Five-Year Therapy with Recombinant Human Insulin-Like Growth Factor-1 in a Patient with Pregnancy-associated Plasma Protein A2 (PAPP-A2) Deficiency
Authors' conclusion
Therapy with rhIGF-1 modestly improved linear growth in one patient with PAPP-A2 deficiency, but without true catch-up. Therapy was associated with pseudotumor cerebri in a sibling. Initial improvement in BMD and glycemic pattern on rhIGF-1 was not sustained during puberty.
Study context
- Population
- Three siblings with PAPP-A2 deficiency
- Participants
- 3 siblings
- Outcome
- Growth on rhIGF-1 (P3)
- Follow-up
- 5 years
Main finding
As reported in the abstract
Height velocity at baseline
3.0 cm/year
Height velocity thereafter
5.0-7.6 cm/year
Change in height SDS
+0.6
Pubertal onset
12.4 year
Observational data: associations, not effects.
Further findings
Bone mineral density (BMD) height-adjusted Z-score (P3)
- Lumbar spine
- +0.4
- Forearm
- −0.2
- Hip
- −0.3
Glucose metabolism
mg/dL
- P2 worsening glucose tolerance, 2-h glucose
- 225
- P3 IFG and hyperinsulinemia initially resolved; Impaired glucose tolerance (IGT) developed during puberty, 2-h glucose
- 152
- All siblings had hyperinsulinemia
- IGT resolved in P1
Safety
Discontinuation
- P2 discontinued therapy due to pseudotumor cerebri
Parts of this Brief are withheld: the automatic checks could not confirm them against the abstract.
Original publication
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