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Phung OJ, Coleman CI, Baker EL, et al. Effectiveness of Recombinant Human Growth Hormone (rhGH) in the Treatment of Patients With Cystic Fibrosis [Internet]. Rockville (MD): Agency for Healthcare Research and Quality (US); 2010 Oct. (Comparative Effectiveness Reviews, No. 23.)

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Effectiveness of Recombinant Human Growth Hormone (rhGH) in the Treatment of Patients With Cystic Fibrosis [Internet].

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Appendix DGlossary

Alltagsleben (Every Day Life)

General quality of life measure developed in German-speaking populations. Higher scores indicate higher quality of life.

Anti-Human Growth Hormone (Anti-hGH) Antibodies

Proteins made by the host immune system that bind and neutralize human growth hormone.


A breakdown product from red blood cell catabolism whose levels are increased in liver disease. Presented in units of mg/dl.

Bio-Electrical Impedance Assessment

Method for estimation of proportion of lean body mass versus fat mass. Lean body mass includes mass from bone, muscle, water, and other body tissues. BIA utilizes electrical current to determine body content from the rate of electrical flow.

Body Mass Index

Measure of body fat relative to height. Presented as a ratio of weight to square of the height. Presented in units kg/m2.

Bone Mineral Content

Measure of the amount of bone in the body. Measurement is usually done with dual energy x-ray absorptiometry (DEXA). Presented in units of weight such as grams.

Burkholderia cepacia

A pathogenic gram negative bacteria which can cause pneumonia in cystic fibrosis patients with underlying lung problems.

Child Health Questionnaire (CHQ)

General health measure containing 75 items in 10 health concepts: physical functioning, bodily pain, role/social limitations attributable to physical condiction, general health perceptions, role/social limitations attributable to emotions, role/social limitations attributable to behavior, mental health behavior problems, self-esteem, and limitations in family activities. Higher scores indicate higher quality of life.

Confidence Intervals

A range that is likely to include the given value. Usually presented as a percent (%). For example, a value with 95% confidence interval implies that when a measurement is made 100 times, it will fall within the given range 95% of the time.

Correlation Coefficient

A value (which usually ranges from zero to one) that indicates the degree of relationship between two variables. For example, a correlation coefficient of one would indicate a strong relationship.

Cystic Fibrosis Quality of Life (CFQoL) Questionnaire

Disease-specific questionnaire consisting of 52 items across nine domains: physical functioning, social functioning, treatment issues, chest symptoms, emotional functioning, concerns for the future, interpersonal relationships, body image, and career issues. Higher scores indicate higher quality of life.

Cystic Fibrosis Questionnaire (CFQ)

Disease-specific questionnaire with 44 items across 12 dimensions: physical functioning, role, vitality, emotional functioning, social, body image, eating disturbances, treatment burden, health perceptions, weight, respiratory symptoms, and digestive symptoms. Higher scores indicate higher quality of life.

Cystic Fibrosis Transmembrane Regulator

Protein that is responsible for transport of sodium and chloride across epithelial membranes. Defects in the protein are responsible for the viscous and excessive secretions in cystic fibrosis.

Cystic Fibrosis-Related Diabetes

A type of glucose intolerance caused by insulin resistance and decreased insulin production (due to scarring of the pancreas).

DerSimonian and Laird Random-Effects Model

A statistical method based on the assumption that the effects observed in different studies (in a meta-analysis) are truly different.

Dual Energy X-Ray Absorptiometry

A common method of measuring bone mineral content (BMC) using two x-ray beams with different energy levels. Absorption due to soft tissue is taken into account (and subtracted out) when determining BMC.

Egger’s Weighted Regression Statistics

A method of identifying and measuring publication bias.

Euro-QOL 5D (EQ-5D)

A multidimension descriptive system of health status. Five dimensions are: mobility, self care, usual activity, pain, discomfort, and anxiety-depression. Higher scores indicate higher quality of life.

Forced Expiratory Volume in One Second

Volume of air forcefully exhaled in one second. Usually presented in units of liters.

