Response to Treatment with Recombinant Human Growth Hormone (rhGH) of Short Stature Children Born Too Small for Gestational Age (SGA) in Selected Centres in Poland
Abstract
:1. Introduction
2. Materials and Methods
2.1. Study Design
2.2. Population
2.3. Data Collection
2.4. Data Analysis
2.5. Statistical Analysis
2.6. Ethical and Legal Considerations
3. Results
3.1. Response to Treatment in the Whole Study Group
3.2. Response to Treatment in Patients with Uncharacteristic Dysmorphic Features
3.3. Response to Treatment in Patients with FAS
3.4. Response to Treatment in Patients with SRS
3.5. Comparison of Criteria for Evaluating Response to Treatment
3.6. rhGH Dose
3.7. IGF-1 Concentration
3.8. Bone Age
3.9. Type of Hypotrophy
4. Discussion
Limitations of Work
5. Conclusions
Author Contributions
Funding
Institutional Review Board Statement
Informed Consent Statement
Data Availability Statement
Acknowledgments
Conflicts of Interest
References
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Characteristic | All Mean (SD) | Uncharacteristic Features of Dysmorphia Mean (SD) | FAS Mean (SD) | SRS Mean (SD) |
---|---|---|---|---|
n | 235 | 31 | 15 | 9 |
Gender | ||||
F | 98 | 13 | 6 | 4 |
M | 137 | 18 | 9 | 5 |
Age at onset of treatment [months] | 109 (35) | 100 (35) | 110 (29) | 84 (30) |
Tanner stage at onset of treatment | ||||
1 | 190 | 27 | 11 | 8 |
2 | 32 | 3 | 4 | 1 |
3 | 10 | 0 | 0 | 0 |
4 | 3 | 1 | 0 | 0 |
Mother’s height, SDS | −1.02 (1.26) | −0.41 (1.84) | −0.53 (0.41) | −0.89 (0.78) |
Father’s height, SDS | −0.98 (1.22) | −0.75 (1.17) | −0.68 (1.40) | −0.84 (1.25) |
MPH | 166 (13) | 169 (11) | 170.6 (5.1) | 168.8 (6.0) |
Birth weight, SDS | −3.24 (1.16) | −3.22 (1.12) | −4.24 (1.48) | −4.43 (1.31) |
Birth length, SDS | −0.98 (1.54) | −0.96 (1.40) | −1.77 (1.51) | −2.28 (2.34) |
GH0 dose [mg/kg/d] | 0.031(0.005) | 0.032 (0.006) | 0.029 (0.005) | 0.031 (0.004) |
GH12 dose [mg/kg/d] | 0.033 (0.005) | 0.034 (0.006) | 0.032 (0.006) | 0.033 (0.004) |
GH24 dose [mg/kg/d] | 0.030 (0.004) | 0.032 (0.004) | 0.032 (0.004) | 0.034 (0.003) |
Ht0, SDS | −3.05 (0.79) | −3.24 (0.86) | −3.00 (0.51) | −4.33 (1.59) |
HV before treatment [cm/year] | 5.19 (1.45) | 5.11 (1.31) | 4.46 (1.07) | 5.27 (1.85) |
BMI0 | 14.73 (2.05) | 14.7 (3.3) | 13.95 (1.39) | 13.03 (0.85) |
BMI12 | 15.36 (2.39) | 15.1 (3.5) | 14.67 (1.56) | 13.71 (1.62) |
BMI24 | 15.71 (2.55) | 15.45 (3.32) | 14.31 (1.49) | 14.89 (2.24) |
Characteristic | All Mean (SD) | Uncharacteristic Features of Dysmorphia Mean (SD) | FAS Mean (SD) | SRS Mean (SD) |
---|---|---|---|---|
Ht0 SDS | −3.05 (0.79) | −3.24 (0.86) | −3.00 (0.51) | −4.33 (1.59) |
Ht12 SDS | −2.48 (0.78) | −2.70 (0.89) | −2.58 (0.58) | −3.60 (1.56) |
