Peerapon Wong

Iron-chelating therapies in a transfusion-dependent thalassaemia population in Thailand: a cost-effectiveness study.

AbstractBackground and Objective: β-Thalassaemia is a major public health problem in Thailand. Use of appropriate iron-chelating agents could prevent thalassaemia-related complications, which are costly to the healthcare system. This study aimed to evaluate the cost effectiveness of deferoxamine (DFO), deferiprone (DFP) and deferasirox (DFX) in Thai transfusion-dependent β-thalassaemia patients from the societal perspective. Methods: A Markov model was used to project the life-time costs and outcomes represented as quality-adjusted life-years (QALYs). Data on the clinical efficacy and safety of all therapeutic options were obtained from a systematic review and clinical trials. Transition probabilities were derived from published studies. Costs were obtained from the Thai Drug and Medical Supply Information Center, Thai national reimbursement rate information and other Thai literature sources. A discount rate of 3% was used. Incremental cost-effectiveness ratios (ICERs) were presented as year 2009 values. A base-case analysis was performed for thalassaemia patients requiring regular blood transfusion therapy, while a separate analysis was performed for patients requiring low (i.e. symptom-dependent, less frequent) blood transfusion therapy. A series of sensitivity analysis and cost-effectiveness acceptability curves were constructed. Results: Compared with DFO, using DFP was dominant with lifetime cost savings of

Frequency of hemoglobin E/β-thalassemia compound heterozygotes with low hemoglobin F phenotype among cases with a diagnosis of hemoglobin E homozygote, determined by high-performance liquid chromatography, in prenatal control program for β-thalassemia

US91 117. Comparing DFX with DFO, the incremental cost was

Prevalence and predictors of cardiac and liver iron overload in patients with thalassemia: A multicenter study based on real-world data

US522 863 and incremental QALY was 5.77 with an ICER of

Evaluation of a Cryopreservation Procedure to Set Up a New Bone Marrow Transplant Unit Using Lymphocyte Proliferation Test

US90 648 per QALY. When compared with DFP, the ICER of DFX was

A blood transfusion leading to misdiagnosis of beta-thalassaemia carrier status

US106445 per QALY. A cost-effectiveness analysis curve showed the probability of DFX being cost effective was 0% when compared with either DFO or DFP, based on the cost-effectiveness cut-off value of

Non-Hodgkin lymphoma in South East Asia: An analysis of the histopathology, clinical features, and survival from Thailand

US2902 per QALY. When compared with DFP, DFX was cost effective only if the DFX cost was as low as

Q Sepharose micro-column chromatography: A simple screening method for identifying beta thalassemia traits and hemoglobin E carriers

US1.68 per 250mg tablet. The results of the analysis in patients requiring low blood transfusion therapy were not different from those of the base-case analysis. Conclusions: Our findings suggest that using DFP is cost saving when compared with conventional therapy, while using DFX is not cost effective compared with either DFO or DFP in Thai patients with transfusiondependent b-thalassaemia. Policy-makers and clinicians may consider using such information in their decision-making process in Thailand.

High-Dose Therapy (HDT) with Hematopoietic Stem Cell Transplantation (HSCT) Is Effective Therapy for Patients with Non-Hodgkin Lymphoma (NHL) and Central Nervous System (CNS) Involvement

