Ultrasonography as Biofeedback to Increase Muscle Activation During the Mendelsohn Maneuver in Healthy Adults.

The Mendelsohn Maneuver (MM) is a therapeutic strategy that targets reduced laryngeal elevation. Both clinicians and clients identify the MM as one of the more difficult interventions to teach and learn. The purpose of this study was to examine the effect of applying real-time ultrasound as visual feedback in teaching the MM to healthy adults. Twenty-four healthy adults were randomized to two-parallel groups. The standard care group (control group) received verbal instruction, verbal reinforcement, and tactile cueing while practicing the maneuver. The experimental group received the same instruction with additional real-time ultrasound as visual biofeedback. Participants completed a single session which consisted of baseline assessment, training, and post-training assessment. Outcomes were submental surface electromyography (sEMG) signal duration, maximum amplitude, and area under the curve. Statistical analysis revealed that training with feedback significantly increased submental sEMG activity during the MM; however, the addition of ultrasound as biofeedback did not significantly increase muscle activation when performing the MM over verbal instruction with verbal/tactile feedback alone. Both groups demonstrated significantly greater muscle activity measured by sEMG when applying the MM. Although the current study did not indicate that adding ultrasound biofeedback was superior to traditional training alone in teaching healthy adults to perform the MM, it does support the clinical use of biofeedback tools for learning swallowing maneuvers. Ultrasound may be a biofeedback option for people with language deficits or differences to learn a swallowing maneuver. Further studies are required to determine the clinical application of ultrasound as biofeedback on people with dysphagia.

Education and Protein Supplementation Improve Nutritional Biomarkers among Hypoalbuminemic Peritoneal Dialysis Patients: A Quasi-Experimental Design.

Protein-energy wasting is prevalent in peritoneal dialysis patients, which causes a heavy burden for individuals and healthcare systems. We aimed to investigate the effect of nutritional education, and/or protein supplementation on nutritional biomarkers in hypoalbuminemic peritoneal dialysis patients. A quasi-experimental study was conducted in two dialysis centers at Taipei Tzu Chi Hospital and Shin Kong Wu Ho-Su Memorial Hospital. Patients were allocated in three groups including control (n = 12), milk protein (n = 21) and soy protein (n = 20). All patients received dietary guidelines from dietitians and completed 3-day dietary records during monthly visits for consecutive three months. Nutrients were analyzed using Nutritionist Professional software. Blood urea nitrogen (BUN), creatinine, albumin, total protein, hemoglobin, serum calcium, phosphorus, sodium, and potassium were assessed monthly. Total cholesterol and triglycerides were measured every three months. After three-month intervention, protein intake (percent of total calories), and serum albumin were significantly increased in three groups. Protein, phosphorus intake, and BUN were increased in two intervention groups. Total serum protein increased in control and milk protein groups, and creatinine increased the control group. Serum phosphorus was not significantly changed. Nutritional education alone, or combined with protein supplementation, significantly improve protein intake, and nutritional status by increasing serum albumin, but not serum phosphorus in hypoalbuminemic peritoneal dialysis patients.

Enhanced proliferation and differentiation of mesenchymal stem cells by astaxanthin-encapsulated polymeric micelles.

Astaxanthin is a highly potent antioxidant which can be extracted from Haematococcus pluvialis when cultivated and induced at high stress conditions. Due to astaxanthin's hydrophobicity, methoxypolyethylene glycol-polycaprolactone (mPEG-PCL) copolymer was synthesized to form polymeric micelles for the encapsulation of astaxanthin. Astaxanthin-loaded polymeric micelles were then used to examine the effects on the proliferation and differentiation of human mesenchymal stem cells (MSCs). Dynamic light scattering (DLS) and Fourier transform infrared spectroscopy (FT-IR) confirmed astaxanthin was encapsulated into mPEG-PCL micelles. Astaxanthin loading and encapsulation efficiency, determined by UV/Vis spectroscopy, were 3.27% and 96.67%, respectively. After 48 h, a total of 87.31% of astaxanthin was released from the polymeric micelles. The drug release profile was better fit by the Michaelis-Menten type model than the power law model. The MSC culture results showed that culture medium supplemented with 0.5 µg/mL astaxanthin-encapsulated polymeric micelles led to a 26.3% increase in MSC proliferation over an 8-day culture period. MSC differentiation results showed that 20 ng/mL astaxanthin-encapsulated polymeric micelles enhanced adipogenesis, chondrogenesis, and osteogenesis of MSCs by 52%, 106%, and 182%, respectively.

