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017—Understanding and Meeting the Needs of Those Using Growth Hormone Injection Devices
ABSTRACTS OF THE PEDIATRIC ENDOCRINOLOGY NURSING SOCIETY CONVENTION
focuses on improving patient compliance and persistence while maximizing patient outcomes during Nutropin GH therapy.
Informational 017—Understanding and Meeting the Needs of Those Using Growth Hormone Injection Devices. Nerissa Kreher, Dorothy Parker, Serono, Inc., Rockland, Boston, MA, Fast Forward Research Ltd., Manchester Road, Wilmslow, UK There are no documented safety/efficacy differences between marketed recombinant human growth hormones (r-hGHs). All are injected subcutaneously over a period of years, usually on a daily basis, which can lead to compliance problems. Beyond syringes and needles, various r-hGH injection devices have been developed to improve convenience for patients, particularly children. Despite advances, room for device improvements remains. Developments that reduce pain are likely to increase compliance. It may also be possible to improve existing features or introduce new ones, such as physicianprogrammed preset dosing, administration/cartridge replacement reminder alarms, or adjustable injection speeds. Some of these features may also help to improve compliance if incorporated without compromising device simplicity. The aims of this open, prospective study were to identify which attributes of an r-hGH injection device are considered most important by parents of r-hGH-treated children, teenage patients, physicians, and nurses with experience of r-hGH injection and to determine how they rate existing devices in these attributes. The opinions of 67 individuals were captured in discussion sessions. Parents, patients, physicians, and nurses were asked to rate 19 device attributes, using a questionnaire, and to rank four different rhGH injection devices (including a conceptual electronic device) in order of preference. Reliability, ease of use, lack of pain during injection, safety in use/storage, and fewer preparatory steps before, during, and after use were considered the most desirable attributes. The data show a preference for an electronic device over an automatic, multidose device; a needle-free device; or a manual, ready-to-use disposable device. In the opinion of parents, patients, physicians, and nurses using r-hGH injection devices, based on the 19 tested attributes, a device combining reliability with simplicity, while delivering therapy with minimal pain, was preferred. An electronic device combining many of the most useful features of existing devices with novel additional functions was the preferred option.
018—Electronic Injection Delivery Device (e-device) for Growth Hormone Therapy. Lynn Board, Nerissa C. Kreher, Paul Desrosiers, Serono, Inc., Rockland, Boston, MA Background: In children and adolescents, growth hormone (GH) therapy requires a long-term commitment to daily subcutaneous injections. Improvements in device delivery methods have been made, including injection pens, autoinjector pens, and needle-free delivery devices. Because of the requirement for daily injections, patient acceptance of devices is important. Developments that improve patient acceptance will likely increase compliance with GH therapy. A novel electronic injection delivery device (e-device) that allows for physician programmed preset dosing, cartridge alarms, and injection monitoring is now available. A user trial, evaluating the performance and handling of this device among subjects, nurses, and physicians, is described. Study Design: A Phase IV international, multicenter, open-label, uncontrolled study enrolling 70 subjects is being conducted.
221
Pediatric subjects with a requirement for Saizen therapy (either naRve or already on therapy) will be included in the study. The objective of the study was to evaluate the e-device performance and handling in everyday practice. The impressions of subjects, nurses, and physicians will be obtained. Evaluation of the graphic interface, instruction manual, training, and training materials will be determined over a 2-month period. At baseline, after informed consent, the patient and parents will be trained on the use of the e-device. After this training session, a questionnaire that will evaluate the training duration, cartridge loading issues, needle attachment and detachment, injection process, functionality, and instructions for use will be completed. After 2 months of patient use, a telephone questionnaire will be administered to assess any problems encountered as well as the impressions of the patient on the usefulness and reliability of e-device functions. Another questionnaire will be administered after 2 months. Information collected will include the patient’s overall impression of the device, thoughts regarding the instructions for use, and the usefulness and reliability of the e-device functions. Safety will be assessed throughout the study. Conclusions: This study will allow the assessment of the acceptability of the e-device in common practice among subjects, nurses, and physicians. Although improvements in GH administration devices have continued, the e-device may represent a definite advance in GH delivery systems. Further studies to assess additional outcomes such as patient compliance and parental stress are planned.
