PMID- 33434161
OWN - NLM
STAT- MEDLINE
DCOM- 20210122
LR  - 20220419
IS  - 1479-683X (Electronic)
IS  - 0804-4643 (Print)
IS  - 0804-4643 (Linking)
VI  - 184
IP  - 2
DP  - 2021 Feb
TI  - Effectiveness and safety of rhIGF1 therapy in patients with or without Laron 
      syndrome.
PG  - 267-276
LID - EJE-20-0325 [pii]
LID - 10.1530/EJE-20-0325 [doi]
AB  - OBJECTIVE: The European Increlex(R) Growth Forum Database Registry monitors the 
      effectiveness and safety of recombinant human insulin-like growth factor-1 
      (rhIGF1; mecasermin, Increlex(R)) therapy in patients with severe primary IGF1 
      deficiency (SPIGFD). We present data from patients with and without a reported 
      genetic diagnosis of Laron syndrome (LS). DESIGN: Ongoing, open-label, 
      observational registry (NCT00903110). METHODS: Children and adolescents receiving 
      rhIGF1 therapy from 10 European countries were enrolled in 2008-2017 (n = 242). 
      The treatment-naive/prepubertal (NPP) cohort (n = 138) was divided into subgroups 
      based on reported genetic diagnosis of LS (n = 21) or non-LS (n = 117). 
      Multivariate analysis of the NPP-non-LS subgroup was conducted to identify 
      factors predictive of growth response (first-year-height standard deviation score 
      (SDS) gain >/= 0.3). Assessments included change in height and weight over 5 years 
      and adverse events (AEs). RESULTS: Height SDS gain from baseline was greater in 
      the NPP-LS than the NPP-non-LS subgroup after 1 years' treatment (P < 0.05). In 
      the NPP-non-LS subgroup, 56% were responders; young age at baseline was a 
      positive independent predictive factor (P < 0.001). NPP-non-LS-responders and the 
      NPP-LS subgroup had a similar mean age (6.07 years vs 7.00 years) at baseline and 
      height SDS gain in year 1 (0.64 vs 0.70), although NPP-non-LS-responders were 
      taller (P < 0.001) at baseline. BMI SDS changes did not differ across subgroups. 
      Treatment-emergent AEs were experienced by 65.3% of patients; hypoglycaemia was 
      most common. CONCLUSIONS: In most NPP children with SPIGFD, with or without LS, 
      rhIGF1 therapy promotes linear growth. The safety profile was consistent with 
      previous studies.
FAU - Bang, Peter
AU  - Bang P
AD  - Division of Pediatrics, Department of Biomedical and Clinical Sciences, Faculty 
      of Health Sciences, Linkoping University, Linkoping, Sweden.
FAU - Woelfle, Joachim
AU  - Woelfle J
AD  - Children's Hospital, University of Erlangen, Erlangen, Germany.
FAU - Perrot, Valerie
AU  - Perrot V
AD  - Ipsen Pharma, Boulogne-Billancourt, France.
FAU - Sert, Caroline
AU  - Sert C
AD  - Ipsen Pharma, Boulogne-Billancourt, France.
FAU - Polak, Michel
AU  - Polak M
AD  - Department of Paediatric Endocrinology, Gynaecology, and Diabetology, AP-HP, 
      Necker-Enfants Malades University Hospital, IMAGINE Institute, University of 
      Paris, Paris, France.
LA  - eng
SI  - ClinicalTrials.gov/NCT00903110
PT  - Journal Article
PT  - Observational Study
PL  - England
TA  - Eur J Endocrinol
JT  - European journal of endocrinology
JID - 9423848
RN  - 0 (Recombinant Proteins)
RN  - 67763-96-6 (Insulin-Like Growth Factor I)
RN  - 7GR9I2683O (mecasermin)
RN  - Insulin-Like Growth Factor I Deficiency
SB  - IM
MH  - Adolescent
MH  - Body Height
MH  - Body Weight/drug effects
MH  - Child
MH  - Female
MH  - Growth/drug effects
MH  - Growth Disorders/*drug therapy
MH  - Hearing Loss, Sensorineural/*drug therapy
MH  - Humans
MH  - Hypoglycemia/blood/chemically induced
MH  - Insulin-Like Growth Factor I/*deficiency/*therapeutic use
MH  - Laron Syndrome/*drug therapy/genetics
MH  - Longitudinal Studies
MH  - Male
MH  - Patient Safety
MH  - Puberty
MH  - Recombinant Proteins/*therapeutic use
MH  - Treatment Outcome
MH  - Young Adult
PMC - PMC7849377
EDAT- 2021/01/13 06:00
MHDA- 2021/01/23 06:00
PMCR- 2021/02/01
CRDT- 2021/01/12 17:19
PHST- 2020/04/17 00:00 [received]
PHST- 2020/11/17 00:00 [accepted]
PHST- 2021/01/13 06:00 [pubmed]
PHST- 2021/01/23 06:00 [medline]
PHST- 2021/01/12 17:19 [entrez]
PHST- 2021/02/01 00:00 [pmc-release]
AID - EJE-20-0325 [pii]
AID - 10.1530/EJE-20-0325 [doi]
PST - ppublish
SO  - Eur J Endocrinol. 2021 Feb;184(2):267-276. doi: 10.1530/EJE-20-0325.