Effects of Phytoestrogen Supplement on Quality of Life of Postmenopausal Women: A Systematic Review and Meta-Analysis of Randomized Controlled Trials.

BACKGROUND: The effect of phytoestrogen on postmenopausal quality of life is unclear. This study evaluated the effects of phytoestrogen supplement on quality of life for postmenopausal women. METHODS: We conducted a systematic review and meta-analysis of randomized controlled trials on the effects of phytoestrogen supplements on the quality of life of postmenopausal women. We searched PubMed, MEDLINE, Embase, and the Cochrane Central Register of Controlled Trials on March 31, 2018, for relevant randomized controlled trials. Two authors independently selected studies, assessed risk of bias, and extracted data. Disagreement was resolved through discussion with a third author. RESULTS: We involved 10 articles in the systematic review. 8 studies and a total of 1,129 subjects were included in the meta-analysis. The questionnaires used in the evaluation of quality of life were as follows: SF-36, 4 studies; MENQOL, 4 studies; For Short Form 36 surveys, phytoestrogen groups scored significantly higher for body pain (mean difference = 3.85, 95% confidence interval [CI] = [1.14, 6.57], P < 0.01), mental health (mean difference = 4.01, 95% CI = [1.49, 6.57], P < 0.01), and role limitations caused by emotional problems domains (mean difference = 3.83, 95% CI = [1.81, 5.85], P < 0.01). No statistically significant difference was obtained from Menopause-Specific Quality of Life surveys (vasomotor domain mean difference 0.14, 95% CI = [-0.08, 0.36], P = 0.20; physical domain mean difference 0.20, 95% CI [-0.08, 0.48], P = 0.15; psychological domain mean difference -0.10, 95% CI [-0.26, 0.07], P = 0.27; sexual domain mean difference -0.17, 95% CI [-0.42, 0.09], P = 0.19). CONCLUSION: Current evidence does not support phytoestrogen supplementation improving postmenopausal quality of life. Further comprehensive trials or long-term follow-up studies are warranted.

Therapeutic mechanism of combined oral chelation therapy to maximize efficacy of iron removal in transfusion-dependent thalassemia major - a pilot study.

OBJECTIVES: Three iron chelators are used to treat transfusion-dependent beta-thalassemia: desferrioxamine (DFO), deferasirox (DFX), and deferiprone (DFP). Compliance is low for DFO as it cannot be administered orally. Combined administration of DFP and DFX is orally available, however, the therapeutic mechanism is unknown. This pilot study investigated the iron removal mechanisms of DFX and DFP treatment in patients with transfusion-dependent thalassemia major. METHODS: Each patient received three treatments sequentially: (1) DFX monotherapy, (2) DFP monotherapy, and (3) DFX/DFP combination therapy with a four-day washout period between each treatment. Urine and stool specimens were collected to determine the primary outcome of iron excretion volumes. RESULTS: The mean iron excretion was seven times higher after combination therapy with DFX and DFP. Monotherapies also increased excretions volumes, though to a significantly lesser degree. Combined administration of DFX and DFP achieves maximum iron removal in transfusion-dependent thalassemia major compared to monotherapy with either drug. CONCLUSIONS: We suggest combination therapy in chronic severe iron overload cases, especially for patients in poor compliance with DFO/DFP combination therapy or those exhibiting poor iron removal from DFX or DFP monotherapy.

Effectiveness of Parenteral Glutamine on Methotrexate-induced Oral Mucositis in Children with Acute Lymphoblastic Leukemia.