Ht24 SDS | −2.19 (0.85) | −2.39 (0.97) | −2.12 (0.56) | −3.32 (1.54) |
ΔHt SDS 12-0 | 0.57 (0.33) | 0.54 (0.43) | 0.43 (0.31) | 0.74 (0.22) |
ΔHt SDS 24-12 | 0.38 (0.33) | 0.35 (0.36) | 0.36 (0.14) | 0.72 (0.21) |
ΔHt SDS 24-0 | 0.96 (0.49) | 1.00 (0.54) | 0.86 (0.19) | 1.48 (0.37) |
HV0 [cm/year] | 5.19 (1.45) | 5.11 (1.31) | 4.46 (1.07) | 5.27 (1.85) |
HV12 [cm/year] | 8.15 (1.75) | 8.05 (1.87) | 7.52 (1.62) | 7.92 (1.54) |
HV24 [cm/year] | 7.17 (1.74) | 7.09 (2.17) | 7.03 (1.13) | 8.20 (1.45) |
After 12 Months | |||
Group | N | ΔHt SDS < 0.3 | ΔHt SDS ≥ 0.3 |
All | 235 (100%) | 39 (17%) | 196 (83%) |
Uncharacteristic features of dysmorphia | 31 (100%) | 5 (16%) | 26 (84%) |
FAS | 15 (100%) | 5 (33%) | 10 (67%) |
SRS | 9 (100%) | 0 (0%) | 9 (100%) |
After 24 Months | |||
Group | N | ΔHt SDS < 0.3 | ΔHt SDS ≥ 0.3 |
all | 151 (100%) | 66 (44%) | 85 (56%) |
Uncharacteristic features of dysmorphia | 19 (100%) | 7 (37%) | 12 (63%) |
FAS | 7 (100%) | 3 (43%) | 4 (57%) |
SRS | 7 (100%) | 0 (0%) | 7 (100%) |
After 12 Months | |||
Group | N | ΔHV < 3 cm/year | ΔHV ≥ 3 cm/year |
all | 218 (100%) | 123 (56%) | 95 (44%) |
Uncharacteristic features of dysmorphia | 29 (100%) | 17 (59%) | 12 (41%) |
FAS | 13 (100%) | 7 (54%) | 6 (46%) |
SRS | 8 (100%) | 5 (62%) | 3 (38%) |
After 24 Months | |||
Group | N | ΔHV < 3 cm/year | ΔHV ≥ 3 cm/year |
all | 136 (100%) | 99 (73%) | 37 (27%) |
Uncharacteristic features of dysmorphia | 18 (100%) | 11 (61%) | 7 (39%) |
FAS | 5 (100%) | 3 (60%) | 2 (40%) |
SRS | 6 (100%) | 4 (67%) | 2 (33%) |
After 12 Months | |||
Tanner Stage | N | ΔHt SDS < 0.3 | ΔHt SDS ≥ 0.3 |
1 | 190 (100%) | 28 (15%) | 162 (85%) |
2 | 32 (100%) | 7 (22%) | 25 (78%) |
3 | 10 (100%) | 2 (20%) | 8 (80%) |
4 | 3 (100%) | 2 (67%) | 1 (33%) |
After 24 Months | |||
Tanner Stage | N | ΔHt SDS < 0.3 | ΔHt SDS ≥ 0.3 |
1 | 132 (100%) | 54 (41%) | 78 (59%) |
2 | 14 (100%) | 10 (71%) | 4 (29%) |
3 | 5 (100%) | 2 (40%) | 3 (60%) |
After 12 Months | |||
Tanner Stage | N | ΔHV < 3 cm/year | ΔHV ≥ 3 cm/year |
1 | 174 (100%) | 103 (59%) | 71 (41%) |
2 | 31 (100%) | 10 (32%) | 21 (68%) |
3 | 10 (100%) | 8 (80%) | 2 (20%) |
4 | 3 (100%) | 2 (67%) | 1 (33%) |
After 24 Months | |||
Tanner Stage | N | ΔHV < 3 cm/year | ΔHV ≥ 3 cm/year |
1 | 117 (100%) | 84 (72%) | 33 (28%) |
2 | 14 (100%) | 10 (71%) | 4 (29%) |
3 | 5 (100%) | 5 (100%) | 0 (0%) |
After 12 Months | |||
Group | N | ΔHt SDS < 0.3 or ΔHV < 3 cm/year | ΔHt SDS < 0.3 and ΔHV < 3 cm/year |
All | 218 (100%) | 126 (58%) | 34 (16%) |
Uncharacteristic features of dysmorphia | 29 (100%) | 18 (62%) | 4 (14%) |
FAS | 13 (100%) | 8 (62%) | 3 (23%) |
SRS | 8 (100%) | 5 (62%) | 0 (0%) |
After 24 Months | |||
Group | N | ΔHt SDS < 0.3 or ΔHV < 3 cm/year | ΔHt SDS < 0.3 and ΔHV < 3 cm/year |
