision05-Sep-2019
Date of Acceptance01-Oct-2019
Date of Web Publication24-Jul-2020

Correspondence Address:
MBBCh Mostafa F Ibrahim
Department of Pediatric, Faculty of Medicine, Al-Azhar University, Assiut, 61661
Egypt
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Source of Support: None, Conflict of Interest: None


DOI: 10.4103/AZMJ.AZMJ_85_19

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  Abstract 


Background Beta-thalassemia is one of the common genetic disorders in our community. Therefore, it is deemed necessary to study and probe it to encompass all aspects of this disorder.
Aim The aim of this study was to determine the adherence of management of β-thalassemia major in Minia General Hospital to an established Standard for the Clinical Care of Children with Thalassemia in the UK.
Patients and methods This study included 100 children with β-thalassemia major who were receiving regular blood transfusion; data of the patients were collected and analyzed for audit against established Standards for the Clinical Care of Children with Thalassemia in the UK; values were expressed in terms of percentage.
Results The diagnosis of β-thalassemia major in our study was made mainly on the basis of the clinical background confirmed by complete blood count, hemoglobin electrophoresis, and serum ferritin level; the frequency of investigations in our study showed that abdominal ultrasound was performed regularly in most of our patients (93%), followed by liver function (74%), serum ferritin (65%), hepatitis marker (57%), echocardiography (47%), and assessments of weight and height − sitting and standing (43%), follicle-stimulating hormone, luteinizing hormone (7%), thyroid-stimulating hormone, triiodothyronine, thyroxine (7%), fundus examination (7%), audiometry (3%), oral glucose tolerance test (3%), vitamin D level (3%), and DEXA scan (1%); the assessment of puberty was not performed. The most common complication in our study was splenomegaly (80%), followed by bone deformities (76%), slowed growth rates (69%), and infection (47%), followed by bone osteoporosis (27%), heart problems (24%), chronic hepatitis, or liver fibrosis (19%). Iron-chelation therapy was used by 53% of our patients. Deferasirox is the main line (38%); hydroxyurea was used by 41% of our patients.
Conclusion As a conclusion, we found that lines of management of β-thalassemia major in Minia General Hospital were not enough and not performed systematically in comparison with Standards for the Clinical Care of Children with Thalassemia in the UK.

Keywords: chelation therapy, iron overload, thalassemia major, UK, β-blood transfusion


How to cite this article:
Ibrahim MF, Ahmed HM, Hassan KH. Clinical audit on management of β-thalassemia major in Minia General Hospital. Al-Azhar Assiut Med J 2020;18:146-51

How to cite this URL:
Ibrahim MF, Ahmed HM, Hassan KH. Clinical audit on management of β-thalassemia major in Minia General Hospital. Al-Azhar Assiut Med J [serial online] 2020 [cited 2020 Oct 25];18:146-51. Available from: http://www.azmj.eg.net/text.asp?2020/18/2/146/290610




  Introduction Top


β-thalassemia is one of the most common monogenic diseases in the world [1].

The clinical manifestations of β-thalassemias are highly variable. They range from asymptomatic cases with mild (silent) mutations, to those with mild hypochromic anemia, to others with moderate and severe lifelong transfusion-dependent anemia and multiorgan involvement [2].

Patients with severe forms present during infancy with worsening anemia and are transfusion dependent for life [3].

Numerous attempts in preclinical and early clinical studies that aim to discover a cure for all patients have met with variable success thus far [4]. Therefore, a majority of patients with transfusion-dependent β-thalassemia are managed medically with regular blood transfusions and iron-chelation therapy for life [5].

β-thalassemia is the most frequent hemoglobinopathy in Egypt, with a carrier rate of 5.3–9% and an estimated 1000 infants born each year with β-thalassemia major among the national 1.5 million live births. The estimated average financial cost for β-thalassemia management in Egypt is $10 million/year and is on the increase. The registered cases of β-thalassemia in the main centers in Egypt are 9912 patients, with the highest numbers in the Hematology Unit of the New Cairo University Children Hospital [6].