019—Smith–Lemli–Opitz Syndrome: A Rare Connection to the Endocrine System. Charlotte Bosmans, Debbie Rawson, Sue Hale, National Institutes of Health, Bethesda, MD Smith–Lemli–Opitz Syndrome (SLOS) is an autosomal recessive genetic disorder caused by a defect in cholesterol biosynthesis. Specifically, a deficiency of 7-dehydrocholesterol reductase, an enzyme that converts 7-dehydrocholesterol to cholesterol in the final step of cholesterol synthesis, results in multiple malformations and mental retardation. Adrenal cortex utilizes cholesterol to synthesize steroid hormones. Although uncompensated adrenal insufficiency is uncommon, corticotropin-releasing hormone testing has demonstrated that many SLOS patients experience compensated adrenal insufficiency. When a patient is acutely ill or under major physiologic stress, early intervention is essential in preventing adrenal crisis. Initial treatment for a very sick or physically traumatized child is infusion of fresh frozen plasma (FFP), which contains cholesterol, the missing molecule in SLOS. FFP not only is used as a prophylactic treatment for adrenal insufficiency but also provides cholesterol for other cells in the body that have become deficient. If the child shows any evidence of adrenal insufficiency, there should be no hesitation in giving stress hormones. Some signs and symptoms of adrenal insufficiency are nausea, vomiting, dehydration, abdominal pain, fever, fatigue, weight loss, hypotension, confusion, and coma. Diagnosis of adrenal insufficiency is made by measuring hormones and electrolytes in the blood and urine. Because most emergency room doctors and surgeons are not familiar with SLOS, these patients may not receive prompt intervention. It is important for nurses to be aware not only of the risks of adrenal insufficiency in this population when they are severely ill or when they need surgery but also of the need for referral to a metabolic geneticist who can recommend appropriate therapy.
020—Stepping Stonesk: A Program of Adherence and Persistence for Nutropin Patients. Cynthia Gordner, Penn State Children’s Hospital, Milton S. Hershey Medical Center, Hershey, PA
focuses on improving patient compliance and persistence while maximizing patient outcomes during Nutropin GH therapy.
Informational 017—Understanding and Meeting the Needs of Those Using Growth Hormone Injection Devices. Nerissa Kreher, Dorothy Parker, Serono, Inc., Rockland, Boston, MA, Fast Forward Research Ltd., Manchester Road, Wilmslow, UK There are no documented safety/efficacy differences between marketed recombinant human growth hormones (r-hGHs). All are injected subcutaneously over a period of years, usually on a daily basis, which can lead to compliance problems. Beyond syringes and needles, various r-hGH injection devices have been developed to improve convenience for patients, particularly children. Despite advances, room for device improvements remains. Developments that reduce pain are likely to increase compliance. It may also be possible to improve existing features or introduce new ones, such as physicianprogrammed preset dosing, administration/cartridge replacement reminder alarms, or adjustable injection speeds. Some of these features may also help to improve compliance if incorporated without compromising device simplicity. The aims of this open, prospective study were to identify which attributes of an r-hGH injection device are considered most important by parents of r-hGH-treated children, teenage patients, physicians, and nurses with experience of r-hGH injection and to determine how they rate existing devices in these attributes. The opinions of 67 individuals were captured in discussion sessions. Parents, patients, physicians, and nurses were asked to rate 19 device attributes, using a questionnaire, and to rank four different rhGH injection devices (including a conceptual electronic device) in order of preference. Reliability, ease of use, lack of pain during injection, safety in use/storage, and fewer preparatory steps before, during, and after use were considered the most desirable attributes. The data show a preference for an electronic device over an automatic, multidose device; a needle-free device; or a manual, ready-to-use disposable device. In the opinion of parents, patients, physicians, and nurses using r-hGH injection devices, based on the 19 tested attributes, a device combining reliability with simplicity, while delivering therapy with minimal pain, was preferred. An electronic device combining many of the most useful features of existing devices with novel additional functions was the preferred option.
