An Urgent Need for Improving Thalassemia Care in Adult Patients Due to a Wide Gap on Current Real-Life Practice and Recommended Clinical Practice Guidelines
Authors: Ekwattanakit Supachai , Hantaweepant Chattree , Khuhapinant Archrob , Siritanaratkul Noppadol , Viprakasit Vip 
Published: April 2021

Abstract
Background
Thalassemia syndromes are the most common hereditary hemolytic anemia worldwide. Since 2013, Thalassemia International Federation (TIF) has launched new standard clinical practice guidelines (CPG) for non-transfusion dependent thalassemia (NTDT) and transfusion dependent thalassemia (TDT). Based on these guidelines, several measures should be routinely implemented such as monitoring and surveillance of thalassemia related complications in order to early detect such complications for a proper clinical management. At present, there is no data in Thailand to show that how physicians are treating thalassemia patients especially on early detection of all complications and whether their real life practice has changed to follow those of both guidelines.
Objectives
To evaluate the performance of routine care, in terms of surveillance for thalassemia related complications and their prevalence in adult thalassemia patients during 2 periods; before and after 2 CPG were published.
Methods
In this retrospective study, we analyzed data from 3,233 adult thalassemia patients who were diagnosed and treated at our clinics at Department of Medicine, Siriraj hospital during 1994-2017. We divided them into 2 groups; those who have been treated and followed-up for 3 consecutive years during 2012-2014 and 2015-2017 (a period before and after the implement of CPG, respectively). Clinical data and laboratory results were collected. Complications were recorded including iron overload (IOL) (ferritin), transaminitis (ALT &gt;3x upper normal limit), diabetes mellitus (DM) or impaired fasting glucose (IFG)(fasting blood sugar), hypothyroidism (thyroid function test), low morning cortisol, vitamin D abnormality, viral infection (hepatitis B and C), osteopenia/osteoporosis (bone mineral density, BMD) and gallstones (ultrasonography or CT scan). To compare the performance of routine care from different treating physician groups, patients were categorized into 3 groups; those who attended thalassemia clinic (Thal) treated by mainly staffs and residents in hematology, those in private hematology clinic (Private) treated by mainly attending hematology staffs and residents in hematology, and those in internal medicine clinic (Non-hem) treated by internists and GP. Prevalence and surveillance rates for each complication between groups were analyzed by Independent t test (Chi-square or Fisher's exact test). This study was approved by local ethical committee.
Results
Total available 459 NTDT and 65 TDT adult patients were studied. Baseline characteristics were shown in Table 1. Three most common complications were osteopenia/osteoporosis (69.8%), gallstones (67.6%) and abnormal vitamin D level (67.6%). IOL is a complication that has been widely evaluated in all treatment groups (93.1%) followed by evaluation of liver function test (82.3%). However the rate of evaluation for other complications were significantly reduced and &lt;25% of patients were evaluated in several complications. This result suggests that the prevalence of thalassemia related complications might be underestimate the true prevalence and early detection of thalassemia related complications is still lacking.
To test whether the implement of CPG has any impact on real-life clinical practice, we found that the rate of complication surveillance has increased significantly for several endocrine complications (DM/IFG, hypothyroid, adrenal insufficiency and low vitamin D) only in the group of patients treated at thalassemia clinic but not in others (Figure1). This suggested that CPG have yet to be implemented by those physicians and further endorsement is highly required.
Conclusion
This study was the first study that evaluated the real-world practical management of thalassemia patient in terms of complication surveillance. In our adult thalassemia population, thalassemia related complications were not uncommon and some occurred early in adulthood. Surveillance rates of these complications were low in all clinics. After implement of thalassemia CPG, the surveillance rates for complications were increased only in patients treated at thalassemia clinic but not in other two clinics. A two different standard of clinical practice even within the same tertiary care hospital such as Siriraj hospital has called for an immediate policy change to improve and standardize a care for thalassemia patients in Thailand.

Disclosures
No relevant conflicts of interest to declare.