Growth Hormone Deficiency, Adult, Hypopituitarism, Pituitary Insufficiency
Conditions
Brief summary
The Hypopituitary Control and Complications Study HypoCCS is a prospective, open label, global, multicentre, observational study on routine clinical care of adults with growth hormone deficiency occurring either isolated or in combination with other pituitary hormone deficiencies. The objective of this observational study is to evaluate long-term safety and health outcomes for adult growth hormone deficient participants with or without somatropin replacement therapy. As an observational study, data are collected only as provided at the discretion of the attending physician. The participant enrolled meet the criteria of growth hormone deficiency in adults as per the Humatrope label in the country where their attending physician practices, and this diagnosis is at the discretion of the attending physician. The decision to receive somatropin or remain untreated is made by the participant in consultation with their attending physician. While treatment of adult growth hormone deficient participants with somatropin has been shown to be safe and effective in clinical trials of 18 months duration, this observational study aims to provide information on health outcome and replacement therapy over longer periods of time for a larger number of participants in the context of the overall disease environment.
Interventions
Dose, frequency and duration are at discretion of attending physician, and determined on an individual basis.
Sponsors
Eli Lilly and Company
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
\- Adult growth hormone deficiency as per the local Humatrope label and as judged by the attending physician
Exclusion criteria
\- As per the local Humatrope label and as judged by the attending physician
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Clinically Significant Adverse Events | Baseline to study completion (approximately 10 years) | A summary of all reported serious adverse events (SAE) and other adverse events regardless of causality are provided in the Adverse Events module of this record. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Cardiovascular Risk Factor-Change From Baseline in Body Mass Index (BMI) | Baseline, interim time point (5 years), and study completion (10 years) | Change in BMI was used as an indicator of cardiovascular risk. Body mass index (BMI) is an estimate of body fat based on body weight divided by height squared. |
| Cardiovascular Risk Factor-Change From Baseline in Systolic (SBP) and Diastolic Blood Pressure (DBP) | Baseline, interim time point (5 years), and study completion (10 years) | Change in SBP and DBP were used as an indicator of cardiovascular risk. |
| Cardiovascular Risk Factor-Change From Baseline in Cholesterol and Triglycerides | Baseline, interim time point (5 years), and study completion (10 years) | Change from baseline in total cholesterol (TC), high-density lipoprotein cholesterol (HDL-C), low-density lipoprotein cholesterol (LDL-C) and triglycerides were used as an indicator of cardiovascular risk and are presented. |
| Percentage of Participants Experiencing a Bone Fracture (Fracture Incidence) | Baseline through 10 years | — |
| Change From Baseline in the Total Z Score of the Disease-specific Module of the Questions of Life Satisfaction (QLS-H). | Baseline, interim time point (5 years), and study completion (10 years) | QLS-H is a self-administered, weighted, quality of life (QoL) questionnaire consisting of 9 items developed for participants with growth hormone deficiency. Scores were corrected for age, gender, and country differences, and expressed as Z-scores based on country-specific reference ranges. Participants indicate how important a certain dimension of QoL is to them and are then questioned as to their degree of satisfaction with that dimension. Each item is rated on a 5-point Likert scale ranging from not important (1) to extremely important (5) and from dissatisfied (1) to very satisfied (5). The weighted score for the degree of satisfaction (weighted satisfaction) with a particular dimension=(importance - 1)x(2 x satisfaction - 5). Total Z-score is obtained by adding the individual item scores of the 9 dimensions, and range from -108 (representing very low satisfaction) to +180 (representing very high satisfaction). |
| Cardiovascular Risk Factor-Change From Baseline in Waist Circumference | Baseline, interim time point (5 years), and study completion (10 years) | Change in waist circumference was used as an indicator of cardiovascular risk. |
Other
| Measure | Time frame |
|---|---|
| Number of Participants Who Died While in the Study | Study enrollment up to approximately 10 years |
Participant flow
Pre-assignment details
Participants were initially enrolled in 2 regional observational studies (B9R-EW-GDDQ \[GDDQ\] initiated in 1997 and B9R-MC-GDEE \[GDEE\] in 1998) which were merged into the global Study B9R-MC-GDGA (GDGA, NCT01088399) in 2002. This record spans approximately 15 years from the initial enrollment in regional studies to completion of Study GDGA.
