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3.
Mymensingh Med J ; 28(2): 364-369, 2019 Apr.
Artigo em Inglês | MEDLINE | ID: mdl-31086152

RESUMO

Thalassaemia patients are the highest consumers of blood. Recurrent exposure to allogenic red cell antigen puts this population at increased risk of alloimmunization. This causes delayed hemolytic transfusion reactions. So transfusion requirement increases. But no data regarding alloimmunization was available in Bangladesh. Aim of this study was to estimate the prevalence of alloimmunization and to find out the potential factors associated with its development. This analytical cross sectional study was done by enrolling 97 patients, received at least 10 units transfusions, through convenient sampling. Indirect and direct antiglobulin tests were done to detect immunization by spin tube technique keeping an autocontrol and carried out in the Department of Haematology & Department of Transfusion Medicine, Bangabandhu Sheikh Mujib Medical University (BSMMU), Dhaka, Bangladesh from March 2016 to March 2017. Polyclonal anti-AHG reagent was used. A total 21 patients developed alloimmunization (21.6%) and 4 patients (4.1%) developed autoimmunization. Age, gender, splenectomy and number of transfusion are shown significant risk factors for alloimmunization. Data from this study demonstrate that the RBC alloimmunization is significantly high in our country. So, pretransfusion antibody screening needs to be initiated in order to ensure safe transfusion and RBC phenotyping should be started before starting first transfusion to prevent alloimmunization.


Assuntos
Incompatibilidade de Grupos Sanguíneos/imunologia , Eritrócitos/imunologia , Isoanticorpos/sangue , Talassemia/imunologia , Talassemia/terapia , Reação Transfusional , Bangladesh/epidemiologia , Antígenos de Grupos Sanguíneos/imunologia , Incompatibilidade de Grupos Sanguíneos/epidemiologia , Transfusão de Sangue/estatística & dados numéricos , Estudos Transversais , Humanos , Prevalência , Talassemia/sangue , Talassemia/epidemiologia
4.
Ann Hematol ; 98(6): 1333-1339, 2019 Jun.
Artigo em Inglês | MEDLINE | ID: mdl-30891614

RESUMO

We determined the prevalence of incidental extracardiac findings (IEF) at Magnetic Resonance Imaging (MRI) potentially related to anemia and hypoxia in age- and sex-matched populations (N = 318) with thalassemia major (TM) and thalassemia intermedia (TI) enrolled in the Myocardial Iron Overload in Thalassemia network. Overall, IEFs were detected in 33.3% and 25.8% of patients with TI and TM, respectively (P = 0.114). TI and TM patients had elevated but comparable prevalence of renal, splenic and liver cysts, and vertebral hemangiomas while TI patients had a significant higher frequency of extramedullary hematopoiesis (EMH) (15.1% vs 4.4%; P = 0.002). The prevalence of total IEFs increased with advancing age. TI non-transfusion-dependent patients had a significantly lower frequency of renal cysts than TI transfusion-dependent patients (8.8% vs 26.4%; P = 0.005). The prevalence of renal cysts in the thalassemic population was significantly higher than that in the general population (19.2% vs 1.9%; P < 0.0001). Our data on renal cysts indicate a significant higher prevalence of these IEFs compared to the general population, suggesting the role of the inappropriate activation of the hypoxia-inducible factor system linked to the chronic hypoxia. The significant prevalence of IEF in thalassemia patients undergoing MRI for iron quantification should prompt the discussion of the inclusion of IEF in the MRI report.