018—Electronic Injection Delivery Device (e-device) for Growth Hormone Therapy. Lynn Board, Nerissa C. Kreher, Paul Desrosiers, Serono, Inc., Rockland, Boston, MA Background: In children and adolescents, growth hormone (GH) therapy requires a long-term commitment to daily subcutaneous injections. Improvements in device delivery methods have been made, including injection pens, autoinjector pens, and needle-free delivery devices. Because of the requirement for daily injections, patient acceptance of devices is important. Developments that improve patient acceptance will likely increase compliance with GH therapy. A novel electronic injection delivery device (e-device) that allows for physician programmed preset dosing, cartridge alarms, and injection monitoring is now available. A user trial, evaluating the performance and handling of this device among subjects, nurses, and physicians, is described. Study Design: A Phase IV international, multicenter, open-label, uncontrolled study enrolling 70 subjects is being conducted.
221
Pediatric subjects with a requirement for Saizen therapy (either naRve or already on therapy) will be included in the study. The objective of the study was to evaluate the e-device performance and handling in everyday practice. The impressions of subjects, nurses, and physicians will be obtained. Evaluation of the graphic interface, instruction manual, training, and training materials will be determined over a 2-month period. At baseline, after informed consent, the patient and parents will be trained on the use of the e-device. After this training session, a questionnaire that will evaluate the training duration, cartridge loading issues, needle attachment and detachment, injection process, functionality, and instructions for use will be completed. After 2 months of patient use, a telephone questionnaire will be administered to assess any problems encountered as well as the impressions of the patient on the usefulness and reliability of e-device functions. Another questionnaire will be administered after 2 months. Information collected will include the patient’s overall impression of the device, thoughts regarding the instructions for use, and the usefulness and reliability of the e-device functions. Safety will be assessed throughout the study. Conclusions: This study will allow the assessment of the acceptability of the e-device in common practice among subjects, nurses, and physicians. Although improvements in GH administration devices have continued, the e-device may represent a definite advance in GH delivery systems. Further studies to assess additional outcomes such as patient compliance and parental stress are planned.
019—Smith–Lemli–Opitz Syndrome: A Rare Connection to the Endocrine System. Charlotte Bosmans, Debbie Rawson, Sue Hale, National Institutes of Health, Bethesda, MD Smith–Lemli–Opitz Syndrome (SLOS) is an autosomal recessive genetic disorder caused by a defect in cholesterol biosynthesis. Specifically, a deficiency of 7-dehydrocholesterol reductase, an enzyme that converts 7-dehydrocholesterol to cholesterol in the final step of cholesterol synthesis, results in multiple malformations and mental retardation. Adrenal cortex utilizes cholesterol to synthesize steroid hormones. Although uncompensated adrenal insufficiency is uncommon, corticotropin-releasing hormone testing has demonstrated that many SLOS patients experience compensated adrenal insufficiency. When a patient is acutely ill or under major physiologic stress, early intervention is essential in preventing adrenal crisis. Initial treatment for a very sick or physically traumatized child is infusion of fresh frozen plasma (FFP), which contains cholesterol, the missing molecule in SLOS. FFP not only is used as a prophylactic treatment for adrenal insufficiency but also provides cholesterol for other cells in the body that have become deficient. If the child shows any evidence of adrenal insufficiency, there should be no hesitation in giving stress hormones. Some signs and symptoms of adrenal insufficiency are nausea, vomiting, dehydration, abdominal pain, fever, fatigue, weight loss, hypotension, confusion, and coma. Diagnosis of adrenal insufficiency is made by measuring hormones and electrolytes in the blood and urine. Because most emergency room doctors and surgeons are not familiar with SLOS, these patients may not receive prompt intervention. It is important for nurses to be aware not only of the risks of adrenal insufficiency in this population when they are severely ill or when they need surgery but also of the need for referral to a metabolic geneticist who can recommend appropriate therapy.
020—Stepping Stonesk: A Program of Adherence and Persistence for Nutropin Patients. Cynthia Gordner, Penn State Children’s Hospital, Milton S. Hershey Medical Center, Hershey, PA