Participants by arm
| Arm | Count |
|---|---|
| Somatropin Treated Participants received somatropin therapy dosed according to the local product label during the study. No form of intervention was imposed on the participants. | 8,716 |
| Untreated Participants did not receive somatropin therapy during the study. No form of intervention was imposed on the participants. | 1,655 |
| Unknown It is unknown whether participants did or did not receive somatropin therapy during the study. No form of intervention was imposed on the participants. | 302 |
| Total | 10,673 |
Withdrawals & dropouts
| Period | Reason | FG000 | FG001 | FG002 |
|---|---|---|---|---|
| Overall Study | Adverse Event | 211 | 16 | 0 |
| Overall Study | Change to or start of somatropin therapy | 234 | 17 | 12 |
| Overall Study | Death | 204 | 45 | 0 |
| Overall Study | Enrollment criteria not met | 48 | 119 | 70 |
| Overall Study | Lack of Efficacy | 298 | 7 | 5 |
| Overall Study | Lost to Follow-up | 1,070 | 278 | 58 |
| Overall Study | Physician Decision | 531 | 88 | 16 |
| Overall Study | Protocol Violation | 10 | 1 | 0 |
| Overall Study | Sponsor decision | 1,573 | 298 | 7 |
| Overall Study | Study summary visit not completed | 2,398 | 283 | 80 |
| Overall Study | Withdrawal by Subject | 544 | 195 | 33 |
Baseline characteristics
| Characteristic | Somatropin Treated | Untreated | Unknown | Total |
|---|---|---|---|---|
| Age, Customized <20 years | 421 participants | 36 participants | 10 participants | 467 participants |
| Age, Customized 20 years to <30 years | 1258 participants | 152 participants | 36 participants | 1446 participants |
| Age, Customized 30 years to <40 years | 1475 participants | 173 participants | 54 participants | 1702 participants |
| Age, Customized 40 years to <50 years | 1975 participants | 318 participants | 76 participants | 2369 participants |
| Age, Customized 50 years to <60 years | 2048 participants | 379 participants | 74 participants | 2501 participants |
| Age, Customized 60 years to <70 years | 1116 participants | 324 participants | 38 participants | 1478 participants |
| Age, Customized >=70 years | 421 participants | 272 participants | 13 participants | 706 participants |
| Age, Customized unknown | 2 participants | 1 participants | 1 participants | 4 participants |
| Body Mass Index (BMI) | 29.7 kilograms per square meter (kg/m^2) STANDARD_DEVIATION 7 | 29.9 kilograms per square meter (kg/m^2) STANDARD_DEVIATION 7 | 30.0 kilograms per square meter (kg/m^2) STANDARD_DEVIATION 6.9 | 29.7 kilograms per square meter (kg/m^2) STANDARD_DEVIATION 7 |
| Diagnosis Leading to GHD Causes other than listed above | 458 participants | 94 participants | 21 participants | 573 participants |
| Diagnosis Leading to GHD Craniopharyngioma | 973 participants | 125 participants | 24 participants | 1122 participants |
| Diagnosis Leading to GHD Idiopathic | 1381 participants | 177 participants | 88 participants | 1646 participants |
| Diagnosis Leading to GHD Other less common specified diagnoses | 1027 participants | 198 participants | 79 participants | 1304 participants |
| Diagnosis Leading to GHD Other Tumor | 653 participants | 137 participants | 7 participants | 797 participants |
| Diagnosis Leading to GHD Pituitary adenoma | 3769 participants | 865 participants | 79 participants | 4713 participants |
| Diagnosis Leading to GHD Pituitary hemorrhage/brain injury | 455 participants | 59 participants | 4 participants | 518 participants |
| Growth Hormone Deficiency (GHD) Onset Type Adult | 6968 participants | 1422 participants | 247 participants | 8637 participants |
| Growth Hormone Deficiency (GHD) Onset Type Childhood | 1702 participants | 194 participants | 29 participants | 1925 participants |