Assuntos
Cistos/epidemiologia , Hemangioma/epidemiologia , Hematopoese Extramedular , Doenças Renais Císticas/epidemiologia , Neoplasias da Coluna Vertebral/epidemiologia , Esplenopatias/epidemiologia , Talassemia/complicações , Adulto , Distribuição por Idade , Anemia/complicações , Transfusão de Sangue , Cistos/diagnóstico por imagem , Cistos/etiologia , Feminino , Hemangioma/diagnóstico por imagem , Hemangioma/etiologia , Humanos , Hipóxia/complicações , Serviços de Informação , Quelantes de Ferro/uso terapêutico , Sobrecarga de Ferro/tratamento farmacológico , Sobrecarga de Ferro/epidemiologia , Sobrecarga de Ferro/etiologia , Itália/epidemiologia , Doenças Renais Císticas/diagnóstico por imagem , Doenças Renais Císticas/etiologia , Imagem por Ressonância Magnética , Masculino , Pessoa de Meia-Idade , Prevalência , Estudos Retrospectivos , Neoplasias da Coluna Vertebral/diagnóstico por imagem , Neoplasias da Coluna Vertebral/etiologia , Esplenopatias/diagnóstico por imagem , Esplenopatias/etiologia , Talassemia/sangue , Talassemia/terapia , Adulto Jovem
5.
PLoS One ; 14(3): e0214148, 2019.
Artigo em Inglês | MEDLINE | ID: mdl-30893381

RESUMO

Disease-related complications and management are different among patients with thalassemia. This study was aimed to review the prevalence, clinical risk factors for the complications and the management in patients with thalassemia in Thailand. A multicenter cross-sectional study was conducted in patients with thalassemia aged ≥ 18 years old. Thalassemia-related complications and management were reviewed. The clinical parameters significantly associated with the complications were analyzed by logistic regression methods. The prevalence of thalassemia-related complications was 100% in patients with transfusion-dependent thalassemia (TDT) and 58.8% in patients with non-transfusion-dependent thalassemia (NTDT). Advanced age was statistically associated with extramedullary hematopoiesis in both TDT and NTDT patients. Splenectomy was a significant risk factor for pulmonary hypertension in both groups of patients. Severe iron overload started earlier in patients with TDT than NTDT and was associated with diabetes mellitus (adjusted odds ratio (AOR) = 6.2, p-value = 0.02). Disease-related complications are more prevalent in patients with TDT than patients with NTDT. Splenectomy and advanced age were important risk factors for developing major complications in both groups. Early screening and management for specific disease-related complications should be considered in patients with thalassemia according to their clinical risk factors.


Assuntos
Hematopoese Extramedular , Hipertensão Pulmonar , Sobrecarga de Ferro , Talassemia , Adulto , Transfusão de Sangue , Estudos Transversais , Feminino , Humanos , Hipertensão Pulmonar/epidemiologia , Hipertensão Pulmonar/etiologia , Hipertensão Pulmonar/fisiopatologia , Sobrecarga de Ferro/epidemiologia , Sobrecarga de Ferro/etiologia , Sobrecarga de Ferro/fisiopatologia , Masculino , Prevalência , Tailândia/epidemiologia , Talassemia/complicações , Talassemia/epidemiologia , Talassemia/fisiopatologia , Talassemia/terapia
6.
Transfus Med ; 29(3): 185-192, 2019 Jun.
Artigo em Inglês | MEDLINE | ID: mdl-30740798

RESUMO

OBJECTIVES: To assess current knowledge of National Heart, Lung and Blood Institutes (NHLBI) and Thalassemia International Federation (TIF) recommendations, blood banking practices and perceived challenges among transfusion services in the management of patients with haemoglobinopathies. BACKGROUND: Previous reports have demonstrated variations in transfusion practices for sickle cell disease (SCD) and thalassemia patients. Recently, NHLBI/TIF have provided transfusion recommendations for patients with haemoglobinopathies. METHODS: A cross-sectional survey was conducted of transfusion services from the state of Georgia previously identified as having SCD/thalassemia populations. The survey assessed transfusion service practices in pre-transfusion testing and blood product selection; awareness/implementation of NHLBI/TIF transfusion-based recommendations and perceived challenges in transfusing haemoglobinopathy patients. RESULTS: Responses were received from 35 of 49 (71%) institutions. Only institutions indicating transfusing SCD or thalassemia patients (32) were included in analysis. Seventy-one percent of non-sickle cell treatment centres (SCTCs) and 20% of non-thalassemia treatment centres follow NHLBI and TIF recommendations to perform a red blood cell phenotype beyond ABO/Rh(D) and provide Rh and Kell prophylactically matched units for SCD and thalassemia patients, respectively. Forty percent of institutions (33% of non-SCTCs) employ RBC genotyping to evaluate the red cell phenotype for SCD patients. Over 77% of institutions do not utilise a reliable method to identify SCD patients prior to transfusion, such as a required question/answer field on type/screen or crossmatch orders. CONCLUSION: Many healthcare systems' transfusion practices for haemoglobinopathy patients are discordant with NHLBI/TIF recommendations. Efforts are needed to increase awareness and implementation of current recommendations among all transfusion services seeing these patients.


