Jaclyn F. Wood and Dr. Barbara Mandleco, Nursing
The purpose of this research project was to talk to children who participated in a clinical trial and obtain their perceptions surrounding their own involvement in the clinical trial. In a previous ORCA funded project I analyzed interviews with the parents to find out their perceptions. In this project I conducted interviews with the children and gained their insight concerning their involvement in the clinical trial.
Salt Lake City Shriners Hospital has needed and desired to have this research project done. Working with their research coordinator, Jeanne Siebert, I was able to gather a sample of five families all of which had a child with the disease Osteogenesis Imperfecta (abbreviated as O.I.). Having this disease the children are unable to build normally dense bone tissue. They develop defective connective tissue in their bones also referred to as mutant collagen. Normal collagen makes up the structure and provides the strength of the bone (Anderson, 2002), but the collagen of these children is recognized as being defective by their bodies and is destroyed or incorporated into their bony extra cellular matrix (Cole, 2002). As a result, these children have weaker, less structured bones and are more susceptible to bone fractures and bone destruction than those with normal healthy bones (O.I. Foundation, 2002).
The children of these five families were enrolled in a clinical trial at Salt Lake City’s Shriners Hospital where they were administered biphosphonate medications in hopes of increasing the density of their bones and thereby reducing their fractures. The biphosphonate medications (alendronate sodium and pomedronate disodium) have proven to decrease the breakdown of the children’s bone tissue.
Through my interviews of these children I have come to understand the stresses and benefits of being in a clinical trial. The following information discusses all of my findings. By examining the perceptions of the children I was able to gather information pertinent to the bettering of clinical trials at Shriners Hospital.
The five children enrolled in the clinical trial at Shriners Hospital ranged in age from 8-19 years old. Three were female and two male. Their grades in school ranged from 3rd grade to a high school graduate. They were all diagnosed with their condition with in one hour of birth except one female who wasn’t diagnosed until nine months of age (when the doctor noticed her sclera where blue-a sure sign of O.I.). Enrollment in the clinical trial of taking biphosphonate mediation for the children occurred as early as December 1998 and as late as February 2003. Previous to this clinical trial only one child had been enrolled in any other clinical trial. Previous treatments they took place in included rodding surgeries, closed reduction surgeries, calcium supplements, and continual oxygen supplement. The hobbies and interests of the children included reading, talking on the phone, playing on the computer, playing outdoors, spending time with pets, participating in art, and even snowboarding.
The children expressed great joy and satisfaction in the current results of the study. These results included increased happiness due to decreased fractures, stronger bones, increased bone densities, increased confidence, and decreased pain levels. Only one child (the youngest of the subjects) felt she hadn’t experienced the full effects of the medication as she still experienced fractures from time to time. The subjects all considered the Shriner staff members as their friends. One young boy said the staff members were like family to him due to the great amount of time he spent with them. The kindness and love they shared with him was greatly appreciated.
Only two negative aspects of the clinical trial were mentioned by the children in the interviews. The stressful anticipation of and the insertion of the I.V. was the first one mentioned. One little girl stated she would only let the anesthesiologist place the I.V. as he does it so easily and in one try. The other negative aspect was the malaise or sickness the children felt the week following the administration of the biphosphonate medication. The weakness and flu like symptoms their bodies experience though, were minimal in comparison with the benefits the children said they experienced.
When asked the future goals the children wanted to pursue in relation to their OI conditions the children replied with varied and creative answers. One child wanted the biphosphonate medication to be developed into an oral form that was flavored according to the patient’s liking. Another child wanted to grow taller and have a sufficiently strong back as his back is so extremely weak. The most common goal of the children was to develop a strong enough medication to totally cure Osteogenesis Imperfecta.
Upon finishing this project I feel great satisfaction and joy in being a researcher for the College of Nursing. The knowledge I gained from the research enables me to be a more educated nurse and researcher. Not only will this study benefit myself, the College of Nursing, and BYU, but also Shriners Hospital. A much more in-depth report has been developed for Shriners. Through the data analysis provided I hope the hospital will have gained the information needed to further develop future clinical trials.