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

Background: Speech or language therapy is typically initiated reactively after a child starts showing delays. Infants with classic galactosemia (CG), an inborn error of metabolism 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). Method: Five children with CG, otherwise healthy, participated in the BBC from approximately 2 to 24 months of age. One of these was randomly selected as control receiving conventional management. 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 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 developmental milestones in communication, motor, and cognition. Outcomes in the treatment cohort were compared to typical children and the control child and to typically developing children represented in test norms and cases described in the literature . The trial is ongoing. Results: 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, and three had higher communication and personal-social skills, compared to the control child with CG. Discussion: 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. Trial registration: ClinicalTrials.gov NCT03838016 (12th February 2019).

Translating principles of precision medicine into speech-language pathology: Clinical trial of a proactive speech and language intervention for infants with classic galactosemia

“…As previously described, 72 , 73 progress is monitored closely throughout the intervention phase (<6 to 24 months) in multiple ways. Once monthly, parents create a day-long audio recording using the Language Environment Analysis (LENA) recording system (LENA Research Foundation, Boulder, CO).…”

“…Disordered language is seen in 50%–78% of children with CG, 65 , 66 , 68 compared with 7%–10% of children generally. 10 Regarding language development, comprehension is affected less by CG than expression 72 , 73 ; mainly, children with CG who have cognitive delays tend to also have delays in language comprehension. 65 Even at the earliest stages, there are some red flags for difficulties with speech and language: many children with CG do not meet cooing and babbling milestones, 74 , 75 , 76 and they are late in producing their first words.…”

Current and Future Treatments for Classic Galactosemia

“…This approach focuses on a preventive intervention program starting at 2 to 4 months of age and consisting of active parental involvement guided by an expert speech language pathologist. Although the results are preliminary and generalizations cannot be made, a trend to beneficial effects on speech and language development was shown [146].…”

“…The other interesting approach is the Babble Boot Camp (BBC), which is focused on a proactive speech and language intervention program [146]. Current guidelines recommend regular extensive screening for speech and language delay, if children do not meet appropriate levels of speech or language treatment is initiated preferably during the first year of life [111].…”

“…Current guidelines recommend regular extensive screening for speech and language delay, if children do not meet appropriate levels of speech or language treatment is initiated preferably during the first year of life [111]. However, since speech and language can be regarded as later-developing skill, deficits and signs of delay will only become apparent after an age greater than 24 and 36 months, respectively [111,146]. This approach focuses on a preventive intervention program starting at 2 to 4 months of age and consisting of active parental involvement guided by an expert speech language pathologist.…”