Assuntos
Anemia Falciforme , Antígenos de Grupos Sanguíneos , Tipagem e Reações Cruzadas Sanguíneas , Transfusão de Sangue , Conhecimentos, Atitudes e Prática em Saúde , Talassemia , Anemia Falciforme/sangue , Anemia Falciforme/genética , Anemia Falciforme/terapia , Bancos de Sangue , Antígenos de Grupos Sanguíneos/sangue , Antígenos de Grupos Sanguíneos/genética , Estudos Transversais , Humanos , Guias de Prática Clínica como Assunto , Talassemia/sangue , Talassemia/genética , Talassemia/terapia
7.
Blood Cells Mol Dis ; 76: 53-58, 2019 05.
Artigo em Inglês | MEDLINE | ID: mdl-30792169

RESUMO

Thalassemia is a common inherited monogenic disease. It is characterized by chronic hemolysis, ineffective erythropoiesis (IE) and iron overload. Despite advances in transfusion practices and chelation therapy, still many limitations in delivering these standard therapies exist. Challenges of currently available standard care and advances in understanding the underlying pathophysiological mechanisms in thalassemia stimulated research towards development of novel therapeutic targets. Agents reducing IE as Jak 2 inhibitors and Activin II receptor traps are promising and are currently in clinical trials. Other approaches targeting iron dysregulation as mini-hepcidins, exogenous transferrin and erythroferrone inhibitors are in preclinical studies. Gene therapy, a rapidly evolving field, has exhibited remarkable progress in recent years. Studies have focused on ß or γ-globin addition, over expression of endogenous γ-globin-activating transcription factors, silencing of γ-globin repressors and genome editing of ß-globin mutations or γ-globin repressors. In this article we provide an overview of emerging recent trends in treatment of thalassemia targeting IE, iron dysregulation and novel curative treatments as gene therapy and gene editing.


Assuntos
Talassemia/terapia , Terapêutica/tendências , Eritropoese , Edição de Genes , Terapia Genética , Humanos , Sobrecarga de Ferro , Talassemia/complicações , Terapêutica/métodos
8.
Blood Transfus ; 17(1): 4-15, 2019 01.
Artigo em Inglês | MEDLINE | ID: mdl-30653458

RESUMO

BACKGROUND: Chronic red blood cell transfusion is the first-line treatment for severe forms of thalassaemia. This therapy is, however, hampered by a number of adverse effects, including red blood cell alloimmunisation. The aim of this systematic review was to collect the current literature data on erythrocyte alloimmunisation. MATERIALS AND METHODS: We performed a systematic search of the literature which identified 41 cohort studies involving 9,256 patients. RESULTS: The prevalence of erythrocyte alloimmunisation was 11.4% (95% CI: 9.3-13.9%) with a higher rate of alloimmunisation against antigens of the Rh (52.4%) and Kell (25.6%) systems. Overall, alloantibodies against antigens belonging to the Rh and Kell systems accounted for 78% of the cases. A higher prevalence of red blood cell alloimmunisation was found in patients with thalassaemia intermedia compared to that among patients with thalassaemia major (15.5 vs 12.8%). DISCUSSION: Matching transfusion-dependent thalassaemia patients and red blood cell units for Rh and Kell antigens should be able to reduce the risk of red blood cell alloimmunisation by about 80%.