Forced Vital Capacity

Total volume of air that can be exhaled forcefully after a deep inhalation. Usually presented in units of liters.


Concentrated deposits of bile that can vary in size and usually form in the gallbladder or bile duct.


A nonessential amino acid whose levels may be depleted in cystic fibrosis patients due to malnutrition and acute stress from infections.

Glutamate-Oxaloacetate Transaminase

Liver enzyme whose levels are increased during liver damage.

Glutamate-Pyruvate Transaminase

Liver enzyme whose levels are increased during liver damage.

Glycosylated Hemoglobin A1c

Measure of the amount of sugar-bound hemoglobin. Marker of plasma sugar concentrations over the past three months. Presented in units of percent (%) of total hemoglobin.

Health Related Quality of Life

Assessment of the overall well-being of a patient. Usually in the form of questionnaires that can be tailored to specific disease states such as cystic fibrosis.

Human Growth Hormone

Also known as somatotropin. A naturally occurring peptide that is responsible for growth of cells in several areas of the body including muscle and bone.


A condition where levels of albumin, which is produced by the liver, are low in the blood. Usually indicative of liver damage.


Measure of degree of variation due to statistical heterogeneity. Usually reported as a percent ranging from 0 to 100.

Idiopathic Short Stature

An unexplained condition where the patient has a significantly lower-than-expected height.

Insulin-Like Growth Factor 1

Protein hormone responsible for growth and development. Levels increase and decrease with growth hormone levels and malnutrition respectively. Also, a potent inhibitor of cell apoptosis; therefore, it is used as a lab marker to evaluate cancer risk in patients receiving recombinant human growth hormone therapy. Presented in units ng/ml.

Insulin-Like Growth Factor-Binding Protein 3

Protein that binds and regulates insulin-like growth factors including insulin-like growth factor 1 (IGF-1). Used as a lab marker to evaluated cancer risk in patients receiving recombinant human growth hormone therapy. Presented in units ng/ml.

Interquartile Range

Collection of values that fall between the 25th percentile and 75th percentile.

Lean Body Mass

Total weight of all components of the body (muscle, bone, and other tissues) excluding the weight from fat. Used as an efficacy endpoint in recombinant growth hormone therapy. Usually presented in kilograms.

Lean Tissue Mass (LTM)

See lean body mass.


An amino acid whose isotope is used to determine protein turnover.

Leucine Oxidation

Measurement made with leucine isotope that represents protein oxidation, which represents elimination of amino acids from the body. Presented in units μmol/kg*h.

Liver Cirrhosis

A condition resulting from chronic disease of the liver where normal tissue has been replaced by fibrous scar tissue.

Medical Outcomes Short Form 36 (SF-36)

General quality of life scale. Contains eight domains: physical functioning, role limiations due to physical problems, social functioning, bodily pain, mental health, role limitations due to emotional problems, vitality, and general health perceptions. Higher scores indicate higher quality of life.


The process of extracting and pooling data from several studies investigating a similar topic to synthesize a final outcome.

Nonoxidative Leucine Disappearance

Measurement made with leucine isotope that represents overall body protein synthesis. Presented in units μmol/kg*h.

Nottingham Health Profile (NHP)

General quality of life questionnaire designed to measure perceived health problems and their impact on activities of daily living. Higher scores indicate poorer health.

Oxidation/[Nonoxidative Leucine Disappearance (NOLD)]

Ratio of leucine oxidation to NOLD provides an estimate of whole body protein catabolism. Presented in units μmol/kg*h.

Oxygen Pulsepeak

Amount of oxygen consumed per heart beat during exercise. Presented in units ml/beat.

Oxygen Uptake

Volume of oxygen used per unit of time. Presented in units mL/min.

Peak Oxygen Uptake

Volume of oxygen used during the last 30 seconds of exercise. Presented in units mL.

Prader-Willi Syndrome

A genetic disorder characterized by short stature, low muscle tone, cognitive disabilities, incomplete sexual development, behavioral problems, and constant feeling of hunger.

Publication Bias

The possibility that published studies may not represent all the studies that have been conducted, and therefore, create bias by being left out of a meta-analysis.

