Institutional review boards and ethics committees have seen it as their main task in the past few decades to protect children from questionable studies; however, they are faced with a new challenge with pediatric drug development.
Systemic sclerosis, also known as scleroderma, is a rare and complex autoimmune connective-tissue
disease. Once considered an untreatable and unpredictable condition, research advancements
have improved our understanding of its disease pathogenesis and clinical phenotypes
and expanded our treatment armamentarium. Early and accurate diagnosis is essential,
while ongoing efforts to risk stratify patients have a central role in predicting
both organ involvement and disease progression. A holistic approach is required when
choosing the optimal therapeutic strategy, balancing the side-effect profile with
efficacy and tailoring the treatment according to the goals of care of the patient.