Assuntos
Transfusão de Eritrócitos/efeitos adversos , Eritrócitos/imunologia , Isoimunização Rh , Sistema do Grupo Sanguíneo Rh-Hr/imunologia , Talassemia , Reação Transfusional , Humanos , Prevalência , Isoimunização Rh/epidemiologia , Isoimunização Rh/imunologia , Isoimunização Rh/prevenção & controle , Talassemia/epidemiologia , Talassemia/imunologia , Talassemia/terapia , Reação Transfusional/epidemiologia , Reação Transfusional/imunologia , Reação Transfusional/prevenção & controle
9.
Transfus Med ; 29(3): 179-184, 2019 Jun.
Artigo em Inglês | MEDLINE | ID: mdl-29691938

RESUMO

BACKGROUND AND OBJECTIVES: Recently, thalassemia has been introduced as a chronic disease. In spite of prolonging life in thalassemia patients, the quality of their life has not significantly improved. One of the challenges that makes their quality of life poor is alloimmunisation which causes several complications to patients by restricting their options. Some individuals are more susceptible to developing an alloantibody than others. They are categorised as responders and non-responders. Determining responders before the first transfusion allows transfusion services to provide compatible blood and prevent alloimmunisation. The aim of the present study was to determine the relationship between HLA-DRB1*15:03, HLA-DRB1*11 and HLA-DRB1*09:01 alleles and alloimmunisation in Iranian thalassemia patients. MATERIALS AND METHODS: Antibody screening tests were performed by tube method, and HLA-DRB1 genotyping was determined by Sequence-Specific Primers (SSP-PCR) in 59 alloimmunised and 205 non-alloimmunised patients. HLA-DRB1 allele frequencies were compared between alloantibody-positive and -negative groups through the χ2 test. RESULTS: HLA-DRB1*15:03 allele frequency was significantly different between groups (P = 0·000, odds ratio (OR) = 4·193). There was a correlation between HLA-DRB1*11 and anti-K (P = 0·000, OR = 6·643). There was no association between HLA-DRB1*09:01 and alloimmunisation (P = 0·350). CONCLUSIONS: According to our results, detecting HLA-DRB1*15:03 and HLA-DRB1*11 alleles are useful in the pre-transfusion test and could determine responder patients and improve transfusion safety.


Assuntos
Transfusão de Sangue , Cadeias HLA-DRB1 , Imunização , Isoanticorpos/imunologia , Qualidade de Vida , Talassemia , Reação Transfusional , Adulto , Alelos , Formação de Anticorpos , Feminino , Frequência do Gene , Cadeias HLA-DRB1/genética , Cadeias HLA-DRB1/imunologia , Humanos , Irã (Geográfico) , Masculino , Pessoa de Meia-Idade , Talassemia/genética , Talassemia/imunologia , Talassemia/terapia , Reação Transfusional/genética , Reação Transfusional/imunologia
10.
Hematology ; 24(1): 208-214, 2019 Dec.
Artigo em Inglês | MEDLINE | ID: mdl-30479186

RESUMO

The study was to determine the prevalence and clinical significances of red blood cell (RBC)-bound IgG as detected by flow cytometry in polytransfused patients with thalassemias. Relationship of the presence of RBC-bound IgG with RBC alloimmunization was also evaluated. This study included 59 polytransfused patients with ß-thalassemia disease. We studied the frequency of RBC autoantibodies and alloimmunization. Direct Coombs test and flow cytometry were performed to detect the presence of RBC autoantibodies while RBC alloantibodies were detected by antibody screening and identification assays. Eight (13.6%) and 34 (57.6%) patients were found a positive direct Coombs test and flow cytometry, respectively. Twenty (33.9%) patients developed RBC alloantibodies. The four most frequent RBC alloantibodies were anti-E (55%), anti-Mia (40%), anti-Di(a) (25%) and anti-c (15%), respectively. There was no significant difference in the presence of RBC-bound IgG between polytransfused with thalassemia patients who developed RBC alloimmunization (13 of 20; 65%) and those without RBC alloantibodies (21 of 39; 53.8%), p = 0.412. Splenectomy and increased transfusion requirement were significantly associated with the presence of RBC-bound IgG but not with RBC alloantibody formation. The overall frequency of RBC alloantibody formation in polytransfused patients with thalassemias was 33.9%. The most common RBC alloantibody was anti-E. RBC autoantibody formation was more frequently detected by flow cytometry (57.6%) than by direct Coombs test (13.6%). Splenectomy was significantly associated with the development of autoreactive RBC-bound IgG antibodies in the polytransfused patients with thalassemias. The presence of the anti-RBC autoantibodies may cause an increase of transfusion requirement.