High-dose methotrexate (HDMTX) is important for children with acute lymphoblastic leukemia (ALL). There is no effective treatment for patients with oral mucositis, which is a major side effect associated with HDMTX. Here, we reviewed the medical records of patients younger than 18 yr with newly diagnosed ALL in our hospitals from 2002 to 2013. According to the nationwide protocol (TPOG-ALL-2002), each patient received four courses of HDMTX (2.5 or 5 g/m2) during consolidation therapy. HDMTX courses with glutamine therapy were as the glutamine group, and intravenous glutamine (0.4 g/kg/day) was started within 48 h after the initiation of HDMTX for 3 consecutive days. HDMTX courses without glutamine were as the control group. A total of 347 HDMTX courses were administrated in the 96 children with ALL during the study period. The incidence of oral mucositis was significantly lower in the glutamine group than in the control group (3.8% vs. 17.6%; P = 0.004). In the glutamine group, no patients suffered from severe oral mucositis. No severe adverse effects associated with glutamine administration were noted. Accordingly, parenteral glutamine appears to be feasible and safe to prevent oral mucositis in patients receiving HDMTX.

PG2, a botanically derived drug extracted from Astragalus membranaceus, promotes proliferation and immunosuppression of umbilical cord-derived mesenchymal stem cells.

ETHNOPHARMACOLOGICAL RELEVANCE: Astragalus membranaceus is used to manage the deficiency of vital energy in traditional Chinese medicine and confirmed to have many biological functions. Mesenchymal stem cells (MSCs) possess immunosuppressive effects, and are widely used for regenerative medicine and immune disorders. AIMS OF STUDY: This study investigated the effects of Astragalus polysaccharides (APS) on umbilical cord-derived MSCs (UCMSCs), including morphology, surface marker expression, proliferation, differentiation, and in-vitro and in-vivo immunosuppressive capacities. MATERIALS AND METHODS: MSCs isolated from umbilical cords were used. PG2 injection, a botanically derived drug containing a mixture of APS, was added into the culture medium to prepare PG2-treated UCMSCs. The morphology, surface marker expression, proliferation, and differentiation of UCMSCs were determined. The in-vitro immunosuppressive effects of UCMSCs were examined by peripheral blood mononuclear cell (PBMC) proliferation assay. The in-vivo effects were evaluated by circulatory inflammation-associated cytokine levels in mice with septic peritonitis induced by cecal ligation and puncture (CLP) operation. RESULTS: Compared with control UCMSCs, UCMSCs had higher population doublings when exposed to PG2-containing medium (P = 0.003). The reduction rates of PBMC proliferation after phytohemagglutinin stimulation increased significantly when UCMSCs were treated with PG2 (P = 0.004). The serum levels of inflammation-associated cytokines, including TNF-α, IL-6, MCP-1, IFN-γ, and IL-1ß, were significantly lower at 6h after CLP in the mice receiving PG2-treated UCMSCs. CONCLUSIONS: Our results demonstrated that PG2 can enhance UCMSC proliferation and their in-vitro and in-vivo immunosuppressive effects. Consequently, UCMSCs can be obtained in earlier passages to avoid senescence, and sufficient cells can be acquired faster for clinical use. With stronger immunosuppressive effects, UCMSCs may treat immune disorders more effectively. Further studies are warranted.

Survival and complication rates in patients with thalassemia major in Taiwan.

BACKGROUND: While transfusion and iron chelation therapy for thalassemia major (TM) has improved dramatically in recent years, the consequences of this improvement (current rates of survival and TM-related complications) remain unknown. METHODS: This nationwide population-based cohort study analyzed 2007-2011 data obtained from the Taiwanese National Health Insurance Research Database. RESULTS: After excluding those patients receiving hematopoietic stem cell transplantation, we enrolled 454 patients with TM who received transfusion and chelation therapy (median age, 17.2 years). Among these patients, the mortality rate was 2.9% in 2007, 2.3% in 2008, 2.9% in 2009, 2.6% in 2010, and 0.7% in 2011. Heart was the most common target organ of TM-related complications. There were 80 patients (17.6%) with arrhythmia and 86 patients (18.9%) with congestive heart failure. Dysfunction of endocrine organs was common, and the most common endocrinopathy was hypogonadism (23.1%), followed by diabetes (21.2%). There were 75 patients (16.5%) with liver cirrhosis and 79 patients (17.4%) with osteoporosis. CONCLUSIONS: Adequate red blood cell transfusion and iron chelation is available to all patients with TM in Taiwan under the universal health insurance system, and has resulted in reduction of TM-related mortality to very low levels. As these patients get older, early detection of complications and adequate intervention are important to quality-of-life improvement.

