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Global developmental delay (GDD) is a subset of developmental disabilities defined as significant delay in two or more of the following developmental domains: gross/fine motor, speech/language, cognition, social/personal, and activities of daily living [1]. Approximately 10 % of children who have a GDD or mental retardation have an underlying cytogenetic abnormality [2]. Despite a variety of treatment options for GDD by the multidisciplinary team including psychologist, occupational therapist, speech therapist, and social worker [3], we cannot usually change the natural course of disease, whether it takes a deteriorating course or a gradually resolving one. Umbilical cord blood transplantation (UCBT) have been attempted in several mice studies for treatment of various neurologic conditions such as ischemic [4], and hemorrhage stroke [5], traumatic brain injury [6], and Alzheimer disease [7]. Moreover, UCBT for children with inherited metabolic disorders, such as Hurler syndrome and Krabbe disease had an effect in prevention of neurological deterioration, and even caused cognitive improvement [8, 9]. Erythropoietin (EPO), a glycoprotein hormone that controls erythropoiesis, also has neuroprotective characteristics reported in several studies using animal models for stroke [10] and traumatic brain injury [11]. Neuroregenerative and angiogenic effects of EPO in injured brain tissues have also been reported [12]. As a neurotrophic factor, EPO served as an adjunctive option for combination therapy to potentiate the efficacy of cell therapy [13]. Recently, our research team reported on the safety and efficacy of allogenic UCBT with concurrent EPO injection in children with cerebral palsy in a randomized, placebo-controlled trial [14]. We also tried to apply the therapeutic strategy to a GDD child with expectation of favorable neurotrophic effect on brain function development. Since the result showed noticeable efficacy, we report on the combination therapy of allogenic UCBT with concurrent EPO injection for a child with GDD who had chromosomal abnormality.

Allogenic umbilical cord blood transplantation for a child with chromosomal abnormality: a case report