Assuntos
Transfusão de Eritrócitos , Eritrócitos/imunologia , Imunoglobulina G/imunologia , Isoanticorpos/imunologia , Talassemia , Reação Transfusional , Adolescente , Adulto , Criança , Pré-Escolar , Feminino , Seguimentos , Humanos , Lactente , Masculino , Pessoa de Meia-Idade , Talassemia/epidemiologia , Talassemia/imunologia , Talassemia/terapia , Reação Transfusional/epidemiologia , Reação Transfusional/imunologia
11.
Transfusion ; 59(1): 177-184, 2019 01.
Artigo em Inglês | MEDLINE | ID: mdl-30443908

RESUMO

BACKGROUND: Hemoglobin E-ß0 thalassemia and homozygous ß0 -thalassemia are the most common chronic transfusion-dependent thalassemias in Thailand. Patients with these conditions can experience clinical complications such as RBC alloimmunization. In this study we aimed to determine the prevalence, alloimmunization risk factors, antigenic exposure, and evaluation of antigen- (C, c, E, e, Mia ) matched RBC transfusion. STUDY DESIGN AND METHODS: Thalassemia patients were recruited from a tertiary care hospital for 10 years from 2008 to 2017. The medical records of transfusion history were reviewed for red cell phenotype both of patients and donors, number of units transfused, and type of alloantibodies. RESULTS: A total of 383 thalassemia patients were identified (178 males and 205 females). The frequency of RBC alloantibodies was 19.3%. Some patients tested positive for more than one antibody type. Autoantibodies were detected in nine individuals. Anti-E (49 [39.5%]), anti-Mia (24 [19.4%]), and anti-c (19 [15.3%]) were the most common antibodies detected. A high rate of alloimmunization was found in splenectomized patients. Risk of alloimmunization increased when more total units of blood had been transfused. A trend toward low alloimmunization rates was noted in the antigen-matched RBC group, where 3.5% (5/143) of patients were alloimmunized. Anti-E and anti-Mia , which may be naturally occurring, were identified in this group. CONCLUSION: Thai patients are more prone to develop antibodies against the Rh and Mia than to the Kell blood group antigens. Provision of at least antigen-matched (C, c, E, e, Mia ) RBCs appears to improve the efficacy of transfusion in thalassemia patients.


Assuntos
Talassemia/imunologia , Talassemia/terapia , Adolescente , Adulto , Autoanticorpos/imunologia , Transfusão de Sangue , Criança , Pré-Escolar , Eritrócitos/imunologia , Feminino , Humanos , Lactente , Isoanticorpos/imunologia , Masculino , Pessoa de Meia-Idade , Prevalência , Fatores de Risco , Centros de Atenção Terciária/estatística & dados numéricos , Talassemia/epidemiologia , Adulto Jovem
13.
Eur J Gastroenterol Hepatol ; 31(2): 248-252, 2019 02.
Artigo em Inglês | MEDLINE | ID: mdl-30358571

RESUMO

BACKGROUND: Thalassemia is a group of inherited hematological disorders caused by mutation in globin's genes. Regular blood transfusion lengthens the life of thalassemia patients but it carries a definite risk of the infections of blood-borne diseases. AIM/OBJECTIVE: The current study was carried out for the frequency distributions and risk factors of hepatitis B virus (HBV) and hepatitis C virus (HCV) infections among thalassemia patients in Hazara regions, Pakistan. METHODS: A total of 324 enrolled thalassemia major patients were diagnosed in five different centers of Hazara regions. The study participants were screened for HBV and HCV using the immunochromatographic techniques test and real-time PCR for immunochromatographic technique-positive specimens. RESULTS: Out of the 324 major thalassemia patients, 24 (7.41%) were diagnosed with HBV and HCV infections. In total, 206 were male patients and the rate of HBV and HCV infections was 0.97% (two patients) and 3.88% (eight patients), respectively. Similarly, 118 were female patients and the rate of HBV-positive patients was 3.39% (four patients) and HCV was 8.47% (10 patients). The results also showed that 50% of HBV and HCV infections were found in the age group of 26-30 years, while 1.81% was found in the age group of 11-15 years. The positive HBV and HCV samples were also verified with the band size of 242 and 227 bp, respectively. CONCLUSION: Thus, to reduce the incidence of HBV and HCV in thalassemia patients, we must call for critical look on the transfusion practices as well as adoption of stricter donor selection.