Safe needling depths of upper back acupoints in children: a retrospective study.

BACKGROUND: Acupuncture is applied for treating numerous conditions in children, but few studies have examined the safe needling depth of acupoints in the pediatric population. In this study, we investigated the depths to which acupuncture needles can be inserted safely in the upper back acupoints of children and the variations in safe depth according to sex, age, weight, and body mass index (BMI). METHODS: We retrospectively studied computed tomography (CT) images of patients aged 4 to 18 years who underwent chest CT at China Medical University Hospital between December 2004 and May 2013. The safe depths of 23 upper back acupoints in the Governor Vessel (GV), Bladder Meridian (BL), Small Intestine Meridian (SI), Gallbladder Meridian (GB) and Spleen Meridian (SP) were measured directly from the CT images. The relationships between the safe depths of these acupoints and sex, age, body weight, and BMI were analyzed. RESULTS: The results indicated significant differences in safe needling depth between boys and girls in most upper back acupoints, except at BL42, BL44, BL45, BL46, GB21 and SP21. Safe depths differed significantly depending on age (p < 0.001), weight (p ≤ 0.01), and BMI (p < 0.05). Multiple regression analysis revealed that weight was the most crucial factor in determining the safe depth. CONCLUSIONS: Sex, age, weight, and BMI are relevant factors in determining the safe needling depths of upper back acupoints in children. Physicians should pay attention to wide variations in needle depth when performing acupuncture.

The Depths from Skin to the Major Organs at Chest Acupoints of Pediatric Patients.

Background. Acupuncture is applied to treat numerous diseases in pediatric patients. Few reports have been published on the depth to which it is safe to insert needle acupoints in pediatric patients. We evaluated the depths to which acupuncture needles can be inserted safely in chest acupoints in pediatric patients and the variations in safe depth according to sex, age, body weight, and body mass index (BMI). Methods. We retrospectively studied computed tomography (CT) images of pediatric patients aged 4 to 18 years who had undergone chest CT at China Medical University Hospital from December 2004 to May 2013. The safe depth of chest acupoints was directly measured from the CT images. The relationships between the safe depth of these acupoints and sex, age, body weight, and BMI were analyzed. Results. The results demonstrated significant differences in depth among boys and girls at KI25 (kidney meridian), ST16 (stomach meridian), ST18, SP17 (spleen meridian), SP19, SP20, PC1 (pericardium meridian), LU2 (lung meridian), and GB22 (gallbladder meridian). Safe depth significantly differed among the age groups (P < 0.001), weight groups (P < 0.05), and BMI groups (P < 0.05). Conclusion. Physicians should focus on large variations in needle depth during acupuncture for achieving optimal therapeutic effect and preventing complications.

Integrative approach to analyze biodiversity and anti-inflammatory bioactivity of Wedelia medicinal plants.

For the development of "medical foods" and/or botanical drugs as defined USA FDA, clear and systemic characterizations of the taxonomy, index phytochemical components, and the functional or medicinal bioactivities of the reputed or candidate medicinal plant are needed. In this study, we used an integrative approach, including macroscopic and microscopic examination, marker gene analysis, and chemical fingerprinting, to authenticate and validate various species/varieties of Wedelia, a reputed medicinal plant that grows naturally and commonly used in Asian countries. The anti-inflammatory bioactivities of Wedelia extracts were then evaluated in a DSS-induced murine colitis model. Different species/varieties of Wedelia exhibited distinguishable morphology and histological structures. Analysis of the ribosomal DNA internal transcribed spacer (ITS) region revealed significant differences among these plants. Chemical profiling of test Wedelia species demonstrated candidate index compounds and distinguishable secondary metabolites, such as caffeic acid derivatives, which may serve as phytochemical markers or index for quality control and identification of specific Wedelia species. In assessing their effect on treating DSS induced-murine colitis, we observed that only the phytoextract from W. chinensis species exhibited significant anti-inflammatory bioactivity on DSS-induced murine colitis among the various Wedelia species commonly found in Taiwan. Our results provide a translational research approach that may serve as a useful reference platform for biotechnological applications of traditional phytomedicines. Our findings indicate that specific Wedelia species warrant further investigation for potential treatment of human inflammatory bowel disease.