Hemoglobin (Hb) E (HBB:c.79G>A)/β-thalassemia disease is the most common thalassemia syndrome in Southeast Asian countries with a high prevalence of Hb E. Even though patients could present with a wide spectrum of clinical severity, the disease accounts for half of the severe β-thalassemia patients worldwide [1]. Among individuals with a carrier state of β-thalassemia or Hb E and homozygous Hb E who could be at-risk couples for Hb E/β-thalassemia, individuals with Hb E/ β-thalassemia themselves, especially with mild severity, may still be able to have their own children and also could be at-risk couples. The problem could occur with few Hb E/β-thalassemia compound heterozygotes who have a very low Hb F phenotype and could be misdiagnosed with Hb E homozygote by Hb analysis [2–4]. Consequently, if their spouses had inherited Hb E allele, they could be at-risk couples for Hb E/β-thalassemia. To date, there are no data regarding the frequency of Hb E/β-thalassemia cases with a low Hb F phenotype in real-life prenatal control situations. Our study was conducted prospectively in prenatal control program for β-thalassemia in the lower north of Thailand, between February 2014 and May 2017. All couples with a phenotypic diagnosis of homozygous Hb E by high-performance liquid chromatography (HPLC: VARIANTTM) in one person, and heterozygous or homozygous Hb E in the other, were recruited. Phenotypic diagnosis of Hb E homozygote comprised of a major fraction of Hb E with Hb F proportion less than 10%, without Hb A. DNA methods to confirm their genotype of Hb E homozygote and to detect other β-thalassemia mutations [5, 6], together with α-thalassemia (Southeast Asian and Thai deletions) and α-thalassemia (3.7and 4.2-kb deletions) determinants [7], were performed in all samples with Hb E homozygote phenotype. The study was approved by the institutional ethical committee. Of the 6023 couples determined by HPLC, there were 792 subjects with a phenotype of Hb E homozygote identified. Among these, 464 couples met our requirement, including 25 with double diagnoses of Hb E homozygote. The mean (± SD) Hb E and Hb F proportions in 489 Hb E homozygotes were 77.87 ± 5.27 and 3.54 ± 1.82%, respectively. After performing genotypic diagnosis, five (1.0%) Hb E/βthalassemia subjects were identified (Fig. 1). All five samples had co-inherited either α-thalassemia or αthalassemia allele. Furthermore, all five cases had a spouse who had inherited Hb E, meaning they were atr isk couples for Hb E/β thalassemia near ly misdiagnosed (Table 1). * Peerapon Wong peeraponw@nu.ac.th

RISK OF A COUPLE HAVING A CHILD WITH SEVERE THALASSEMIA SYNDROME; PREVALENCE IN LOWER NORTHERN THAILAND

Prevalence of cardiac and liver iron overload in patients with thalassemia in real-world practice may vary among different regions especially in the era of widely-used iron chelation therapy. The aim of this study was to determine the prevalence of cardiac and liver iron overload in and the management patterns of patients with thalassemia in real-world practice in Thailand. We established a multicenter registry for patients with thalassemia who underwent magnetic resonance imaging (MRI) as part of their clinical evaluation. All enrolled patients underwent cardiac and liver MRI for assessment of iron overload. There were a total of 405 patients enrolled in this study. The mean age of patients was 18.8±12.5years and 46.7% were male. Two hundred ninety-six (73.1%) of patients received regular blood transfusion. Prevalence of cardiac iron overload (CIO) and liver iron overload (LIO) was 5.2% and 56.8%, respectively. Independent predictors for iron overload from laboratory information were serum ferritin and transaminase for both CIO and LIO. Serum ferritin can be used as a screening tool to rule-out CIO and to diagnose LIO. Iron chelation therapy was given in 74.6%; 15.3% as a combination therapy.

Secondary central nervous system relapse in diffuse large B cell lymphoma in a resource limited country: result from the Thailand nationwide multi-institutional registry

A bone marrow transplant (BMT) is one kind of standard treatment modality in advanced hemato-oncology. In order to set up a BMT unit, one of the important steps before starting a clinical program is to evaluate the cryopreservation procedure for stem cell storage. Twenty one bags of buffy coat were used to be the testing specimens. They were processed and frozen according to cryopreservation protocol and kept in liquid nitrogen for 2 weeks. The evaluation process was carried out with a lymphocyte proliferation test together with trypan blue staining. By measuring the optical density of each lymphocyte containing well after stimulation, the lymphocyte proliferation value (LPV) could be obtained. When comparing them before and after cryopreservation, the LPV was 2.064 ± 0.379 (mean ± SD) and 1.913 ± 0.546, (p = 0.314), respectively. At 2 weeks after cryopreservation, comparing between the frozen group and the unfrozen control, the LPV was 1.913 ± 0.546 and 0.486 ± 0.453, (p < 0.05), respectively. The LPV showed clear efficacy of the procedure, especially for preserving the cellular proliferation function. Our model of the cryopreservation procedure evaluation at pre-clinical phase by use of a buffy coat and lymphocyte proliferation test seems feasible for newly-established small BMT units. With these results, clinical transplantations can be performed with more confidence.