Assuntos
Transfusão de Sangue , Hepatite B/epidemiologia , Hepatite C/epidemiologia , Talassemia/terapia , Reação Transfusional/epidemiologia , Adolescente , Adulto , Distribuição por Idade , Criança , Pré-Escolar , Feminino , Hepatite B/diagnóstico , Hepatite B/prevenção & controle , Hepatite B/transmissão , Hepatite C/diagnóstico , Hepatite C/prevenção & controle , Hepatite C/transmissão , Humanos , Incidência , Lactente , Masculino , Paquistão/epidemiologia , Fatores de Risco , Talassemia/diagnóstico , Talassemia/epidemiologia , Reação Transfusional/diagnóstico , Reação Transfusional/prevenção & controle , Adulto Jovem
14.
Eur J Haematol ; 102(2): 123-130, 2019 Feb.
Artigo em Inglês | MEDLINE | ID: mdl-30300449

RESUMO

OBJECTIVES: To evaluate the long-term efficacy and safety of deferasirox therapy in a large observational cohort of children with transfusion-dependent thalassemia (TDT) and sickle cell anemia (SCA) in Turkey. METHODS: This was a multicenter, prospective cohort study including TDT and SCA patients aged 2-18 years with iron overload (≥100 mL/kg of pRBC or a serum ferritin [SF] level >1000 µg/L) receiving deferasirox. Patients were followed for up to 3 years according to standard practice. RESULTS: A total of 439 patients were evaluated (415 [94.5%] TDT, 143 [32.6%] between 2 and 6 years). Serum ferritin levels consistently and significantly decreased across 3 years of deferasirox therapy from a median of 1775.5 to 1250.5 µg/L (P < 0.001). Serum ferritin decreases were noted in TDT (1804.9 to 1241 µg/L), SCA (1655.5 to 1260 µg/L), and across age groups of 2-6 years (1971.5 to 1499 µg/L), 7-12 years (1688.5 to 1159.8 µg/L), and 13-18 years (1496.5 to 1107 µg/L). Serum ferritin decreases were also noted for all deferasirox dose groups but only significant in patients with doses ≥30 mg/kg/d (n = 120, -579.6 median reduction, P < 0.001). Only 9 (2%) patients had adverse events suspected to be related to deferasirox. Serum creatinine slightly increased but remained within the normal range. CONCLUSIONS: Deferasirox has long-term efficacy and safety in children with TDT and SCA, although higher doses (≥30 mg/kg/d) may be required to achieve iron balance.


Assuntos
Anemia Falciforme/complicações , Deferasirox/uso terapêutico , Quelantes de Ferro/uso terapêutico , Sobrecarga de Ferro/tratamento farmacológico , Sobrecarga de Ferro/etiologia , Talassemia/complicações , Adolescente , Anemia Falciforme/terapia , Biomarcadores , Transfusão de Sangue , Criança , Pré-Escolar , Estudos de Coortes , Deferasirox/administração & dosagem , Deferasirox/efeitos adversos , Feminino , Ferritinas/sangue , Ferritinas/metabolismo , Humanos , Ferro/sangue , Ferro/metabolismo , Quelantes de Ferro/administração & dosagem , Quelantes de Ferro/efeitos adversos , Sobrecarga de Ferro/metabolismo , Masculino , Talassemia/terapia , Resultado do Tratamento , Turquia
15.
Intern Emerg Med ; 14(3): 365-370, 2019 04.
Artigo em Inglês | MEDLINE | ID: mdl-29948832

