Thalassemia Major and Sickle Cell Disease in Adolescents and Young Adults

Yacobovich, Joanne; Tamary, Hannah

doi:10.1159/000360235

Cited by 13 publications

(12 citation statements)

References 75 publications

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“…On the other hand, all forms of treatment can be inconvenient, time-consuming, and result in unpleasant side effects, all which could potentially impact physical and emotional functioning of patients. [ 2 3 6 9 13 14 30 34 ] In our study, the DFX-treated pediatric and adult patients with BTM or SCA were heavy iron overloaded in agreement with previous reports. [ 20 26 35 36 37 38 39 40 41 42 ] The lack of treatment may explain the high ferritin levels.…”

Section: Discussionsupporting

confidence: 92%

Quality of life, clinical effectiveness, and satisfaction in patients with beta thalassemia major and sickle cell anemia receiving deferasirox chelation therapy

Şenol

Tiftik

Ünal

et al. 2016

J Basic Clin Pharma

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Objectives:There is a need to remove excess iron with iron chelation therapy (ICT) to avoid the serious clinical sequelae associated with iron overload in patients with beta thalassemia major (BTM) and sickle cell anemia (SCA). Due to the effects of the diseases and their treatments, ICT is still a major reason for unsatisfactory compliance. The aim of this single-center observational study was to evaluate the quality of life, clinical effectiveness, and satisfaction in pediatric and adult patients with BTM and SCA receiving deferasirox (DFX) chelation therapy.Methods:In this study, 37 pediatric and 35 adult patients with BTM or SCA receiving DFX for at least 6 months participated. Upon receipt of Informed Consent Form, Case Report Form, Demographic Data Collection Form, Child Health Questionnaire-Parent Form, Life Quality Survey Short Form-36, and ICT Satisfaction Survey were used to obtain data for the effectiveness of ICT and parameters that may affect compliance to treatment and life quality of the participants.Results:As a main index for the effectiveness of DFX chelation therapy, serum ferritin levels were higher than the normal values in the patients receiving DFX. The increased ferritin levels were also associated with hematological and biochemical abnormalities. Our findings regarding quality of life and satisfaction with DFX chelation therapy indicated that the patients with BTM or SCA had lower scores. Overall, problems with treatment regimen and side effects appeared to be common causes of poor compliance to DFX chelation therapy.Conclusions:Our findings suggest that health care providers should be aware of the importance of monitoring iron load with timely initiation of DFX chelation therapy and ongoing adjustments to chelation regimens and/or transfusion methods to decrease hospitalizations and improve compliance to ICT of the patients with BTM and SCA.

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Section: Discussionsupporting

confidence: 92%

Quality of life, clinical effectiveness, and satisfaction in patients with beta thalassemia major and sickle cell anemia receiving deferasirox chelation therapy

Şenol

Tiftik

Ünal

et al. 2016

J Basic Clin Pharma

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“…Thalassemia patients had lower BMI due to many factors such as growth hormone deficiency, hypothyroidism, hypogonadism, low Hb levels, bone disorders and desferrioxamine toxicity. 45,46 This result corresponds to prior study by Estrella-Castillo et all that showed subjects with lower weight have poorer cognitive compared to normal population. 47 Study by Joo et all concluded that subjects that have lower BMI has progressive risk to have Alzheimer's compared to normal 13 while Suemoto et all showed that decrease BMI leads to decreased of memory function within 12 years.…”

Section: Discussionsupporting

confidence: 90%

Cognitive Impairment Detection in Adult Thalassemia Patient Using Moca-Ina

Wardoyo

Gamayani

Ong

et al. 2021

MNJ

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Background: Cognitive impairment in thalassemia patients are prevalent, therefore early detection of cognitive impairment in adult thalassemia patients is crucial for prevention. Montreal Cognitive Assessment (MoCA) is a public domain cognition screening tools that covers all cognitive domains in detecting mild cognitive impairments. Objective: To compare cognitive function between adult thalassemia patients and healty control by using Indonesia version of MoCA test (MoCA-Ina) Methods: This prospective observational analytic with case control study, compared the total scores and scores of each domain of cognition between adult thalassemia patients and healthy subjects at the Medical Hematology Oncology Clinic of Dr. Hasan Sadikin General Hospital, Bandung, Indonesia using MoCA-Ina from August to October 2018. Results: A total of 32 thalassemia subjects and 50 healthy subjects were conducted. A total of 16(50%) subjects in the case group had a value of MoCA-Ina <26, while only 1(2%) healthy control had a value of MoCA-Ina <26. The median total MoCA-Ina score in case and control groups were 25.5 and 27.50 (p <0.001). The median score of memory domains, executive functions and visuospatial of the case and control groups were 3 versus 4 (p <0.001), 3 versus 3.5 (p <0.001) and 3.53 ± 0.671 versus 3.88 ± 0.385 (p <0.003), respectively. Conclusion: Adults thalassemia patients have lower score in total MoCA-Ina, domains of memory, executive function and visuospatial score compared to healthy control.