| Growth Hormone Deficiency (GHD) Onset Type Unknown | 46 participants | 39 participants | 26 participants | 111 participants |
| IGF-I Standard Deviations Score (SDS) | -2.72 standard deviation scores STANDARD_DEVIATION 2.23 | -2.82 standard deviation scores STANDARD_DEVIATION 1.97 | -2.39 standard deviation scores STANDARD_DEVIATION 2.03 | -2.73 standard deviation scores STANDARD_DEVIATION 2.19 |
| Insulin-like Growth Factor-I (IGF-I) | 100.4 micrograms per liter (mcg/L) STANDARD_DEVIATION 78 | 75.0 micrograms per liter (mcg/L) STANDARD_DEVIATION 55.3 | 95.5 micrograms per liter (mcg/L) STANDARD_DEVIATION 63.7 | 96.4 micrograms per liter (mcg/L) STANDARD_DEVIATION 75.2 |
| Maximum Growth Hormone (GH) Peak on Stimulation Test Any test except GHRH + Arg (n=6579;1178;168;7925) | 1.1 nanograms per milliliter (ng/mL) STANDARD_DEVIATION 1.9 | 2.0 nanograms per milliliter (ng/mL) STANDARD_DEVIATION 3.7 | 5.2 nanograms per milliliter (ng/mL) STANDARD_DEVIATION 6.7 | 1.3 nanograms per milliliter (ng/mL) STANDARD_DEVIATION 2.5 |
| Maximum Growth Hormone (GH) Peak on Stimulation Test GHRH + Arginine (Arg, n=1175, 103, 51, 1329) | 2.5 nanograms per milliliter (ng/mL) STANDARD_DEVIATION 3.5 | 5.2 nanograms per milliliter (ng/mL) STANDARD_DEVIATION 8.1 | 4.4 nanograms per milliliter (ng/mL) STANDARD_DEVIATION 8.1 | 2.8 nanograms per milliliter (ng/mL) STANDARD_DEVIATION 4.4 |
| Race/Ethnicity, Customized African Descent | 226 participants | 96 participants | 14 participants | 336 participants |
| Race/Ethnicity, Customized Caucasian | 6058 participants | 1080 participants | 236 participants | 7374 participants |
| Race/Ethnicity, Customized Declined To Provide | 387 participants | 82 participants | 12 participants | 481 participants |
| Race/Ethnicity, Customized East/Southeast Asian | 460 participants | 129 participants | 7 participants | 596 participants |
| Race/Ethnicity, Customized Hispanic | 153 participants | 55 participants | 16 participants | 224 participants |
| Race/Ethnicity, Customized Other | 38 participants | 10 participants | 2 participants | 50 participants |
| Race/Ethnicity, Customized Unknown | 1370 participants | 198 participants | 12 participants | 1580 participants |
| Race/Ethnicity, Customized Western Asian | 24 participants | 5 participants | 3 participants | 32 participants |
| Region of Enrollment Austria | 77 participants | 1 participants | 1 participants | 79 participants |
| Region of Enrollment Belgium | 55 participants | 1 participants | 0 participants | 56 participants |
| Region of Enrollment Canada | 63 participants | 51 participants | 0 participants | 114 participants |
| Region of Enrollment Czech Republic | 77 participants | 5 participants | 0 participants | 82 participants |
| Region of Enrollment Denmark | 151 participants | 5 participants | 0 participants | 156 participants |
| Region of Enrollment France | 452 participants | 218 participants | 6 participants | 676 participants |
| Region of Enrollment Germany | 553 participants | 87 participants | 19 participants | 659 participants |
| Region of Enrollment Hungary | 102 participants | 2 participants | 0 participants | 104 participants |
| Region of Enrollment Iceland | 10 participants | 1 participants | 1 participants | 12 participants |
| Region of Enrollment Italy | 1242 participants | 64 participants | 49 participants | 1355 participants |
| Region of Enrollment Japan | 407 participants | 102 participants | 0 participants | 509 participants |
| Region of Enrollment Netherlands | 460 participants | 1 participants | 0 participants | 461 participants |
| Region of Enrollment Norway | 17 participants | 0 participants | 0 participants | 17 participants |