RESUMO

Heart disease remains a leading cause of morbidity and mortality in transfusion-dependent thalassemia (TDT), which can be attributed to several factors but primarily develops in the setting of iron overload. This was a retrospective cohort study utilizing Webthal® patient data from five major centers across Italy. Patients without heart disease were followed-up for 10 years (2000-2010) and data were collected for demographics, splenectomy status, serum ferritin and hemoglobin levels, and comorbidities associated with heart disease. Among 379 patients analyzed (mean age 22.9 ± 5.1 years, 47.8% men), 44 (cumulative incidence: 11.6%) developed heart disease during the period of observation. Splenectomy (p = 0.002) and serum ferritin level (p < 0.001) were the only risk factors with significant association with heart disease. A serum ferritin threshold of ≥ 3000 ng/mL was the best predictor for the development of heart disease (86.4% sensitivity and 92.8% specificity, AUC: 0.912, 95% CI 0.852-0.971, p < 0.001). On multivariate analysis, only a serum ferritin level ≥ 3000 ng/mL remained significantly and independently associated with increased risk of heart disease (HR: 44.85, 95% CI 18.85-106.74), with a 5- and 10-year heart disease-free survival of 58 and 39%. The association between iron overload and heart disease in patients with TDT is confirmed, yet a new serum ferritin level of 3000 ng/mL to flag increased risk is suggested.


Assuntos
Ferritinas/análise , Cardiopatias/complicações , Talassemia/complicações , Talassemia/terapia , Adolescente , Adulto , Área Sob a Curva , Transfusão de Sangue/métodos , Transfusão de Sangue/tendências , Distribuição de Qui-Quadrado , Criança , Estudos de Coortes , Feminino , Ferritinas/efeitos adversos , Ferritinas/sangue , Cardiopatias/fisiopatologia , Humanos , Itália , Masculino , Curva ROC , Estudos Retrospectivos , Fatores de Risco , Análise de Sobrevida , Talassemia/fisiopatologia
16.
J Pediatr Hematol Oncol ; 41(1): e47-e50, 2019 01.
Artigo em Inglês | MEDLINE | ID: mdl-30080754

RESUMO

An open-label, pilot study was conducted to evaluate deferasirox/deferiprone combination chelation therapy in adult patients with transfusion-dependent thalassemia and severe iron overload. Enrollment proved difficult. Nine patients (median age, 27.4 y; ferritin, 4965 ng/mL; liver iron concentration, 28.5 mg/g dry weight; cardiac T2*, 13.3 ms) received treatment. Two were withdrawn for treatment-related adverse effects. Arthralgia (4 patients) and gastrointestinal symptoms (5 patients) were common; no episodes of neutropenia/agranulocytosis occurred. Adherence difficulties were common. Of 6 patients with 12 to 18 months follow-up, 3 showed improvement in cardiac T2* and 2 in liver iron. Combination oral chelation may be effective but adverse effects and adherence challenges may limit efficacy.


Assuntos
Transfusão de Sangue , Deferasirox/administração & dosagem , Deferiprona/administração & dosagem , Sobrecarga de Ferro/tratamento farmacológico , Talassemia/terapia , Adulto , Deferasirox/efeitos adversos , Deferiprona/efeitos adversos , Quimioterapia Combinada , Feminino , Seguimentos , Humanos , Sobrecarga de Ferro/etiologia , Masculino , Projetos Piloto
18.
Hematology Am Soc Hematol Educ Program ; 2018(1): 361-370, 2018 11 30.
Artigo em Inglês | MEDLINE | ID: mdl-30504333

RESUMO

Transfusion combined with chelation therapy for severe ß thalassemia syndromes (transfusion-dependent thalassemia [TDT]) has been successful in extending life expectancy, decreasing comorbidities and improving quality of life. However, this puts lifelong demands not only on the patients but also on the health care systems that are tasked with delivering long-term treatment and comprehensive support. Prevention programs and curative approaches are therefore an important part of overall strategy. Curative treatments alter the dynamic of a patient's health care costs, from financial commitment over 50 years, into a potential "one-off" investment. Since the 1980s, this has usually been available only to the 30% or so of young children with matched sibling donors. By improving the safety of matched related donors and haploidentical hematopoietic stem cell transplants, the potential size of the donor pool for curative therapies may be increased. Recent advances in gene therapy demonstrate that even patients lacking a matched donor can be rendered transfusion independent with an autograft of genetically modified autologous stem cells, with a low short-term risk. Noncurative treatments are also of potential value by decreasing use of blood and chelators and decreasing hospital visits. An example is luspatercept, an activin-receptor trap that modifies transforming growth factor-ß signaling, thereby increasing the efficiency of erythropoiesis. This has entered phase 3 clinical trials for TDT and non-TDT and, usefully increases in both Hb and quality of life in non-TDT as well as decreasing transfusion requirements in TDT. Other novel noncurative treatments are entering clinical trials such improvement of erythropoiesis through pharmacological manipulation of hepcidin and iron metabolism.


