Sickle Cell Disease: Monitoring, Current Treatment, and Therapeutics Under Development

Carolyn Hoppe1, Lynne Neumayr1

  • 1Department of Pediatrics, Division of Hematology/Oncology, UCSF Benioff Children's Hospital Oakland, 747 52nd Street, Oakland, CA 94609, USA.

Insights

Updated clinical care for sickle cell disease in children includes early screening for organ injury and expanded use of red cell transfusion and hydroxyurea. This review covers current standards, monitoring, and emerging treatments.

Area of Science:

  • Pediatrics
  • Hematology
  • Genetics

Background:

  • Sickle cell disease (SCD) significantly impacts children's health, necessitating optimized clinical management.
  • Advances in screening and therapeutics have evolved the standard of care for pediatric SCD.
  • Early detection of organ damage is crucial for improving outcomes in children with SCD.

Purpose of the Study:

  • To review current best practices for managing sickle cell disease in children.
  • To outline screening recommendations, monitoring strategies, and treatment approaches for pediatric SCD.
  • To discuss novel pharmacologic agents currently in clinical trials for SCD.

Main Methods:

  • Literature review of current screening protocols for organ injury in pediatric SCD.
  • Analysis of expanded indications for red cell transfusion and hydroxyurea therapy.
  • Review of ongoing clinical trials for new SCD pharmacologic agents.

Main Results:

  • Current standards emphasize comprehensive screening and early intervention for organ damage.
  • Hydroxyurea and red cell transfusions are integral components of pediatric SCD management.
  • Several novel agents show promise in ongoing clinical investigations.

Conclusions:

  • The standard of care for pediatric sickle cell disease has advanced with improved screening and therapeutic options.
  • Continuous monitoring and tailored treatment are essential for managing SCD in children.
  • Emerging pharmacologic therapies offer potential for future treatment advancements in SCD.

Related Concept Videos

Therapeutic Drug Monitoring: Affecting Factors01:29

Therapeutic Drug Monitoring: Affecting Factors

Therapeutic Drug Monitoring (TDM) is the clinical practice of measuring specific drug levels in a patient's blood or body tissues to manage and optimize therapy. TDM is crucial for drugs with narrow therapeutic windows, like warfarin and phenytoin, where incorrect doses can lead to treatment failure or severe side effects. This monitoring ensures the dosage administered is within a safe and effective range. The factors affecting therapeutic drug monitoring include:Patient-Specific Factors:a.
211
Parkinson's Disease: Treatment01:24

Parkinson's Disease: Treatment

Neurodegenerative disorders, such as Parkinson's Disease (PD), involve the gradual and irreversible destruction of neurons in particular brain areas. These disorders exhibit standard features like proteinopathies, selective vulnerability of some neurons, and an interaction of intrinsic properties, genetics, and environmental influences in neural injury.
Parkinson's Disease is primarily a result of the loss of dopaminergic neurons in the substantia nigra pars compacta. The cornerstone of...
1.0K
Alzheimer's Disease: Treatment01:22

Alzheimer's Disease: Treatment

Alzheimer's Disease (AD), a neurodegenerative disorder, is pathologically identified by amyloid plaques and neurofibrillary tangles composed of tau protein. AD pharmacotherapy aims to manage cognitive symptoms, delay disease progression, and treat behavioral symptoms. The treatment is primarily symptomatic and palliative, with no definitive disease-modifying therapy available. Cholinesterase inhibitors, including donepezil (Aricept), rivastigmine (Exelon), and galantamine (Razadyne), are...
850
Therapeutic Drug Monitoring: Overview and Classification01:16

Therapeutic Drug Monitoring: Overview and Classification

Therapeutic Drug Monitoring (TDM) is a clinical practice that measures specific drug levels in a patient's blood at designated intervals to ensure the drug concentration stays within a therapeutic range. This monitoring is crucial for optimizing individual dosage regimens, enhancing therapeutic efficacy, and minimizing drug-related toxicity. TDM is vital for drugs with narrow therapeutic windows, significant variability in pharmacokinetics, and a clear correlation between plasma levels and...
326
Therapeutic Drug Monitoring: Drug Analysis Methods01:26

Therapeutic Drug Monitoring: Drug Analysis Methods

Therapeutic Drug Monitoring (TDM) is a clinical practice that measures specific drug levels in a patient's blood or body tissues to tailor drug therapy effectively. This monitoring is critical for managing drugs with narrow therapeutic indices like digoxin and phenytoin, ensuring they are both safe and effective. For instance, monitoring theophylline levels in asthma patients involves precision and sensitivity to adjust doses according to individual responses to therapy, ensuring efficacy and...
187
Drugs for Treatment of Crohn's Disease in IBD Using Glucocorticoids01:21

Drugs for Treatment of Crohn's Disease in IBD Using Glucocorticoids

Glucocorticoids, a class of anti-inflammatory drugs, are pivotal in treating moderate to severe Crohn's disease by inducing remission. They exhibit their anti-inflammatory action by inhibiting the production of inflammatory cytokines such as tumor necrosis factor (TNF)-α, interleukin (IL)-1, and chemokines like IL-8. In addition, they reduce the expression of inflammatory cell adhesion molecules and inhibit gene transcription of nitric oxide synthase, phospholipase A2, cyclooxygenase-2...
462