| Region of Enrollment Spain | 427 participants | 55 participants | 2 participants | 484 participants |
| Region of Enrollment Sweden | 355 participants | 3 participants | 2 participants | 360 participants |
| Region of Enrollment United Kingdom | 538 participants | 15 participants | 6 participants | 559 participants |
| Region of Enrollment United States | 3730 participants | 1044 participants | 216 participants | 4990 participants |
| Sex: Female, Male Female | 4158 Participants | 714 Participants | 155 Participants | 5027 Participants |
| Sex: Female, Male Male | 4558 Participants | 941 Participants | 147 Participants | 5646 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk | EG002 affected / at risk |
|---|---|---|---|
| deaths Total, all-cause mortality | — / — | — / — | — / — |
| other Total, other adverse events | 4,912 / 8,420 | 591 / 1,269 | 0 / 4 |
| serious Total, serious adverse events | 1,602 / 8,420 | 288 / 1,269 | 0 / 4 |
Outcome results
Primary
Clinically Significant Adverse Events
A summary of all reported serious adverse events (SAE) and other adverse events regardless of causality are provided in the Adverse Events module of this record.
Time frame: Baseline to study completion (approximately 10 years)
Population: All participants with baseline and at least 1 post-baseline visit whose study therapy was known (treated or untreated).
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Somatropin Treated | Clinically Significant Adverse Events | Myocardial Infarction | 0.8 percentage of participants |
| Somatropin Treated | Clinically Significant Adverse Events | Death | 2.58 percentage of participants |
| Somatropin Treated | Clinically Significant Adverse Events | Malignancy | 4.0 percentage of participants |
| Untreated | Clinically Significant Adverse Events | Death | 3.78 percentage of participants |
| Untreated | Clinically Significant Adverse Events | Malignancy | 5.3 percentage of participants |
| Untreated | Clinically Significant Adverse Events | Myocardial Infarction | 1.4 percentage of participants |
Secondary
Cardiovascular Risk Factor-Change From Baseline in Body Mass Index (BMI)
Change in BMI was used as an indicator of cardiovascular risk. Body mass index (BMI) is an estimate of body fat based on body weight divided by height squared.
Time frame: Baseline, interim time point (5 years), and study completion (10 years)
Population: All participants with baseline and at least 1 post-baseline visit whose study therapy was known (treated or untreated) and had BMI data.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Somatropin Treated | Cardiovascular Risk Factor-Change From Baseline in Body Mass Index (BMI) | Change in BMI at 5 years (n=2541, 218) | 0.63 kilogram per square meter (kg/m^2) | Standard Deviation 3.15 |
| Somatropin Treated | Cardiovascular Risk Factor-Change From Baseline in Body Mass Index (BMI) | Change in BMI at 10 years (n=884, 64) | 1.14 kilogram per square meter (kg/m^2) | Standard Deviation 3.67 |
| Untreated | Cardiovascular Risk Factor-Change From Baseline in Body Mass Index (BMI) | Change in BMI at 5 years (n=2541, 218) | -0.10 kilogram per square meter (kg/m^2) | Standard Deviation 3.25 |
| Untreated | Cardiovascular Risk Factor-Change From Baseline in Body Mass Index (BMI) | Change in BMI at 10 years (n=884, 64) | -0.32 kilogram per square meter (kg/m^2) | Standard Deviation 2.67 |
Secondary
Cardiovascular Risk Factor-Change From Baseline in Cholesterol and Triglycerides
Change from baseline in total cholesterol (TC), high-density lipoprotein cholesterol (HDL-C), low-density lipoprotein cholesterol (LDL-C) and triglycerides were used as an indicator of cardiovascular risk and are presented.
