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Effect of recombinant insulin-like growth factor-1 treatment on short-term linear growth in a child with Majewski osteodysplastic primordial dwarfism type II and hepatic insufficiency.

Abstract

We report the case of a boy affected by severe intrauterine and postnatal growth retardation, microcephaly, facial dysmorphisms and postnecrotic cirrhosis, diagnosed at birth as having Seckel syndrome, and subsequently confirmed as Majewski osteodysplastic primordial dwarfism type II (MOPD II) on the basis of clinical and radiological features of skeletal dysplasia. At our observation (6 years 7 months) he presented height -10.3 standard deviation score (SDS), weight -22.1 SDS, head circumference -8 SDS, delayed bone age of 4 years with respect to chronological age. In consideration of the low levels of insulin-like growth factor-1 (IGF-1) as well as of hepatic insufficiency, we started the treatment with recombinant human IGF-1 (rhIGF-1) at the dose of 0.04 mg/kg in 2 doses/day, with an increase of 0.04 mg/kg after 1 week until the maximum dose of 0.12 mg/kg. We observed an early response to rhIGF-1 treatment, with a shift of height velocity from 1.8 cm/year (-4.6 SDS) at 4 cm/year (-1.9 SDS), and an increase in bone age of 1.5 years during the first 6 months. rhIGF-1 treatment does not seem to be able to replace the physiological action of IGF-1 in patients with MOPD II and hepatic insufficiency, however, it seems to preserve the typical growth pattern of MOPD II patients, avoiding a further widening of the growth deficiency in these subjects.

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    MeSH

    Body Height
    Body Weight
    Child
    Dwarfism
    Fetal Growth Retardation
    Hepatic Insufficiency
    Humans
    Insulin-Like Growth Factor I
    Male
    Microcephaly
    Osteochondrodysplasias
    Recombinant Proteins

    Pub Type(s)

    Journal Article

    Language

    eng

    PubMed ID

    23612698

    Citation

    Faienza, Maria Felicia, et al. "Effect of Recombinant Insulin-like Growth Factor-1 Treatment On Short-term Linear Growth in a Child With Majewski Osteodysplastic Primordial Dwarfism Type II and Hepatic Insufficiency." Journal of Pediatric Endocrinology & Metabolism : JPEM, vol. 26, no. 7-8, 2013, pp. 771-4.
    Faienza MF, Acquafredda A, D'Aniello M, et al. Effect of recombinant insulin-like growth factor-1 treatment on short-term linear growth in a child with Majewski osteodysplastic primordial dwarfism type II and hepatic insufficiency. J Pediatr Endocrinol Metab. 2013;26(7-8):771-4.
    Faienza, M. F., Acquafredda, A., D'Aniello, M., Soldano, L., Marzano, F., Ventura, A., & Cavallo, L. (2013). Effect of recombinant insulin-like growth factor-1 treatment on short-term linear growth in a child with Majewski osteodysplastic primordial dwarfism type II and hepatic insufficiency. Journal of Pediatric Endocrinology & Metabolism : JPEM, 26(7-8), pp. 771-4. doi:10.1515/jpem-2012-0397.
    Faienza MF, et al. Effect of Recombinant Insulin-like Growth Factor-1 Treatment On Short-term Linear Growth in a Child With Majewski Osteodysplastic Primordial Dwarfism Type II and Hepatic Insufficiency. J Pediatr Endocrinol Metab. 2013;26(7-8):771-4. PubMed PMID: 23612698.
    * Article titles in AMA citation format should be in sentence-case
    TY - JOUR T1 - Effect of recombinant insulin-like growth factor-1 treatment on short-term linear growth in a child with Majewski osteodysplastic primordial dwarfism type II and hepatic insufficiency. AU - Faienza,Maria Felicia, AU - Acquafredda,Angelo, AU - D'Aniello,Mariangela, AU - Soldano,Lucia, AU - Marzano,Flaviana, AU - Ventura,Annamaria, AU - Cavallo,Luciano, PY - 2012/12/10/received PY - 2013/02/18/accepted PY - 2013/4/25/entrez PY - 2013/4/25/pubmed PY - 2013/12/16/medline SP - 771 EP - 4 JF - Journal of pediatric endocrinology & metabolism : JPEM JO - J. Pediatr. Endocrinol. Metab. VL - 26 IS - 7-8 N2 - We report the case of a boy affected by severe intrauterine and postnatal growth retardation, microcephaly, facial dysmorphisms and postnecrotic cirrhosis, diagnosed at birth as having Seckel syndrome, and subsequently confirmed as Majewski osteodysplastic primordial dwarfism type II (MOPD II) on the basis of clinical and radiological features of skeletal dysplasia. At our observation (6 years 7 months) he presented height -10.3 standard deviation score (SDS), weight -22.1 SDS, head circumference -8 SDS, delayed bone age of 4 years with respect to chronological age. In consideration of the low levels of insulin-like growth factor-1 (IGF-1) as well as of hepatic insufficiency, we started the treatment with recombinant human IGF-1 (rhIGF-1) at the dose of 0.04 mg/kg in 2 doses/day, with an increase of 0.04 mg/kg after 1 week until the maximum dose of 0.12 mg/kg. We observed an early response to rhIGF-1 treatment, with a shift of height velocity from 1.8 cm/year (-4.6 SDS) at 4 cm/year (-1.9 SDS), and an increase in bone age of 1.5 years during the first 6 months. rhIGF-1 treatment does not seem to be able to replace the physiological action of IGF-1 in patients with MOPD II and hepatic insufficiency, however, it seems to preserve the typical growth pattern of MOPD II patients, avoiding a further widening of the growth deficiency in these subjects. SN - 0334-018X UR - https://www.unboundmedicine.com/medline/citation/23612698/Effect_of_recombinant_insulin_like_growth_factor_1_treatment_on_short_term_linear_growth_in_a_child_with_Majewski_osteodysplastic_primordial_dwarfism_type_II_and_hepatic_insufficiency_ L2 - https://www.degruyter.com/doi/10.1515/jpem-2012-0397 DB - PRIME DP - Unbound Medicine ER -