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Efficacy and risks in the use of human stem cells in the treatment of children with cerebral palsy

Cerebral palsy (CP), depending on disease severity, can result in diminished quality of life not only from decreased function but from societal stigmatization. Coordinating various care provider appointments, expense of short-term treatments, difficulty navigating transportation, and relying on caregivers can complicate patient and family lives. The costs of CP are substantial on the healthcare system, with one managed Medicaid database averaged across 15 U.S states finding the average annual Medicaid costs for children with CP to be 15 times higher than children without CP and averaged to $22, 383 United States dollar (USD) compared to $1,358 USD respectively (Pulgar et al., 2019). Cost effective treatment and effective prevention strategies are increasingly warranted for the CP population.
Due to the varying manifestations associated with CP, a standardized treatment for this condition is challenging. Current treatments may enhance quality of life and temporarily reduce pain or discomfort, but they do not cure CP. While perinatal prevention strategies potentially provide the greatest chance to prevent CP from occurring and should be the focus of health care policy, financial barriers remain especially with strained health care budgets. While cure remains elusive, focus on treatments and prevention strategies to limit disease impact is paramount.

In the last decade some attention has turned to the use of stem cell treatments in children and adolescents with CP to provide more impactful outcomes with earlier intervention potentially limiting the devastating musculoskeletal effects seen with severe disease. While primarily results from clinical trials both nationally and internationally suggest stem cell treatment increases gross motor function in children and adolescents, questions remain whether these treatments provide clinically meaningful improvement compared to traditional therapies.
The goal of this thesis is to discuss the current pharmaceuticals and non- pharmaceutical treatments with rehabilitative therapies that are historically used to reduce severity of secondary manifestations associated with CP in children. Human stem cell clinical trials for CP will be reviewed to assess efficacy and risks as this treatment is translated into clinical practice for children. In addition to stem cell treatment, public health practices of prevention during prenatal visits will be reviewed as it is an encouraging method to reduce preterm births which are a risk factor for CP development.

Identiferoai:union.ndltd.org:bu.edu/oai:open.bu.edu:2144/45578
Date04 February 2023
CreatorsJazrawi, Taylor
ContributorsWisco, Jonathan
Source SetsBoston University
Languageen_US
Detected LanguageEnglish
TypeThesis/Dissertation
RightsAttribution 4.0 International, http://creativecommons.org/licenses/by/4.0/

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