Longitudinal effect of disease‐modifying therapy on left ventricular diastolic function in children with sickle cell anemia

Rai, Parul; Okhomina, Victoria; Kang, Guolian; Martinez, Hugo R.; Hankins, Jane S.; Joshi, Vijaya M.

doi:10.1002/ajh.26911

Cited by 4 publications

(2 citation statements)

References 52 publications

Supporting

Mentioning

Contrasting

Order By: Relevance

“…A major side effect of chronic RBC transfusions is the build-up of excess, toxic deposits of iron in various organs, which results in tissue damage and organ malfunction [ 6 , 7 , 8 ]. Congestive cardiac failure is the main cause of mortality in heavily iron-loaded beta thalassemia major (TM) patients who have no access to chelation therapy for the removal of excess iron [ 9 ].…”

Section: Introductionmentioning

confidence: 99%

See 1 more Smart Citation

Iron Load Toxicity in Medicine: From Molecular and Cellular Aspects to Clinical Implications

Kontoghiorghes

2023

IJMS

View full text Add to dashboard Cite

Iron is essential for all organisms and cells. Diseases of iron imbalance affect billions of patients, including those with iron overload and other forms of iron toxicity. Excess iron load is an adverse prognostic factor for all diseases and can cause serious organ damage and fatalities following chronic red blood cell transfusions in patients of many conditions, including hemoglobinopathies, myelodyspasia, and hematopoietic stem cell transplantation. Similar toxicity of excess body iron load but at a slower rate of disease progression is found in idiopathic haemochromatosis patients. Excess iron deposition in different regions of the brain with suspected toxicity has been identified by MRI T2* and similar methods in many neurodegenerative diseases, including Alzheimer’s disease and Parkinson’s disease. Based on its role as the major biological catalyst of free radical reactions and the Fenton reaction, iron has also been implicated in all diseases associated with free radical pathology and tissue damage. Furthermore, the recent discovery of ferroptosis, which is a cell death program based on free radical generation by iron and cell membrane lipid oxidation, sparked thousands of investigations and the association of iron with cardiac, kidney, liver, and many other diseases, including cancer and infections. The toxicity implications of iron in a labile, non-protein bound form and its complexes with dietary molecules such as vitamin C and drugs such as doxorubicin and other xenobiotic molecules in relation to carcinogenesis and other forms of toxicity are also discussed. In each case and form of iron toxicity, the mechanistic insights, diagnostic criteria, and molecular interactions are essential for the design of new and effective therapeutic interventions and of future targeted therapeutic strategies. In particular, this approach has been successful for the treatment of most iron loading conditions and especially for the transition of thalassemia from a fatal to a chronic disease due to new therapeutic protocols resulting in the complete elimination of iron overload and of iron toxicity.

show abstract

Section: Introductionmentioning

confidence: 99%

“…A major side effect of chronic RBC transfusions is the build-up of excess, toxic deposits of iron in various organs, which results in tissue damage and organ malfunction [6][7][8].…”

Section: Introductionmentioning

confidence: 99%

Iron Load Toxicity in Medicine: From Molecular and Cellular Aspects to Clinical Implications

Kontoghiorghes

2023

IJMS

View full text Add to dashboard Cite

show abstract

Regression Approaches to Assess Effect of Treatments That Arrest Progression of Symptoms

Ortega‐Villa,

Nason,

Fay

et al. 2024

Statistics in Medicine

View full text Add to dashboard Cite

Motivated by a small sample example in neonatal onset multisystem inflammatory disease (NOMID), we propose a method that can be used when the interest is testing for an association between a changes in disease progression with start of treatment compared to historical disease progression prior to treatment. Our method estimates the longitudinal trajectory of the outcome variable and adds an interaction term between an intervention indicator variable and the time since initiation of the intervention. This method is appropriate for a situation in which the intervention slows or arrests the effect of the disease on the outcome, as is the case in our motivating example. By simulation in small samples and restricted sets of treatment initiation times, we show that the generalized estimating equations (GEE) formulation with small sample adjustments can bound the Type I error rate better than GEE and linear mixed models without small sample adjustments. Permutation tests (permuting the time of treatment initiation) is another valid approach that can also be useful. We illustrate the methodology through an application to a prospective cohort of NOMID patients enrolled at the NIH clinical center.

show abstract

Biventricular systolic and diastolic function in adolescents with sickle cell anemia and sickle cell traits – Systematic review

Lemos,

Ribeiro,

de Andrade Martins

2023

Progress in Pediatric Cardiology

View full text Add to dashboard Cite

Longitudinal effect of disease‐modifying therapy on left ventricular diastolic function in children with sickle cell anemia

Cited by 4 publications

References 52 publications

Iron Load Toxicity in Medicine: From Molecular and Cellular Aspects to Clinical Implications

Iron Load Toxicity in Medicine: From Molecular and Cellular Aspects to Clinical Implications

Regression Approaches to Assess Effect of Treatments That Arrest Progression of Symptoms

Biventricular systolic and diastolic function in adolescents with sickle cell anemia and sickle cell traits – Systematic review

Contact Info

Product

Resources

About