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“…In this study, sickle cell patients with HbSS genotypes had their menarche late at 16 years of age. Puberty delay is usually observed in SCD and is proportional to the severity of the disease 18. The delivery was programmed at the 37th week; this attitude is the one reported by many authors 19,20.…”

Section: Discussionmentioning

confidence: 85%

Homozygous Deletion Alfa-Thalassemia and Hereditary Persistence of Fetal Hemoglobin, Two Genetic Factors Predictive the Reduction of Morbidity and Mortality During Pregnancy in Sickle Cell Patients . A Report From Democratic Republic of Congo

Mikobi

Lukusa

Muamba

et al. 2019

Mediterr J Hematol Infect Dis

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FHb and alpha-thal are two genetic factors that modulate the clinical expression of sickle cell disease. Objective: to determine the beneficial role of FHb and alpha-thal on fetal and maternal morbidity during pregnancy in sickle cell patients. This is a documentary and analytical study that included 960 deliveries of homozygous sickle cell patients. The deliveries were divided into three genotype subgroups: Hb-SS / alpha-thal, HbSS / HPFH and HbSS. The diagnosis of SCD and the quantification of FHb were performed by the capillary electrophoresis technique. The diagnosis of SCD has been confirmed by the molecular test. The diagnosis of alpha-thal was made by the multiplex ligation dependent probe amplification (MLPA) technique. Sickle cell pregnancies were followed according to the protocol of care in force in our service. The variables of interest were: hematological variables, sickle cell crises during pregnancy, maternal and fetal complications. Statistical analyzes were performed with SPSS 20.0 software. Means and standard deviations were compared with the Student's t and Annova tests. The value of p <0.05 was considered the significance level. The Hb-SS / alpha-thal and HbSS / HPFH genotypes were observed in 101 and 121 women respectively. Otherwise 758 women had the HbSS genotype. The morbidity related to sickle cell complications in the mother and fetus, were less frequent in the Hb-SS / alpha-thal and HbSS / HPFH groups. The statistical differences were statistically significant. This study showed a significant protective effect of alpa-thal and HPFH during pregnancy in sickle-cell pregnant women. FHb and alpha-thal are two genetic factors that modulate the clinical expression of sickle cell disease. Objective: to determine the beneficial role of FHb and alpha-thal on fetal and maternal morbidity during pregnancy in sickle cell patients. This is a documentary and analytical study that included 960 deliveries of homozygous sickle cell patients. The deliveries were divided into three genotype subgroups: Hb-SS / alpha-thal, HbSS / HPFH and HbSS. The diagnosis of SCD and the quantification of FHb were performed by the capillary electrophoresis technique. The diagnosis of SCD has been confirmed by the molecular test. The diagnosis of alpha-thal was made by the multiplex ligation dependent probe amplification (MLPA) technique. Sickle cell pregnancies were followed according to the protocol of care in force in our service. The variables of interest were: hematological variables, sickle cell crises during pregnancy, maternal and fetal complications. Statistical analyzes were performed with SPSS 20.0 software. Means and standard deviations were compared with the Student's t and Annova tests. The value of p <0.05 was considered the significance level. The Hb-SS / alpha-thal and HbSS / HPFH genotypes were observed in 101 and 121 women respectively. Otherwise 758 women had the HbSS genotype. The morbidity related to sickle cell complications in the mother and fetus, were less frequent in the Hb-SS / alpha-thal and HbSS / HPFH groups. The statistical differences were statistically significant. This study showed a significant protective effect of alpa-thal and HPFH during pregnancy in sickle-cell pregnant women.

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Thalassemia Major and Sickle Cell Disease in Adolescents and Young Adults

Cited by 13 publications

References 75 publications

Quality of life, clinical effectiveness, and satisfaction in patients with beta thalassemia major and sickle cell anemia receiving deferasirox chelation therapy

Quality of life, clinical effectiveness, and satisfaction in patients with beta thalassemia major and sickle cell anemia receiving deferasirox chelation therapy

Cognitive Impairment Detection in Adult Thalassemia Patient Using Moca-Ina

Homozygous Deletion Alfa-Thalassemia and Hereditary Persistence of Fetal Hemoglobin, Two Genetic Factors Predictive the Reduction of Morbidity and Mortality During Pregnancy in Sickle Cell Patients . A Report From Democratic Republic of Congo

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