This section was summarized by CDR staff based on the input provided by patient groups. It has not been systematically reviewed. It has been reviewed by the submitting patient groups.
1. Brief Description of Patients Supplying Input
Due to the absence of an organized patient group for AGHD in Canada, input was received from individual patients. Two patients with AGHD provided input:
Patient A — a woman diagnosed in 1982 as having AGHD as a result of severe head trauma, and
Patient B — a woman who had been treated by transphenoidal resection and radiation for Cushing disease.
Both women live in British Columbia. Patient A has been in contact with Eli Lilly, the maker of Humatrope (somatropin), in an effort to coordinate with other AGHD patients and to encourage provincial funding of somatropin for AGHD. Patient B is one of the recipients of funding from Eli Lilly to start an advocacy group for AGHD patients.
2. Condition and Current Therapy-Related Information
Patient A had used human GH injections in the past, but had not used them for 17 years. In the meantime, she tried antidepressants and melatonin to control anxiety and depression, diet and exercise to maintain her weight, and an inhaler to control what was presumed to be asthma. None were effective. She suffered from hypoglycemia, which led to anxiety, depression, an inability to do strenuous physical activity, muscle cramps due to lactic acidosis, amenorrhea, and difficulty sleeping. The combination of hypoglycemia and insomnia left her “foggy,” greatly reducing her ability to focus on tasks such as driving and affecting her productivity at work. In May 2012, her coworkers found her barely conscious at her desk, and she was taken to the hospital where her post-prandial blood sugar was 3.8 mmol/L and her insulin-like growth factor-1 (IGF-1) was in the mid-20s ng/mL rather than the 86 ng/mL to 271 ng/mL range appropriate for her gender and age. She experienced severe suicidal thoughts after this episode. A questionnaire administered by her endocrinologist indicated struggles with mood, social relations, and sleep loss.
Patient A’s caregiver reported unpredictable and uncontrollable mood swings; he felt helpless to do anything but constantly monitor and attempt to calm her to ensure that no harm came to her or those around her.
Patient A noted that doctors were reluctant to prescribe GH as it is not recognized as being beneficial for those with AGHD. However, given her recurring depression, social isolation, and suicidal ideation, she believed the long-term risks of somatropin treatment were negligible compared with her current quality of life and was thus desperate to try it.
Patient B postponed trying GH therapy for four years due to financial constraints. She was required to work, as she is the primary breadwinner in her family, but was unable to do anything else due to fatigue. Instead of participating in her life or spending time with her husband and young son, she needed to sleep for up to 16 hours a day, and her sleep was interrupted up to 10 times a night. She also experienced osteopenia, leading to multiple fractures due to accidents that should have caused only minor injuries.
3. Related Information About the Drug Being Reviewed
Although neither patient has used Genotropin — Patient A has experience with Humatrope and Patient B with Omnitrope — both consider the benefits and barriers to access of somatropin in general to be relevant.
After slowly increasing her dose of somatropin, Patient A no longer suffers from hypoglycemia or lactic acidosis, and her IGF-1 is within the normal range. Her mental health has improved dramatically and, apart from an occasional burning sensation at the injection site, she has suffered no adverse effects after a year of use. The control of her hypoglycemia has allowed her to resume physical exercise, reducing her risk of diabetes and heart disease, and she now has better social relationships with friends, family, and colleagues. She finds the new pen devices considerably easier to use than the injections she used in the past, reducing the risk of over- or under-dosage. Patient A’s employer reported that the lack of focus, lack of energy, and irritability that she displayed before starting on somatropin have tremendously improved, making her a much more positive and productive member of the team while greatly improving her personal well-being. Patient A has coverage for somatropin through her employer, but her lifetime benefits are limited, and she is concerned about what will happen when they run out.
Patient B credits somatropin with allowing her to become a functioning wife and mother for the first time in her child’s life. After starting treatment, her sleep greatly improved, and she feels better both physically and cognitively. She is now able to take her son to swimming and Tai Kwon Do, she has taken up running again, feels stronger, and has the energy to meet with politicians, the media, and other patients to advocate for those with AGHD. She states she is terrified by the thought of having to do without treatment. She acknowledges that somatropin therapy is expensive, but sees the lack of coverage as a major barrier to the ability of patients with AGHD to be contributing members of society, regardless of their province of residence.