All | 136 (100%) | 105 (77%) | 54 (40%) |
Uncharacteristic features of dysmorphia | 18 (100%) | 11 (61%) | 6 (40%) |
FAS | 5 (100%) | 3 (60%) | 2 (40%) |
SRS | 6 (100%) | 4 (67%) | 0 (0%) |
Characteristic | All Mean (SD) | Uncharacteristic Features of Dysmorphia Mean (SD) | FAS Mean (SD) | SRS Mean (SD) |
---|---|---|---|---|
IGF-1 Percentile at Baseline | ||||
<3 percentile | 14 | 4 | 0 | 1 |
3–97 percentile | 219 | 26 | 14 | 8 |
>97 percentile | 0 | 0 | 0 | 0 |
IGF-1 percentile after 12 months | ||||
<3 percentile | 1 | 0 | 0 | 0 |
3–97 percentile | 222 | 30 | 14 | 9 |
>97 percentile | 8 | 1 | 1 | 0 |
IGF-1 percentile after 24 months | ||||
<3 percentile | 1 | 0 | 0 | 0 |
3–97 percentile | 140 | 17 | 7 | 6 |
>97 percentile | 7 | 1 | 0 | 1 |
Characteristic | All Mean (SD) | Uncharacteristic Features of Dysmorphia Mean (SD) | FAS Mean (SD) | SRS Mean (SD) |
---|---|---|---|---|
n | 235 | 31 | 15 | 9 |
BA [months] | ||||
Baseline | 84 (39) | 75 (41) | 86 (33) | 54 (37) |
After 12 months | 101 (39) | 92 (41) | 107 (27) | 72 (37) |
After 24 months | 112 (37) | 101 (42) | 98 (16) | 74 (20) |
CA-BA [months] | ||||
Baseline | 25 (15) | 25 (17) | 24 (14) | 30 (14) |
After 12 months | 19 (15) | 20 (18) | 15 (16) | 24 (12) |
After 24 months | 16 (16) | 18 (18) | 14 (16) | 20 (17) |
BA/CA ratio | ||||
Baseline | 0.75 (0.16) | 0.72 (0.20) | 0.77 (0.15) | 0.60 (0.20) |
After 12 months | 0.83 (0.15) | 0.80 (0.18) | 0.89 (0.13) | 0.72 (0.17) |
After 24 months | 0.86 (0.14) | 0.84 (0.19) | 0.88 (0.13) | 0.78 (0.20) |
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Glińska, M.; Walczak, M.; Wikiera, B.; Pyrżak, B.; Majcher, A.; Paluchowska, M.; Gawlik, A.; Antosz, A.; Kusz, M.; Bossowski, A.; et al. Response to Treatment with Recombinant Human Growth Hormone (rhGH) of Short Stature Children Born Too Small for Gestational Age (SGA) in Selected Centres in Poland. J. Clin. Med. 2022, 11, 3096. https://doi.org/10.3390/jcm11113096
Glińska M, Walczak M, Wikiera B, Pyrżak B, Majcher A, Paluchowska M, Gawlik A, Antosz A, Kusz M, Bossowski A, et al. Response to Treatment with Recombinant Human Growth Hormone (rhGH) of Short Stature Children Born Too Small for Gestational Age (SGA) in Selected Centres in Poland. Journal of Clinical Medicine. 2022; 11(11):3096. https://doi.org/10.3390/jcm11113096
Chicago/Turabian StyleGlińska, Marta, Mieczysław Walczak, Beata Wikiera, Beata Pyrżak, Anna Majcher, Monika Paluchowska, Aneta Gawlik, Aleksandra Antosz, Marcin Kusz, Artur Bossowski, and et al. 2022. "Response to Treatment with Recombinant Human Growth Hormone (rhGH) of Short Stature Children Born Too Small for Gestational Age (SGA) in Selected Centres in Poland" Journal of Clinical Medicine 11, no. 11: 3096. https://doi.org/10.3390/jcm11113096