Toward a paradigm shift from deficit-based to proactive speech and language treatment: Randomized pilot trial of the Babble Boot Camp in infants with classic galactosemia

Abstract

<ns4:p><ns4:bold>Background:</ns4:bold> Speech and language therapy is typically initiated reactively after a child shows delays. Infants with classic galactosemia (CG), a metabolic disease with a known high risk for both speech and language disorders, hold the keys towards evaluating whether preventive treatment is effective when the risks are known at birth. We present pilot data from a randomized parallel trial of an innovative proactive speech and language intervention program, the Babble Boot Camp (BBC). </ns4:p><ns4:p> <ns4:bold>Method</ns4:bold>: Five children with CG, otherwise healthy, participated in the study from approximately 2 to 24 months of age. One of these was randomly selected as control receiving conventional management, which typically starts at age 2-3 years. A pediatric speech-language pathologist met weekly via telepractice with the parents in the treatment cohort. Parents implemented the prespeech, speech, and language stimulation and expansion activities according to the intervention protocol. The control child was still too young for conventional treatment. Primary outcome measures were speech sound production complexity in babble and speech and expressive vocabulary size. Secondary outcome measures were vocalization rates and developmental milestones in communication, motor, and cognition. The trial is ongoing.</ns4:p><ns4:p> <ns4:bold>Results</ns4:bold>: All four treated children had higher speech sound skills in babble, three had higher speech sound skills in meaningful speech, two had higher expressive vocabularies, three had higher global developmental scores, and two had higher vocalization rates, compared to the control child with CG.</ns4:p><ns4:p> <ns4:bold>Discussion:</ns4:bold> Given the high risk for speech and language delays in children with CG, finding on-schedule abilities in two or more of the treated children but not the untreated child is unexpected under random conditions. The trends toward beneficial effects of the BBC on speech sound production, expressive language, and communication milestones warrant appropriately powered larger clinical trials with full randomization.</ns4:p><ns4:p> <ns4:bold>Trial registration: </ns4:bold>ClinicalTrials.gov <ns4:ext-link xmlns:ns3="http://www.w3.org/1999/xlink" ext-link-type="uri" ns3:href="https://clinicaltrials.gov/ct2/show/NCT03838016">NCT03838016</ns4:ext-link> (12 <ns4:sup>th</ns4:sup> February 2019).</ns4:p>

Tools for standardized data collection: Speech, Language, and Hearing measurement protocols in the PhenX Toolkit

“…Finally, a more comprehensive understanding of genotype-phenotype associations in communication disorders in general has the potential for personalized and proactive clinical translations based on the principles of precision medicine. An example of such a translation is the Babble Boot Camp©, a package of activities and routines currently undergoing clinical trial, designed to prevent or mitigate severe speech and language disorders in infants with predicable genetic risk, in this case a newborn diagnosis of classic galactosemia (Peter et al in, press;Peter, Potter et al, 2019).…”

Galactosemia: Biochemistry, Molecular Genetics, Newborn Screening, and Treatment

“…BBC is a preventive speech intervention program constating active parental involvement guided by a speech specialist. A trend between speech outcomes has been observed in four children with CG receiving BBC as compared to one child with CG who did not receive BBC, supporting the potential benefit of this approach [ 80 ].…”

Screenless Teletherapy and Silent Telesupervision: Leveraging Technology for Innovative Service Delivery and Clinician Training in Speech-language Pathology during the COVID-19 Era

“…Typical in-person speech-language pathology sessions are 30-60 minutes, once to twice per week. Shorter and more frequent sessions-which would not be feasible with in-person service delivery-are easily accomplished using telepractice (e.g., 10-minute parent coaching provided multiple times per week 78 ). There is some support for cost savings associated with early intervention delivered via teletechnology compared to in-person formats, 67,79 however systematic cost analysis with more diverse populations and broader demographic regions is needed.…”

“…In terms of client perceptions and experiences using teletechnology, some stakeholders may believe that services delivered via teletechnology are substandard to in-person care. [72][73][74][75][76][77][78][79][80] Buy-in is a necessary component of any intervention. Given the widespread use of teletechnology due to the COVID-19 pandemic, it is possible that attitudes toward effectiveness of teletherapy will continue to improve with increased familiarity and use.…”