Time frame: Baseline, interim time point (5 years), and study completion (10 years)
Population: All participants with baseline and at least 1 post-baseline visit whose study therapy was known (treated or untreated) and had cholesterol or triglyceride data.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Somatropin Treated | Cardiovascular Risk Factor-Change From Baseline in Cholesterol and Triglycerides | Change in TC at 5 years (n=1865, 184) | -13.0 milligrams per deciliter (mg/dL) | Standard Deviation 45.74 |
| Somatropin Treated | Cardiovascular Risk Factor-Change From Baseline in Cholesterol and Triglycerides | Change in TC at 10 years (n=668, 56) | -17.9 milligrams per deciliter (mg/dL) | Standard Deviation 52.87 |
| Somatropin Treated | Cardiovascular Risk Factor-Change From Baseline in Cholesterol and Triglycerides | Change in HDL-C at 5 years (n=1667, 176) | 1.47 milligrams per deciliter (mg/dL) | Standard Deviation 16.67 |
| Somatropin Treated | Cardiovascular Risk Factor-Change From Baseline in Cholesterol and Triglycerides | Change in HDL-C at 10 years (n=615, 51) | 5.00 milligrams per deciliter (mg/dL) | Standard Deviation 17.4 |
| Somatropin Treated | Cardiovascular Risk Factor-Change From Baseline in Cholesterol and Triglycerides | Change in LDL-C at 5 years (1435, 166) | -10.7 milligrams per deciliter (mg/dL) | Standard Deviation 41.32 |
| Somatropin Treated | Cardiovascular Risk Factor-Change From Baseline in Cholesterol and Triglycerides | Change in LDL-C at 10 years (n=522, 47) | -16.4 milligrams per deciliter (mg/dL) | Standard Deviation 44.05 |
| Somatropin Treated | Cardiovascular Risk Factor-Change From Baseline in Cholesterol and Triglycerides | Change in Triglycerides at 5 years (n=1581, 178) | -9.51 milligrams per deciliter (mg/dL) | Standard Deviation 99.67 |
| Somatropin Treated | Cardiovascular Risk Factor-Change From Baseline in Cholesterol and Triglycerides | Change in Triglycerides at 10 years (n=571, 51) | -16.4 milligrams per deciliter (mg/dL) | Standard Deviation 107.8 |
| Untreated | Cardiovascular Risk Factor-Change From Baseline in Cholesterol and Triglycerides | Change in Triglycerides at 10 years (n=571, 51) | -55.8 milligrams per deciliter (mg/dL) | Standard Deviation 90.12 |
| Untreated | Cardiovascular Risk Factor-Change From Baseline in Cholesterol and Triglycerides | Change in TC at 5 years (n=1865, 184) | -14.6 milligrams per deciliter (mg/dL) | Standard Deviation 46.02 |
| Untreated | Cardiovascular Risk Factor-Change From Baseline in Cholesterol and Triglycerides | Change in LDL-C at 5 years (1435, 166) | -11.5 milligrams per deciliter (mg/dL) | Standard Deviation 35.91 |
| Untreated | Cardiovascular Risk Factor-Change From Baseline in Cholesterol and Triglycerides | Change in TC at 10 years (n=668, 56) | -43.1 milligrams per deciliter (mg/dL) | Standard Deviation 55.81 |
| Untreated | Cardiovascular Risk Factor-Change From Baseline in Cholesterol and Triglycerides | Change in Triglycerides at 5 years (n=1581, 178) | -0.85 milligrams per deciliter (mg/dL) | Standard Deviation 115.7 |
| Untreated | Cardiovascular Risk Factor-Change From Baseline in Cholesterol and Triglycerides | Change in HDL-C at 5 years (n=1667, 176) | -0.54 milligrams per deciliter (mg/dL) | Standard Deviation 13.82 |
| Untreated | Cardiovascular Risk Factor-Change From Baseline in Cholesterol and Triglycerides | Change in LDL-C at 10 years (n=522, 47) | -29.3 milligrams per deciliter (mg/dL) | Standard Deviation 41.44 |
| Untreated | Cardiovascular Risk Factor-Change From Baseline in Cholesterol and Triglycerides | Change in HDL-C at 10 years (n=615, 51) | 0.39 milligrams per deciliter (mg/dL) | Standard Deviation 14.7 |
Secondary
Cardiovascular Risk Factor-Change From Baseline in Systolic (SBP) and Diastolic Blood Pressure (DBP)
Change in SBP and DBP were used as an indicator of cardiovascular risk.