Hospice shared-care saved medical expenditure and reduced the likelihood of intensive medical utilization among advanced cancer patients in Taiwan--a nationwide survey.

PURPOSE: Hospice shared care (HSC) is a new care model that has been adopted to treat inpatient advanced cancer patients in Taiwan since 2005. Our aim was to assess the effect of HSC on medical expenditure and the likelihood of intensive medical utilization by advanced cancer patients. METHODS: This is a nationwide retrospective study. HSC was defined as using "Hospice palliative care (HPC) teams to provide consultation and service to advanced cancer patients admitted in the nonhospice care ward." There were 120,481 deaths due to cancer between 2006 and 2008 in Taiwan. Patients receiving HSC were matched by propensity score to patients receiving usual care. Of the 120,481 cancer deaths, 12,137 paired subjects were matched. Medical expenditures for 1 year before death were assessed between groups using a database from the Bureau of National Health Insurance. Paired t and McNemar's tests were applied for comparing the medical expenditure and intensive medical utilization before death between paired groups. RESULTS: Compared to the non-HSC group, subjects receiving HSC had a lower average medical expenditure per person (US$3,939 vs. US$4,664; p<0.001). The HSC group had an adjusted net savings of US$557 (13.3%; p<0.001) in inpatient medical expenditure per person compared with the non-HSC group. Subjects that received different types of HPC had 15.4-44.9% less average medical expenditure per person and significantly lower likelihood of intensive medical utilization than those that did not receive HPC. CONCLUSIONS: HSC is associated with significant medical expenditure savings and reduced likelihood of intensive medical utilization. All types of HPC are associated with medical expenditure savings.

Antitumor therapy using nanomaterial-mediated thermolysis.

Nanomaterial-based systems present several novel therapeutic opportunities for cancer therapy based solely upon their unique physical and chemical properties. Despite advances in current cancer treatment, collateral damage to neighboring healthy tissues still cannot be avoided. By exploiting the strong optical and/or electromagnetic properties offered by nanomaterials, they are being employed as thermal nanoscalpels for the ablation of cancer cells. Through surface functionalization, these nanomaterials can be specifically targeted to tumorous tissue allowing for an increase in therapeutic potential and reduction in side effects. Moreover, these features provide nanomaterials with a tunable surface which can be used to modify optical, magnetic, thermal and mechanical properties. This review highlights carbon nanomaterials, nanogolds, magnetic nanoparticles and emerging hybrids applied for the thermolysis of cancer cells.

Photothermolysis of glioblastoma stem-like cells targeted by carbon nanotubes conjugated with CD133 monoclonal antibody.

CD133(+) cells in glioblastoma (GBM) display cancer stem cell-like properties and have been considered as the culprit of tumor recurrence, justifying exploration of potential therapeutic modalities targeting CD133(+) cancer stem-like cells (CSCs). For photothermolysis studies, GBM-CD133(+) and GBM-CD133(-) cells mixed with various ratios were challenged with single-walled carbon nanotubes (SWNTs) conjugated with CD133 monoclonal antibody (anti-CD133) and then irradiated with near-infrared laser light. Results show that GBM-CD133(+) cells were selectively targeted and eradicated, whereas GBM-CD133(-) cells remained viable. In addition, in vitro tumorigenic and self-renewal capability of GBM-CD133(+) treated with localized hyperthermia was significantly blocked. Furthermore, GBM-CD133(+) cells pretreated with anti-CD133-SWNTs and irradiated by near-infrared laser 2 days after xenotransplantation in nude mice did not exhibit sustainability of CSC features for tumor growth. Taken altogether, our studies demonstrated that anti-CD133-SWNTs have the potential to be utilized as a thermal-coupling agent to effectively target and destroy GBM CSCs in vitro and in vivo. FROM THE CLINICAL EDITOR: Glioblastoma remains one of the most notorious cancer from the standpoint of recurrence and overall resistance to therapy. CD133+ stem cells occur among GBM cells, and may be responsible for the huge recurrence risk. This paper discusses a targeted elimination method of these cells, which may enable more efficient therapy in an effort to minimize or prevent recurrence.