Clinical audit is defined by National Institute for Clinical Excellence as ‘A quality improvement process that seeks to improve patient care and outcomes through systematic review of care against explicit criteria and implementation of change. Aspects of structure, processes, and outcomes of care are selected and systematically evaluated against explicit criteria. Where indicated, changes are implemented at individual, team, or service level and further monitoring is used to confirm improvement in healthcare delivery’ [7].

Clinical audit runs in a cycle where the first step is the development of standards. Traditionally, standards have been developed by systematic reviews to identify sets of criteria that constitute optimal care. Once standards have been developed, actual practice is measured and compared with standards (best practice). Gaps in current practice are identified; recommendations are made and implemented [7].


  Aim Top


The aim of this study was to determine the adherence of management of β-thalassemia major in Minia General Hospital to an established Standard for the Clinical Care of Children with Thalassemia in the UK (3rd ed.), published by UK Thalassaemia Society in 2016 ISBN (print edition): 978-1-900254-20-5.


  Patients and methods Top


This study was designed as an audit to evaluate the quality of medical care provided to children with β-thalassemia major delivered by the Pediatric Department in Minia General Hospital, Egypt, in addition to identification of the management, therapy, and patient’s compliance, with the aim of determining potentials for improvement.

A number of children with β-thalassemia major who were receiving regular blood transfusion were recruited. They were registered for transfusion management. A total of 100 patients were recruited (62 males and 38 females).

Inclusion criteria

  1. Known cases with β-thalassemia major irrespective of their sex.
  2. Transfused, as a part of their management, at least 10 U of blood at our unit.
  3. Age: younger than 13 years.


Exclusion criteria

  1. Known cases with β-thalassemia major who had received transfusion of less than 10 U of blood at our unit.
  2. Age: older than 13 years.


A review of the patient’s medical history was performed for every patient on the basis of data obtained from the thalassemia patient follow-up card.

Ethical consideration and consent

Informed consent was obtained from the parents for collection of data and the research under the approval of Azhar Assiut University and Minia General Hospital.

The following data were collected and recorded for each patient in a master sheet for the audit:
  1. Sociodemographic data characteristics such as name, age, and sex.
  2. Use of iron-chelating agents, blood transfusion (frequency, volume, pretransfusion hemoglobin level), splenectomy, and indication of splenectomy and complications.
  3. Complete history.
  4. Physical examination.
  5. Investigations that were recommended to be performed regularly:
    1. Every 3 months:
      1. Liver function test.
      2. Serum ferritin level.
    2. Every 6 months
      1. Assessments of weight and height − sitting and standing (from diagnosis until final adult height).
      2. Assessment of puberty more than 10 years.
      3. Calcium, phosphate, alkaline phosphatase levels more than 12 years.
    3. Every 1 year:
      1. Echocardiography more than 10 years.
      2. Fundus examination more than 10 years.
      3. Audiometry more than 10 years.
      4. Thyroid-stimulating hormone (TSH), triiodothyronine (T3), thyroxine (T4) more than 12 years.
      5. Follicle-stimulating hormone (FSH), luteinizing hormone (LH) more than 10 years.
      6. Oral glucose tolerance test more than 10 years.
      7. Vitamin D level more than 2 years.
      8. Hepatitis viral screening [hepatitis B virus (HBV), hepatitis C virus (HCV)].
      9. Radiography (abdomen–hand–skull) more than 10 years.
      10. Abdominal ultrasound (liver–spleen–gall bladder).
    4. Every 2 years:
      1. DEXA scan.


Treatment that was administered to the patient other than transfusion

  1. Iron-chelating agents.
  2. Hydroxyurea.
  3. Supportive treatment (vitamins and minerals).


Statistical analysis

Data were collected, revised, verified, coded, and then entered into a PC for statistical analysis, which was carried out using the IBM SPSS statistical package, version 20 (IBM; IBM SPSS Inc., Chicago, USA).