Time frame: Baseline, interim time point (5 years), and study completion (10 years)
Population: All participants with baseline and at least 1 post-baseline visit whose study therapy was known (treated or untreated) and BP data.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Somatropin Treated | Cardiovascular Risk Factor-Change From Baseline in Systolic (SBP) and Diastolic Blood Pressure (DBP) | Change in SBP at 5 years (n=2746, 272) | 1.04 millimeters of mercury (mmHg) | Standard Deviation 17.49 |
| Somatropin Treated | Cardiovascular Risk Factor-Change From Baseline in Systolic (SBP) and Diastolic Blood Pressure (DBP) | Change in SBP at 10 years (n=960, 73) | 1.98 millimeters of mercury (mmHg) | Standard Deviation 18.81 |
| Somatropin Treated | Cardiovascular Risk Factor-Change From Baseline in Systolic (SBP) and Diastolic Blood Pressure (DBP) | Change in DBP at 5 years (n=2740, 272) | -0.85 millimeters of mercury (mmHg) | Standard Deviation 11.11 |
| Somatropin Treated | Cardiovascular Risk Factor-Change From Baseline in Systolic (SBP) and Diastolic Blood Pressure (DBP) | Change in DBP at 10 years (n=955, 73) | -1.23 millimeters of mercury (mmHg) | Standard Deviation 11.85 |
| Untreated | Cardiovascular Risk Factor-Change From Baseline in Systolic (SBP) and Diastolic Blood Pressure (DBP) | Change in DBP at 10 years (n=955, 73) | -2.84 millimeters of mercury (mmHg) | Standard Deviation 13.37 |
| Untreated | Cardiovascular Risk Factor-Change From Baseline in Systolic (SBP) and Diastolic Blood Pressure (DBP) | Change in SBP at 5 years (n=2746, 272) | -0.48 millimeters of mercury (mmHg) | Standard Deviation 18.88 |
| Untreated | Cardiovascular Risk Factor-Change From Baseline in Systolic (SBP) and Diastolic Blood Pressure (DBP) | Change in DBP at 5 years (n=2740, 272) | -0.76 millimeters of mercury (mmHg) | Standard Deviation 11.71 |
| Untreated | Cardiovascular Risk Factor-Change From Baseline in Systolic (SBP) and Diastolic Blood Pressure (DBP) | Change in SBP at 10 years (n=960, 73) | 3.71 millimeters of mercury (mmHg) | Standard Deviation 20.04 |
Secondary
Cardiovascular Risk Factor-Change From Baseline in Waist Circumference
Change in waist circumference was used as an indicator of cardiovascular risk.
