Pathophysiology And Management Of Sickle Cell Disease: New Therapeutic Approaches And Disease-Modifying Treatments

Abstract

Background: The genetic disorder sickle cell disease results in the production of abnormal HbS and causes changes in red blood cell shape, alongside vaso-occlusion, breakdown, and continuous inflammation. People who have SCD experience severe health problems and death risks because of medical conditions such as pain emergencies together with strokes, and organ tissue deterioration. The treatment landscape now focuses on altering disease advancement patterns while enhancing patient quality of care.

Objectives: Evaluating innovative SCD therapeutic and disease-modifying treatments requires measuring their effects on clinical aspects, as well as VOC frequency and red blood cell haemoglobin levels in SCD patients.

Study design: A prospective cohort study.

Place and duration of study. Department of Microbiology Nowshera Medical College Nowsher,Kpk-Pakistan From jan 2019 to jan 2020

Methods: 50 SCD patients for twelve months by analyzing their standard treatments in combination with hydroxyurea, voxelotor, crizanlizumab and gene therapy protocols. Scientists monitored SCD patients throughout 12 months through assessments of haemoglobin measurements and VOC incidents, along with tests of biomarkers linked to hemolysis. Mean comparison tests, along with standard deviation (SD) measurements, allowed researchers to verify significant results through p-values representing the determined parameters. Doctors verified that patients aged 12 to 50 years old with confirmed SCD would be included in the study. The research enrolled 50 patients (mean age: 26.4 ± 6.2 years) and excluded individuals with various blood diseases. The voxelotor group experienced a significant elevation in their haemoglobin levels, as indicated by p=0.03, whereas therapeutic effects became visible through decreased VOC counts in crizanlizumab recipients (p=0.02). The sustained expression of HbF as a result of gene therapy reduced the markers indicating hemolysis. Hydroxyurea treatment led to better blood test results; however, it produced inconsistent results regarding the decrease in VOC events.

Conclusion: Available disease-modifying drugs show the potential to lower the morbidity related to SCD. Crizanlizumab succeeds in minimising VOC occurrences, while voxelotor stabilises haemoglobin structures, and gene therapy presents a possible cure for the disease. Longitudinal research must continue to prove both the long-term effectiveness and security rates authentically. SDC management could see revolutionary changes through the incorporation of these therapies into clinical settings.