Dear Doctor,
Mother's Day was celebrated just last weekend and in honor of Mother's Day, I would like to write a letter on behalf of the parents who are fighting for the lives of their chronically ill children. As a mother to a boy who suffers from chronic conditions resulting from a craniopharyngioma, I understand all too well the hardships that face the families who deal with the devastating effects of life-threatening and life-changing illnesses.
Our story of desperation to find help for my son's untreated hypothalamic obesity and hyperphagia is documented in detail in this blog and led to our successful experimentation with oxytocin and a publication of a case report in the JCEM https://www.ncbi.nlm.nih.gov/pubmed/29220529. On the topic of oxytocin's effects on appetite reduction, energy metabolism, weight loss, lipolysis, etc., there exist a good number of basic science (animal model) research, some preclinical studies, but insufficient drug trial studies as of yet. There have been some clinical studies in the PWS population and currently, there are a couple of ongoing oxytocin and obesity studies now (for children at CHOP https://clinicaltrials.gov/ct2/show/NCT02849743 and for adults at Mass General https://clinicaltrials.gov/ct2/show/NCT03043053). We eagerly await the results with baited breath and hope that the results they find will provide more help to sufferers. Please see post #8 in this blog for a selected and non-exhaustive list of some pertinent published papers.
Since I have begun this oxytocin experimentation and blog, I believe that it has given others hope for a condition whose treatment options are poor to nonexistent. Many parents and patients have told me that they have asked for a prescription from their endocrinologists but have been turned down. Most endocrinologists cite "lack of research" but many appear not to know about the important role of this missing hormone. It's no wonder there is ignorance among endocrinologists since it is not replaced as a standard of care for hypopituitarism and not even mentioned in the Endocrine Society's Clinical Practice Guideline for Hormone Replacement in Adults. https://academic.oup.com/jcem/article/101/11/3888/2764912. I find it both perplexing and maddening that untreated symptoms (obesity/hyperphagia, social impairment, among others) which correspond almost perfectly with the functioning role of a missing hormone (oxytocin), are totally overlooked by clinical endocrinology in the care for craniopharyngioma patients and others with hypothalamic obesity and hyperphagia.
Although my words are shared here in this blog, I know that other families are also crying out for help and feeling largely unheard. The families I know who have asked their doctors for help often feel that no help is offered because, well, evidence-based treatment options are not readily available for this condition. When they receive the "no" to their request for an experimental agent like oxytocin, they are told that "there is insufficient research" or "we don't yet know about the safety or long term effects." I understand the need for more research and I understand the needs for precaution; my sense is that most physicians do not understand that patients and their families are living RIGHT NOW without safety and without health. To be asked to wait into the future for safe, effective and tested treatment is akin to telling the starving peasants, "let them eat cake."
I ask you, as a physician and a parent, to consider some of the real HO and hyperphagia cases (due to craniopharyngioma). The four cases below contain actual details of real cases but the identifying information (gender, age) has been changed.
Imagine that this was your own child:
1. You lock up all of your food for fear your child will eat continuously due to his hyperphagia. Even small kitchen appliances are locked up (such as can openers) lest he find food left out. One day, a can of beans is accidentally left out. Your child finds the can of beans, attempts to open it by smashing the top against the sharp corner of the kitchen counter, pierces a hole in the top, pries the rest of the can open with his bare hands, and cuts his hands deep enough in the process to require stitches. Every last bean was eaten before his cut hand was discovered. When you ask him why he hurt himself so badly over it, he says simply, "because I was hungry, mommy." You ask him to promise you that he will never do it again. He looks down and shakes his head but cannot make the promise.
2. You lock up all of your food for fear your child will eat continuously due to her hyperphagia. Your child is obese and gaining weight steadily despite a very stringent and healthy diet. One night, she successfully picks open the lock on the freezer and you wake up to find her gnawing on a frozen and raw chicken breast. You are also required to lock up garbage to be mindful of the food waste that is thrown away for fear that it will also attract your hungry child.
3. Your young teenager has suffered for most of his life with hyperphagia and obesity and is finally taken out of public school because his school continues to fail in keeping him safe from food. Too many parties at school, tossed food in the garbage cans, and unwatched lunches belonging to other children, are strategically and stealthily snuck by your child. He has stolen money from you and has stolen food from stores. You are required to quit your job so that you can stay home to home school your child. The loss of income and the stress on your family is felt by all members of the family. Your marriage and mental health suffer. Your teen reports that he is suicidal over the loss of a normal life.
4. Your child suffers from rapid weight gain but does not have an excessive appetite. Still, no matter how strict her diet, she cannot lose weight. She has become so obese that she cannot find clothes that fit her properly. Her obesity has caused her to have difficulties walking. She comes home from school crying every day because children are cruel. She eats lunch alone in the bathroom to avoid their tormenting stares. She is bright but her grades are suffering because she has become increasingly avoidant and often refuses to go to school. She tells you that she cannot face the children at school because she is "fat and ugly." Your child is only 10 years old and you are terrified of how much worse it will be when she becomes an adolescent.
What would you do if your child experienced what I described above? If nothing exists now to treat hyperphagia, would you wait for medical science to find the effective treatments and for FDA approval? Or would you hasten medical science and consider alternative treatments, even if novel or experimental?
Most parents I know who are living through these gut wrenching situations are willing to do anything to save their children from the hellish circumstances that accompany HO and hyperphagia. I know that scientific inquiry takes time and that it is important to investigate the efficacy and safety of drug treatments. I don't expect a physician to apply treatment to a patient without knowing the safety, side effect, and therapeutic efficacy profiles of the drug. I only ask that when sitting with your patients and their families, please be sensitive when refusing a novel treatment because "there's insufficient research to ascertain safety and efficacy" when your patient's currently lived experience is already the farthest thing from safe or effective. Out of respect to the families, please consider what you would do if it were your child suffering before you dismiss their request for a novel intervention such as oxytocin. If you are not conversant on the existing research, please read it and consider the pros and cons on prescribing versus withholding treatment.
Thank you.
Well said Eugenie! Thank you!
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