Time frame: Baseline, interim time point (5 years), and study completion (10 years)
Population: All participants with baseline and at least 1 post-baseline visit whose study therapy was known (treated or untreated) and had waste circumference data.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Somatropin Treated | Cardiovascular Risk Factor-Change From Baseline in Waist Circumference | Change in circumference at 5 years (n=1719, 133) | 0.85 centimeters (cm) | Standard Deviation 9.06 |
| Somatropin Treated | Cardiovascular Risk Factor-Change From Baseline in Waist Circumference | Change in circumference at 10 years (n=610, 34) | 3.57 centimeters (cm) | Standard Deviation 10.22 |
| Untreated | Cardiovascular Risk Factor-Change From Baseline in Waist Circumference | Change in circumference at 5 years (n=1719, 133) | 0.10 centimeters (cm) | Standard Deviation 8.52 |
| Untreated | Cardiovascular Risk Factor-Change From Baseline in Waist Circumference | Change in circumference at 10 years (n=610, 34) | 3.03 centimeters (cm) | Standard Deviation 7.76 |
Secondary
Change From Baseline in the Total Z Score of the Disease-specific Module of the Questions of Life Satisfaction (QLS-H).
QLS-H is a self-administered, weighted, quality of life (QoL) questionnaire consisting of 9 items developed for participants with growth hormone deficiency. Scores were corrected for age, gender, and country differences, and expressed as Z-scores based on country-specific reference ranges. Participants indicate how important a certain dimension of QoL is to them and are then questioned as to their degree of satisfaction with that dimension. Each item is rated on a 5-point Likert scale ranging from not important (1) to extremely important (5) and from dissatisfied (1) to very satisfied (5). The weighted score for the degree of satisfaction (weighted satisfaction) with a particular dimension=(importance - 1)x(2 x satisfaction - 5). Total Z-score is obtained by adding the individual item scores of the 9 dimensions, and range from -108 (representing very low satisfaction) to +180 (representing very high satisfaction).
Time frame: Baseline, interim time point (5 years), and study completion (10 years)
Population: All participants with baseline and at least 1 post-baseline visit whose study therapy was known (treated or untreated) and has a Total Z Score.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Somatropin Treated | Change From Baseline in the Total Z Score of the Disease-specific Module of the Questions of Life Satisfaction (QLS-H). | Change in Total Z score at 5 years (n=475, 39) | 0.70 Z-score | Standard Deviation 1.46 |
| Somatropin Treated | Change From Baseline in the Total Z Score of the Disease-specific Module of the Questions of Life Satisfaction (QLS-H). | Change in Total Z score at 10 years (n=103, 3) | 0.53 Z-score | Standard Deviation 1.39 |
| Untreated | Change From Baseline in the Total Z Score of the Disease-specific Module of the Questions of Life Satisfaction (QLS-H). | Change in Total Z score at 5 years (n=475, 39) | 0.21 Z-score | Standard Deviation 1.14 |
| Untreated | Change From Baseline in the Total Z Score of the Disease-specific Module of the Questions of Life Satisfaction (QLS-H). | Change in Total Z score at 10 years (n=103, 3) | -1.01 Z-score | Standard Deviation 1.49 |
Secondary
Percentage of Participants Experiencing a Bone Fracture (Fracture Incidence)
Time frame: Baseline through 10 years
Population: All participants with baseline and at least 1 post-baseline visit and who provided bone fracture information.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Somatropin Treated | Percentage of Participants Experiencing a Bone Fracture (Fracture Incidence) | 3.75 percentage of participants |
| Untreated | Percentage of Participants Experiencing a Bone Fracture (Fracture Incidence) | 4.02 percentage of participants |
| Unknown | Percentage of Participants Experiencing a Bone Fracture (Fracture Incidence) | 0.00 percentage of participants |
Other Pre-specified
Number of Participants Who Died While in the Study
Time frame: Study enrollment up to approximately 10 years
Population: All participants with baseline and at least 1 post-baseline visit.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Somatropin Treated | Number of Participants Who Died While in the Study | 229 participants |
| Untreated | Number of Participants Who Died While in the Study | 50 participants |
| Unknown | Number of Participants Who Died While in the Study | 0 participants |