Assuntos
Terapia Genética/métodos , Transplante de Células-Tronco/métodos , Talassemia/terapia , Doadores não Relacionados , Aloenxertos , Autoenxertos , Humanos , Talassemia/genética , Talassemia/metabolismo
19.
PLoS One ; 13(12): e0208102, 2018.
Artigo em Inglês | MEDLINE | ID: mdl-30507954

RESUMO

Chronic blood transfusions are responsible to cause iron overload, which leads to several complications to end organs and osteoporosis. Iron chelation is needed to remove iron excess and to contain bone-mass loss. Deferasirox is the most recent oral iron chelator that prevents transfusion related iron overload complications. Recently Eltrombopag (ELT) iron chelating properties are emerging. ELT is an agonist at Thrombopoietin receptor, used in treatment of thrombocytopenia. We tested ELT and Deferasirox in iron overloaded osteoclasts from thalassemic patients and donors measuring intracellular iron, TRAP expression and osteoclast activity. We confirmed ELT iron chelation capacity also in bone tissue and a synergic effect when used with Deferasirox. Moreover, having demonstrated its effects on osteoclast activity, we suggest for the first time that ELT could ameliorate bone tissue's health reducing bone mass loss.


Assuntos
Benzoatos/farmacologia , Hidrazinas/farmacologia , Quelantes de Ferro/farmacologia , Sobrecarga de Ferro/tratamento farmacológico , Osteoporose/prevenção & controle , Pirazóis/farmacologia , Talassemia/terapia , Reação Transfusional/tratamento farmacológico , Adulto , Benzoatos/uso terapêutico , Transfusão de Sangue , Osso e Ossos/efeitos dos fármacos , Osso e Ossos/metabolismo , Diferenciação Celular , Deferasirox/farmacologia , Deferasirox/uso terapêutico , Avaliação de Medicamentos , Feminino , Ferritinas/sangue , Voluntários Saudáveis , Humanos , Hidrazinas/uso terapêutico , Ferro/análise , Ferro/metabolismo , Quelantes de Ferro/uso terapêutico , Sobrecarga de Ferro/sangue , Sobrecarga de Ferro/complicações , Leucócitos Mononucleares , Osteoclastos/efeitos dos fármacos , Osteoclastos/fisiologia , Osteoporose/etiologia , Cultura Primária de Células , Pirazóis/uso terapêutico , Receptores de Trombopoetina/antagonistas & inibidores , Talassemia/complicações , Reação Transfusional/sangue , Reação Transfusional/complicações
20.
Complement Ther Clin Pract ; 33: 71-76, 2018 Nov.
Artigo em Inglês | MEDLINE | ID: mdl-30396630

RESUMO

BACKGROUND AND PURPOSE: This study investigated the disclosure of complementary and alternative medicine (CAM) use to health care providers by Malaysian thalassemia patients. METHODS: The semi-structured interviews were audio taped, transcribed verbatim, and translated into English. RESULTS: Thematic analysis identified four themes: 1) reason for CAM disclosure, 2) attempt to disclose CAM, 3) withdrawal from CAM disclosure, and 4) non-disclosure of CAM use. The reason for patients' disclosure of CAM use to healthcare providers is because they wanted to find information about CAM and were afraid of the interaction between the conventional medicine and CAM. Patients also disclosed the use of CAM because they were not satisfied with the conventional medicine that had caused them harm. CONCLUSION: Effective communication between patients and health care providers is important, especially for patients who are undergoing conventional thalassemia treatment, for fear that there is an interaction between conventional treatment and CAM use.


Assuntos
Terapias Complementares , Revelação , Pessoal de Saúde , Relações Médico-Paciente , Talassemia/terapia , Estudos de Coortes , Conhecimentos, Atitudes e Prática em Saúde , Humanos , Malásia
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