The following had been carried out.

Descriptive statistics

  1. For qualitative data: number (n) and percentage (%).
  2. For quantitative data: mean and SD.


Analytical statistics

  1. Independent quantitative data comparison among two groups by an independent-sample t test and among three groups by the one-way analysis of variance test.
  2. Comparison of categorical data by the χ2 test.



  Result Top


The study was designed as an audit on 100 cases (62 males and 38 females) with β-thalassemia major in children admitted to the Pediatric Department in Minia General Hospital.

A number of children receiving a regular blood transfusion were included. They were registered for transfusion management.

The frequency of investigations in our audit and results showed that 65% of the patients underwent assessments of serum ferritin level every 3 months, 74% of patients underwent serum liver function tests every 3 months, 43% of patients underwent Assessments of weight and height − sitting and standing every 6 months, none of the patients underwent assessment of puberty more than 10 years every 6 months, 10% of patients underwent assessments of calcium, phosphate, alkaline phosphatase levels more than 12 years every 6 months, 46.7% of patients underwent echocardiography more than 10 years every 1 year, 6.7% of patients underwent fundus examination more than 10 years every 1 year, 3.3% of patients underwent audiometry more than 10 years every 1 year, 6.7% of patients underwent assessments of TSH, T3, and T4 more than 12 years every 1 year, 6.7% of patients underwent assessments of FSH, LH more than 10 years every 1 year, 3.3% of patients underwent an oral glucose tolerance test more than 10 years, 3% of patients underwent assessments of vitamin D level more than 2 years every 1 year, 57% of patients underwent hepatitis viral screening (HBV, HCV) every 1 year, 16.7% of patients underwent radiography (abdomen–hand–skull) more than 10 years every 1 year, 93% of patients underwent abdominal ultrasound (liver–spleen–gall bladder) every 1 year, and 1% of patients underwent a DEXA scan every 2 years ([Figure 1]).
Figure 1 Frequency of investigations in our audit.

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In terms of complications in our audit, the frequency of complications included bone deformities (76%), splenomegaly (80%), infection (35%), slowed growth rate (69%), liver fibrosis (19%), cardiac problems (24%), and osteoporosis (27%) ([Table 1]).
Table 1 Incidence of complications in our audit

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In terms of the frequency of use and type of iron-chelating agents administered to our patients, the study showed that 38% were on Deferasirox, 9% were on Deferiprone, none were on Desfereal, and 53% were not receiving iron-chelating agents ([Table 2]).
Table 2 Frequency of use and type of iron-chelating agents administered to our patients

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In terms of the frequency of use of use of hydroxyurea and other supportive measures (vitamins and minerals) administered to our patients, the study showed that 41% of the patients were receiving hydroxyurea and 59% of patients were not receiving hydroxyurea, whereas 97% of the patients were receiving supportive treatment (vitamins and minerals) and 3% of the patients were not receiving supportive treatment ([Table 3]).
Table 3 Frequency of use of use of hydroxyurea and other supportive measures (vitamins and minerals) administered to our patients

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  Discussion Top


Our study was designed as an audit on 100 cases of children admitted to the Pediatric Department in Minia General Hospital by performing follow-up of cases during frequent visits to our department. The results in our study were compared with the guidelines released by established Standards for the Clinical Care of Children with Thalassemia in the UK (3rd ed., 2016) [8], which is appropriate for our country and appropriate for thalassemia major patients.

Our study disagreed with investigations that must be done every 3 months in Standards for the Clinical Care of Children with Thalassemia in the UK which all (100%) patients should perform them as 65% only of patients performed. Serum ferritin as a test was not available in our hospital and a large number of patients had no health insurance, which made monitoring difficult as many of them could not afford the cost, whereas 74% of patients only underwent assessments of liver functions despite availability, but some patients were not willing to undergo the test regularly.