Characterization of elemental and structural composition of corrosion scales and deposits formed in drinking water distribution systems.

Corrosion scales and deposits formed within drinking water distribution systems (DWDSs) have the potential to retain inorganic contaminants. The objective of this study was to characterize the elemental and structural composition of extracted pipe solids and hydraulically-mobile deposits originating from representative DWDSs. Goethite (alpha-FeOOH), magnetite (Fe(3)O(4)) and siderite (FeCO(3)) were the primary crystalline phases identified in most of the selected samples. Among the major constituent elements of the deposits, iron was most prevalent followed, in the order of decreasing prevalence, by sulfur, organic carbon, calcium, inorganic carbon, phosphorus, manganese, magnesium, aluminum and zinc. The cumulative occurrence profiles of iron, sulfur, calcium and phosphorus for pipe specimens and flushed solids were similar. Comparison of relative occurrences of these elements indicates that hydraulic disturbances may have relatively less impact on the release of manganese, aluminum and zinc, but more impact on the release of organic carbon, inorganic carbon, and magnesium.

Update on thalassemia treatment in Taiwan, including bone marrow transplantation, chelation therapy, and cardiomyopathy treatment effects.

Over the past few decades, Taiwan has seen striking improvements in the life expectancy of its 400 registered beta-thalassemia major (beta-TM) patients due mainly to adequate transfusion regimens and effective iron chelation therapy. Since 1995, Taiwanese citizens have enjoyed universal health care through National Health Insurance (NIH), receiving comprehensive treatment at minimal cost. In 1984, a national program for thalassemia prevention, control, and hematopoietic stem cell transplantation (HSCT) was initiated. Recent data show 1- and 2-year event-free survival rates of 85 and 78%, respectively. Chelation agents like deferoxamine (DFO), deferiprone (L1) and deferasirox (DFRA) are available in Taiwan, and therapy is tailored to individuals based on drug availability and tissue distribution of iron load. Intensive chelation regimens combining L1 and DFO are recommended in patients with cardiac complications, while DFRA has been found to be effective in reducing serum ferritin, with acceptable side effects. Here, we report advances in thalassemia treatment in Taiwan and suggest treatment guidelines.

Iron chelation therapy for patients with myelodysplastic syndrome.

Chronic blood transfusions are necessary for patients with hereditary anemia such as thalassemia, and for patients with myelodysplastic syndrome (MDS) who become anemic and transfusion-dependent. A common consequence of chronic transfusion is iron accumulation that can lead to organ damage. While there is general agreement regarding the value of iron chelation therapy to reduce the detrimental effects of iron overload in thalassemia major, the same is not true for MDS. The malignant nature of the disease and the relatively high cost of iron chelation therapy make cost-effectiveness an issue of great concern. Furthermore, the positive assessment of a drug's cost-effectiveness in one country does not necessarily justify its use in another country. More prospective studies are needed to identify the best iron chelator for patients with MDS as well as to identify those patients who will benefit most from iron chelation therapy.

In vitro photothermal destruction of neuroblastoma cells using carbon nanotubes conjugated with GD2 monoclonal antibody.

