JMIR Publications

ABSTRACT

Background:

Sickle Cell Disease (SCD) is a severe, inherited hemoglobin disorder characterized by chronic hemolysis, vaso-occlusive crises (VOCs), and systemic inflammation. Hydroxyurea is the standard conventional pharmacotherapy for SCD, but it has certain limitations, necessitating the need to explore other safe and effective treatment options for SCD. Ayurveda interventions offer a potential therapeutic approach complementary to conventional medicine for SCD management with anti-inflammatory, immunomodulator, and hematopoietic properties.

Objective:

This RCT will evaluate the efficacy and safety of an Ayurveda therapeutic regimen as an adjunct to Hydroxyurea in SCD management, assessing its impact on hematological parameters, inflammatory biomarkers, VOC frequency, and overall quality of life.

Methods:

A prospective, randomized, open-label, blinded endpoint (PROBE) study will be conducted on individuals of any gender aged 18 years or above and diagnosed with SCD (with HbS levels more than 60% and a history of at least one VOC per year over the past three years). Individuals with acute VOC or any severe infection requiring hospitalization, a history of significant co-morbidities, or hematopoietic stem cell transplantation will not be considered. The study will be conducted at All India Institute of Medical Sciences, Bhopal, India. A total of 100 participants will undergo random assignment in a 1:1 ratio to receive either an Ayurveda regimen (Dadimadi Ghrita, Punarnavadi Mandura, and Vasaguduchyadi Kwatha) as an add-on to Hydroxyurea, or Hydroxyurea alone for eight months. The primary outcome will be a change in hemoglobin electrophoresis parameters (HbS, HbF, HbA) and frequency of VOC episodes over eight months. The secondary outcome measures include the change in the levels of pro-inflammatory markers (IL-6, IL-8, CRP, TGF-β) and lactate dehydrogenase, frequency of hospitalization for VOCs and blood transfusions, and health-related quality of life (SF-8 questionnaire). The safety will be evaluated by recording the incidence of adverse events and changes in liver and kidney function tests from baseline.

Results:

The recruitment of study participants initiated on November 1, 2023. Till the second week of February 2025, 83 participants have been enrolled in the study.

Conclusions:

This RCT protocol outlines a rigorous study design aimed to explore the potential benefits of integrated therapeutic regimen comprising of Ayurveda interventions and standard conventional care in the long-term management of SCD through validated clinical and laboratory parameters. The outcomes of this study can address the needs and challenges associated with SCD management, and inform future management protocols. Clinical Trial: Clinical Trial Registry of India - CTRI/2023/03/051032