Our study disagreed with investigations that must be done every 6 months in Standards for the Clinical Care of Children with Thalassemia in the UK which all (100%) patients should perform them as only 43.0% of patients performed. Assessments of weight, height, and sitting were not performed and documented regularly, especially for the older children, but none of the patients underwent assessment of puberty more than 10 years as it was not requested and performed by the doctors in our hospital, whereas 10% of our patients only underwent assessment of calcium, phosphate, and alkaline phosphatase levels more than 12 years as two of these three tests were not available in our hospital; also, it was caused by decreased counseling to parents of its importance and importance of follow up every 6 months.

Our study disagreed with investigations that must be done every 1 year in Standards for the Clinical Care of Children with Thalassemia in the UK which all (100%) patients should perform them as only 46.7% of patients performed echo it was not available at our hospital and patients afforded the cost which is expensive. Therefore, many patients presented with cardiac problems, whereas only 6.7% of patients underwent fundus examination because of decreased referral of patients to the ophthalmology unit and also decreased counseling to parents, and 3.3% of patients underwent audiometry older than 10 year but the test was not available in our hospital, while 6.7% of patients older than 10 years performed FSH, LH as it was only requested when menstrual disturbances develops. Also, 6.7% of patients older than 12 years underwent assessments of TSH, T3, and T4 as these were performed only when patients manifested thyroid disorder symptoms; it was also expensive, whereas 3.3% of patients older than 10 years of age performed the oral glucose tolerance test as it was not requested routinely by doctors in our hospital and random blood sugar was assessed instead. Impaired glucose regulation and diabetes must be detected early to enable prompt treatment of hyperglycemia and intensification of iron-chelation therapy. Very intensive combined chelation can improve or normalize glucose metabolism in patients with impaired glucose regulation or noninsulin-dependent diabetes, mainly through marked increase of insulin secretion [9].

Our study had a number of limitations also with investigations that must be done every 1 year in Standards for the Clinical Care of Children with Thalassemia in the UK which all (100%) patients should perform them as 2% only of patients older than 2 years performed vitamin D level test as it was expensive and majority of patients on oral vitamin D and other supportive treatment, while 93% of patients performed abdominal ultrasound every 1 year every 1 year as it was available and free in our hospital, while 16.7% of patients performed radiography (abdomen–hand–skull) older than 10 years although it was available in our hospital but it was not requested by doctors in hospital, while 57% of patients performed hepatitis marker it was caused by decreased counseling to parents and patients of its importance and importance of follow up every year.

Our study disagreed with investigations that must be done every 2 years in Standards for the Clinical Care of Children with Thalassemia in the UK which all (100%) patients should perform them but only 1% of β-thalassemia major patients performed DEXA scan as it was very expensive and not available most of the time.Our audit was in agreement with a clinical audit done on 100 patients with β-thalassemia major at Assiut University children’s hospital as they found that investigations should be performed regularly in β-thalassemia patients: serum ferritin (63%), liver function (74%), fundus examination (5%), echocardiography (44%), blood glucose (32%), FSH, LH (2%), TSH, T3, T4 (2%), and hepatitis marker (45%). Also, none of the patients underwent a DEXA scan. The most common complication in this study was bone deformities (87.0%), followed by infection (47.0%) and slowed growth rates (45.0%), followed by chronic hepatitis or liver fibrosis (27%), endocrine problems (21.0%), bone osteoporosis and fractures (20.0%), and finally 27% glands affection in children older than 10 years hypogonadism in of patients, diabetes mellitus 0.0% of patients, and hypoparathyroidism 5.0% of patients [10].

This is in agreement with a study carried out on 200 patients with β-thalassemia major in El-Minia Governorate, which found that the frequency of HCV for all studied children where 77 (38.5%) were confirmed positive for anti-HCV antibodies. The remaining 123 (61.5%) were seronegative [11].

