Contributions
Abstract: PB2186
Type: Publication Only
Background
Autism Spectrum Disorders (ASD) are severe heterogeneous neurodevelopmental abnormalities characterized by dysfunctions in social interactions and communication skills, restricted interests, repetitive, and stereotypic verbal and non-verbal behaviors. The etiology of ASD remains unknown, but recent studies suggest a possible association with altered immune responses and ASD. Inflammation in the brain and Central Nervous System has been reported with microglia activation and increased cytokine production in postmortem brain specimens of individuals with ASD. Other studies have established a correlation between ASD and family history of autoimmune diseases, associations with MHC complex haplotypes, and abnormal levels of various inflammatory cytokines and immunological markers in the blood. The paracrine, secretome, and immunomodulatory effects of stem cells would appear to be the likely mechanisms of application for ASD therapeutics.
Aims
Evaluation the benefits of HSCT in patients with ASD.
Methods
We describe two cases of patients with ASD who underwent HSCT for acute lymphoblastic leukemia (ALL) and whose symptoms were markedly decreased like an improvement of social interaction, communication, and behaviors.
Results
The first patient is an 11-year-old girl with ASD who was diagnosis with Ph-positive ALL in October 2011 (at the end of treatment, BCR-ABL remained positive). She underwent a matched sibling HSCT in March 2015. The conditioning regimen was total body irradiation (TBI) and cyclophosphamide. During the 20-month follow-up period, we observed improvement in social interaction, communication, and behaviours. According to The Childhood Autism Rating Scale – CARS, prior to HSCT, she had a score of 39 (Severe Symptoms of ASDisorder), and she currently scores 30 (Mild-to-Moderate Symptoms of ASD).
The second case is a 7-year-old boy with ASD, Asperger Syndrome, who was diagnosis with ALL in September 2012. He presented with bone marrow and testicular relapse in May 2015 and underwent a matched unrelated HSCT in November 2015. The conditioning regimen was Etoposide, ATG and TBI. During the 12-month follow-up period, we observed improvement in social interaction, communication, and behaviours. According to CARS, prior to HSCT he had a score of 30 (Mild-to-Moderate Symptoms of ASD), and he currently scores 24 (Minimal-to-No Symptoms of ASD).
There is no treatment for ASD thus every effort to minimize the symptoms are valuable. In both cases, social interaction was significantly increased, and the aggressive behaviors decreased. Clinical cases have reported responses in autistic children receiving cord blood CD34+ cells.
Conclusion
Several incurable neurological disorders have shown benefits with cellular therapy. Thus, autism should be explored as an indication. Clinical studies are an immediate need to fully explore its potential in autism.
Session topic: 22. Stem cell transplantation - Clinical
Abstract: PB2186
Type: Publication Only
Background
Autism Spectrum Disorders (ASD) are severe heterogeneous neurodevelopmental abnormalities characterized by dysfunctions in social interactions and communication skills, restricted interests, repetitive, and stereotypic verbal and non-verbal behaviors. The etiology of ASD remains unknown, but recent studies suggest a possible association with altered immune responses and ASD. Inflammation in the brain and Central Nervous System has been reported with microglia activation and increased cytokine production in postmortem brain specimens of individuals with ASD. Other studies have established a correlation between ASD and family history of autoimmune diseases, associations with MHC complex haplotypes, and abnormal levels of various inflammatory cytokines and immunological markers in the blood. The paracrine, secretome, and immunomodulatory effects of stem cells would appear to be the likely mechanisms of application for ASD therapeutics.
Aims
Evaluation the benefits of HSCT in patients with ASD.
Methods
We describe two cases of patients with ASD who underwent HSCT for acute lymphoblastic leukemia (ALL) and whose symptoms were markedly decreased like an improvement of social interaction, communication, and behaviors.
Results
The first patient is an 11-year-old girl with ASD who was diagnosis with Ph-positive ALL in October 2011 (at the end of treatment, BCR-ABL remained positive). She underwent a matched sibling HSCT in March 2015. The conditioning regimen was total body irradiation (TBI) and cyclophosphamide. During the 20-month follow-up period, we observed improvement in social interaction, communication, and behaviours. According to The Childhood Autism Rating Scale – CARS, prior to HSCT, she had a score of 39 (Severe Symptoms of ASDisorder), and she currently scores 30 (Mild-to-Moderate Symptoms of ASD).
The second case is a 7-year-old boy with ASD, Asperger Syndrome, who was diagnosis with ALL in September 2012. He presented with bone marrow and testicular relapse in May 2015 and underwent a matched unrelated HSCT in November 2015. The conditioning regimen was Etoposide, ATG and TBI. During the 12-month follow-up period, we observed improvement in social interaction, communication, and behaviours. According to CARS, prior to HSCT he had a score of 30 (Mild-to-Moderate Symptoms of ASD), and he currently scores 24 (Minimal-to-No Symptoms of ASD).
There is no treatment for ASD thus every effort to minimize the symptoms are valuable. In both cases, social interaction was significantly increased, and the aggressive behaviors decreased. Clinical cases have reported responses in autistic children receiving cord blood CD34+ cells.
Conclusion
Several incurable neurological disorders have shown benefits with cellular therapy. Thus, autism should be explored as an indication. Clinical studies are an immediate need to fully explore its potential in autism.
Session topic: 22. Stem cell transplantation - Clinical