Q Statistic

A test to assess the presence of statistical heterogeneity among several studies.

Quality of Well-Being Scale (QWB)

General health status measurement system, which assigns scores to levels of well-being based on social preferences and physical function. Three dimensions measure are mobility, physical activity, and social activity. Higher scores indicate higher quality of life.

Questions on Life Satisfaction

General health status measure on eight dimensions (friends/acquaintances, leisure time/hobbies, general health, income/financial security, occupation/work, housing/living condition, family life/children, and partner relationship/sexuality) and eight health-related dimensions (physical condition/fitness, ability to relax, energy/zest for life, mobility, vision and hearing, freedom from anxiety, freedom from aches and pain, and independence from help/care). Higher score indicates higher quality of life.

Rate of Appearance of Leucine

Measurement made with leucine isotope that represents the rate of protein breakdown. Presented in units μmol/kg*h.

Recombinant Human Growth Hormone

Also known as somatropin. A synthetic form of the naturally occurring human growth hormone that is used for a variety of disorders including growth hormone deficiency, weight management, and possibly cystic fibrosis.

Relative Risks

The ratio of an event occurring in an exposed group to an event occurring in a non-exposed group in a given population.

Sensitivity Analyses

A ‘what if’ analysis that helps determine the robustness of a study. Helps determine the degree of importance of each variable for a given outcome.

Sickness Impact Profile (SIP)

Quality of life assessment tool which contains 136 items grouped into 12 categories of activity, evaluating behavioral dysfunction, physical dysfunction which summarizes three categories (ambulation, mobility, body care and movement), and psychosocial dysfunction which summarizes four categories (social interaction, communication, alertness, and emotional behavior). Higher scores indicate poorer health.

Skinfold Thickness

Measure of the amount of fat under the skin, determined by caliper. Measurements at several sites are required because the proportion of fat at each site varies with age, gender, and race. Skinfold measurements typically taken at triceps, subscapular, and supra-ileac sites.

Standard Deviations

A measure of the variability of a data set. For a simple data set with numbers, can be calculated using the following formula:


  • σ is standard deviation
  • xm is the average
  • ∑(x-xm) is the sum of xm subtracted from each individual number x
  • N is the total number of values
  • Note: Other formulas also exist.
Standard Deviation Scores

See Z-Scores.

Statistical Heterogeneity

Variability in the observed effects among studies in a meta-analysis. Steatorrhea: The presence of excessive amounts of fat in stools. Usually occurs secondary to pancreatic disease.

Sweat Test

A standard diagnostic test for cystic fibrosis that measures the concentration of chloride in a patient’s sweat after induction of sweat glands via iontophoresis. A chloride concentration of 60 mmol/L or greater is consistent with the diagnosis of cystic fibrosis, while a concentration of 40 to 60 mmol/L is borderline.

Tanner Staging

A scale based of the physical development of primary and secondary sexual characteristics including presence and quality of pubic hair (males and females), breast size (females), and testicular volume (males). Stages range from 1 to 5 in increasing development.


Hormone produced by the thyroid gland that regulates the metabolic rate of the body.

Trim-and-Fill Method

In cases where publication bias is detected, theoretical studies are statistically either imputed or removed to yield a theoretical result.

Turner Syndrome

A genetic disorder of the sex chromosomes resulting in a variety of physical abnormalities including short stature.


Volume of air obtained during the last 30 seconds of exercise. Presented in units L/min.


Unit of power that helps measure rate of energy expenditure.

Weighted Mean Difference

Composite endpoint determined by the pooling of continuous data from all studies in the meta-analysis. Each study’s mean, standard deviation, and sample size is taken into account to determine its level of contribution to the composite endpoint. For example, a study with a large sample size will have a greater impact on the composite endpoint than a similar study with a small sample size.


The expected weight for a given height on a reference population growth chart.


Difference between the value for an individual and the median value of the reference population, divided by the standard deviation of the reference population.

Z-Score (or SDScore or SDS)=(observed value)-(median reference value)Standard deviation of reference population

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