A study by Iqbal et al. [12] in Pakistan was not in agreement with our study as the frequency of HCV infection in β-thalassemia cases was found to be 19% (12% in males and 7% in females).

Recommendations

As a result of our findings, we recommend that children screened should do the following:
  1. Follow-up of hemoglobin level, maintenance of good hemoglobin levels by regular transfusion of adequate amount of blood, and assessment of post-transfusion hemoglobin levels in all patients as a routine investigation.
  2. Screening blood used for transfusion can prevent transmission of HBV and HCV.
  3. Regular measurement of serum ferritin, liver function, and other investigations in β-thalassemic children are important to decrease the incidence of complications.
  4. Provide adequate amount of HIB vaccination and pneumococcal vaccination for splenectomized patients. Use of prophylactic penicillin and up-to-date vaccination is recommended in splenectomized patients.
  5. Psychological support of β-thalassemia children is important.
  6. Use of iron-chelation therapy to improve survival.
  7. Good counseling to the patients and their parent about importance of follow up investigation to the patient health.
  8. Updated written guidelines should be put in any hematology units. Provide consultation at key milestones including at diagnosis, at initiation of regular transfusions, at initiation of chelation therapy, at times of major complications, and at transfer to an adult clinic in addition to annual review.


Financial support and sponsorship

Nil.

Conflicts of interest

There are no conflicts of interest.



 
  References Top

1.
Mettananda S, Higgs DR. Molecular basis and genetic modifiers of thalassemia. Hematol Oncol Clin North Am 2018; 32:177–191.  Back to cited text no. 1
    
2.
Weatherall DJ, Clegg JB. The thalassaemia syndromes. 4th. Oxford: Blackwell Science Ltd; 2001.  Back to cited text no. 2
    
3.
Taher AT, Weatherall DJ, Cappellini MD. Thalassaemia. Lancet 2018; 391:155–167.  Back to cited text no. 3
    
4.
Thompson AA, Walters MC, Kwiatkowski J, Rasko JEJ, Ribeil JA, Hongeng S et al. Gene therapy in patients with transfusion-dependent beta-thalassemia. N Engl J Med 2018; 378:1479–1493.  Back to cited text no. 4
    
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Marcon A, Motta I, Taher AT, Cappellini MD. Clinical complications and their management. Hematol Oncol Clin North Am 2018; 32:223–236.  Back to cited text no. 5
    
6.
El-Beshlawy A, Youssry I. Prevention of hemoglobinopathies in Egypt. Hemoglobin 2009; 33 (Suppl 1):S14–S20.  Back to cited text no. 6
    
7.
National Institute for Clinical Excellence. Principles for best practice in clinical audit. Oxford: Radcliffe Medical Press; 2002.  Back to cited text no. 7
    
8.
UK Thalassaemia Society. Standards for the Clinical Care of Children with Thalassaemia in the UK; 2016. Available at: standards.ukts.org. Accessed: (IBM SPSS Inc., Chicago, USA).  Back to cited text no. 8
    
9.
Rebulla P, Modell B. Transfusion requirements and effects in patients with thalassemia major. Cooleycare program. Lancet 1991; 337:277–280.  Back to cited text no. 9
    
10.
Ahmed SE. Clinical audit on thalassemia management at Assiut University Children’s Hospital [master degree]. Assiut: Assiut Faculty of Medicine; 2015.  Back to cited text no. 10
    
11.
Sawy AM. Detection of hepatitis C infection among β-thalassemia major children in El-Minia Governorate − Egypt [master degree]. Minia: Minia Faculty of Medicine; 2016.  Back to cited text no. 11
    
12.
Iqbal MA, Ghafoor MB, Malik SA, Leghari MS, Azhar M5U. Audit of beta-thalassemia cases at Sheikh Zayed Medical College/Hospital, Rahim Yar Khan. JSZMC 2015; 6:811–815.  Back to cited text no. 12
    


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