Despite aggressive multimodality therapy, most neuroblastoma-bearing patients relapse and survival rate remains poor. Exploration of alternative therapeutic modalities is needed. Carbon nanotubes (CNTs), revealing optical absorbance in the near-infrared region, warrant their merits in photothermal therapy. In order to specifically target disialoganglioside (GD2) overexpressed on the surface of neuroblastoma stNB-V1 cells, GD2 monoclonal antibody (anti-GD2) was conjugated to acidified CNTs. To examine the fate of anti-GD2 bound CNTs after incubation with stNB-V1 cells, rhodamine B was labeled on carboxylated CNTs functionalized with and without anti-GD2. Our results illustrated that anti-GD2-linked CNTs were extensively internalized by neuroblastoma cells via GD2-mediated endocytosis. In addition, we showed that anti-GD2 bound CNTs were not ingested by PC12 cells without GD2 expression. After anti-GD2 conjugated CNTs were incubated with neuroblastoma cells for 6 h and endocytosed by the cells, CNT-laden neuroblastoma cells were further irradiated with an 808 nm near-infrared (NIR) laser with intensity ramping from 0.6 to 6 W cm(-2) for 10 min which was then maintained at 6 W cm(-2) for an additional 5 min. Post-NIR laser exposure, and after being examined by calcein-AM dye, stNB-V1 cells were all found to undergo necrosis, while non-GD2 expressing PC12 cells all remained viable. Based on the in vitro study, CNTs bound with anti-GD2 have the potential to be utilized as a therapeutic thermal coupling agent that generates heat sufficient to selectively kill neuroblastoma cells under NIR laser light exposure.

Effects of chelation therapy on cardiac function improvement in thalassemia patients: literature review and the Taiwanese experience.

The iron chelators deferoxamine (DFO) and deferiprone (L1) have demonstrated their ability to normalize cardiac function in patients with iron overload-induced cardiac disease. However, conventional chelation with subcutaneous DFO fails to prevent iron deposition in two-thirds of thalassemia major patients, placing them at risk of heart failure and its complications. Deferiprone appears to be more effective in cardiac iron removal. The detection and management of heart complications have improved dramatically over the last 7 years. Non invasive techniques of quantifying iron burden via magnetic resonance imaging (MRI) have been validated. A better understanding of cardiac pathophysiology and improved ability to detect at-risk populations are yielding better outcomes and reduced morbidity. We continue to appraise readily available bedside tools for monitoring thalassemia patients with heart complications, and here we summarize studies from the literature and our own findings. Deferiprone chelation was found to be of statistically significant benefit in upgrading cardiac function and reducing iron accumulation. The use of echocardiography and MRI to closely monitor cardiac functions associated with iron overload complications and mortality has proved quite practical.

Effect of deferiprone on liver iron overload and fibrosis in hepatitis-C-virus-infected thalassemia.

To assess the effects of liver iron overload and fibrosis after treatment with a chelating agent in hepatitis C virus (HCV)-infected thalassemia, from April 1999 to July 2004, 45 patients with thalassemia major (age range 9-33 years, mean 19.3) received daily deferiprone (L1) for 23-60 months (75 mg/kg). The patients were divided into two groups on the basis of their hepatitis status (27 with, 18 without). Their serum was analyzed for alanine aminotransferase (GPT), aspartate aminotransferase (GOT), bilirubin (total/direct), r-glutamyl transpeptidase (r-GT), alkaline phosphatase (Alk-P), and ferritin. Liver iron overload and fibrosis were defined by a senior pathologist. No significant differences were demonstrated in serum levels of GPT, GOT, bilirubin, r-GT, Alk-P or ferritin; comparison was made for each group before and after L1 treatment. Iron scores were 2.3 +/- 0.9 and 2.8 +/- 0.9 for the hepatitis C negative and positive groups, respectively (p = 0.07), with liver fibrosis scores of 1.0 +/- 0.5 and 0.4 +/- 0.52 (p = 0.56). The two scores were not higher for the positive group. There was no evidence of: 1) greater iron overload and fibrosis in the HCV-infected thalassemic patients; 2) L1 inducing progressive hepatic fibrosis or worsening iron overload in HCV-infected thalassemic patients after long-term therapy; 3) further damage to liver cells associated with L1 treatment.