Treatment of severe primary IGF-1 deficiency using rhIGF-1 preparation – first three years of Polish experience

Petriczko, Elżbieta; Jackowski, Tomasz; Horodnicka‐Józwa, Anita; Wikiera, Beata; Noczyńska, Anna; Korpal-Szczyrska, Maria; Birkholz-Walerzak, Dorota; Małecka-Tendera, Ewa; Kalina-Fraska, Barbara; Kalina, Maria; Barg, Ewa; Beń‐Skowronek, Iwona; Szewczyk, Leszek; Hilczer, Maciej; Smyczyńska, Joanna; Stawerska, Renata; Lewiński, Andrzej; Ziora, Katarzyna; Bossowski, Artur; Pietrewicz, Edyta; Pyrżak, Beata; Kędzia, Andrzej W.; Szalecki, Mieczysław; Kilian, Agnieszka; Walczak, Mieczysław

doi:10.5603/ep.a2018.0074

Cited by 10 publications

(12 citation statements)

References 9 publications

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“…In Poland, only children with GHI confirmed by IGF-GT have been qualified to rIGF-I therapy. Very recently, Petriczko et al [34] have published the data on FH of the first 27 patients with severe primary IGFD who completed treatment and attained FH. The average increase of hSDS in them was 1.45.…”

Section: Discussionmentioning

confidence: 99%

Significance of Direct Confirmation of Growth Hormone Insensitivity for the Diagnosis of Primary IGF-I Deficiency

Smyczyńska

Hilczer

et al. 2020

JCM

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Primary insulin-like growth factor-I (IGF-I) deficiency is a synonym of growth hormone (GH) insensitivity (GHI), however the necessity of direct confirmation of GH resistance by IGF-I generation test (IGF-GT) is discussed. GHI may disturb intrauterine growth, nevertheless short children born small for gestational age (SGA) are treated with GH. We tested the hypothesis that children with appropriate birth size (AGA), height standard deviation score (SDS) <−3.0, GH peak in stimulation tests (stimGH) ≥10.0 µg/L, IGF-I <2.5 centile, and excluded GHI may benefit during GH therapy. The analysis comprised 21 AGA children compared with 6 SGA and 20 GH-deficient ones, with height SDS and IGF-I as in the studied group. All patients were treated with GH up to final height (FH). Height velocity, IGF-I, and IGF binding protein-3 (IGFBP-3) concentrations before and during first year of treatment were assessed. Effectiveness of therapy was better in GHD than in IGF-I deficiency (IGFD), with no significant difference between SGA and AGA groups. All but two AGA children responded well to GH. Pretreatment IGF-I and increase of height velocity (HV) during therapy but not the result of IGF-GT correlated with FH. As most AGA children with apparent severe IGFD benefit during GH therapy, direct confirmation of GHI seems necessary to diagnose true primary IGFD in them.

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Section: Discussionmentioning

confidence: 99%

Significance of Direct Confirmation of Growth Hormone Insensitivity for the Diagnosis of Primary IGF-I Deficiency

Smyczyńska

Hilczer

et al. 2020

JCM

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“…This section includes 2 additional relevant studies (4 reports) [20][21][22]24 included in the sponsor's submission to CADTH. These were considered to address important gaps in the evidence included in the systematic review.…”

Section: Other Relevant Evidencementioning

confidence: 99%

“…One report of 1 single-arm trial 24 was included. The report presents data on the growth outcomes and harms observed in the first 3 years of treatment with mecasermin in patients with SPIGFD from Poland.…”

Section: Polish Study On Increlexmentioning

confidence: 99%

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Mecasermin (Increlex)

Team¹

2022

cjht

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CADTH reimbursement reviews are comprehensive assessments of the clinical effectiveness and cost-effectiveness, as well as patient and clinician perspectives, of a drug or drug class. The assessments inform non-binding recommendations that help guide the reimbursement decisions of Canada's federal, provincial, and territorial governments, with the exception of Quebec. This review assesses mecasermin (Increlex), 10 mg/mL (40 mg/4mL vial), sterile solution for subcutaneous injection. Indication: Treatment of growth failure in children and adolescents from 2 to 18 years with confirmed SPIGFD. SPIGFD is defined by: height standard deviation score ≤ −3.0 basal IGF-1 levels below the 2.5th percentile for age and gender growth hormone sufficiency exclusion of secondary forms of IGF-1 deficiency, such as malnutrition, hypopituitarism, hypothyroidism, or chronic treatment with pharmacologic doses of anti-inflammatory steroids SPIGFD includes patients with mutations in the GHR gene/Laron’s syndrome, post-GHR signalling pathway, and IGF-1 gene defects.

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Alterations in Stem Cell Populations in IGF-1 Deficient Pediatric Patients Subjected to Mecasermin (Increlex) Treatment

Grubczak

Stożek

Starosz

et al. 2022

Stem Cell Rev and Rep

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Pathway involving insulin-like growth factor 1 (IGF-1) plays significant role in growth and development. Crucial role of IGF-1 was discovered inter alia through studies involving deficient patients with short stature, including Laron syndrome individuals. Noteworthy, despite disturbances in proper growth, elevated values for selected stem cell populations were found in IGF-1 deficient patients. Therefore, here we focused on investigating role of these cells—very small embryonic-like (VSEL) and hematopoietic stem cells (HSC), in the pathology. For the first time we performed long-term observation of these populations in response to rhIGF-1 (mecasermin) therapy. Enrolled pediatric subjects with IGF-1 deficiency syndrome were monitored for 4–5 years of rhIGF-1 treatment. Selected stem cells were analyzed in peripheral blood flow cytometrically, together with chemoattractant SDF-1 using immunoenzymatic method. Patients’ data were collected for correlation of experimental results with clinical outcome. IGF-1 deficient patients were found to demonstrate initially higher levels of VSEL and HSC compared to healthy controls, with their gradual decrease in response to therapy. These changes were significantly associated with SDF-1 plasma levels. Correlations of VSEL and HSC were also reported in reference to growth-related parameters, and IGF-1 and IGFBP3 values. Noteworthy, rhIGF-1 was shown to efficiently induce development of Laron patients achieving at least proper rate of growth (compared to healthy group) in 80% of subjects. In conclusion, here we provided novel insight into stem cells participation in IGF-1 deficiency in patients. Thus, we demonstrated basis for future studies in context of stem cells and IGF-1 role in growth disturbances. Graphical abstract

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Treatment of severe primary IGF-1 deficiency using rhIGF-1 preparation – first three years of Polish experience

Cited by 10 publications

References 9 publications

Significance of Direct Confirmation of Growth Hormone Insensitivity for the Diagnosis of Primary IGF-I Deficiency

Significance of Direct Confirmation of Growth Hormone Insensitivity for the Diagnosis of Primary IGF-I Deficiency

Mecasermin (Increlex)

Alterations in Stem Cell Populations in IGF-1 Deficient Pediatric Patients Subjected to Mecasermin (Increlex) Treatment

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