The View from Inside: More Confusion (and Coziness) than Consent

Janet Green

(published in the American Journal of Bioethics, vol. 10, no. 9, Sept. 2010)

I call myself an accidental advocate. Although I have served on the boards of several major New York hospitals, that work grew out of my family’s history in healthcare, not out of my own medical history. I came to be a patient advocate because I was born with congenital adrenal hyperplasia (CAH). When I found out that I wasn’t the only woman of my kind, I reached out to other people who might have suffered from the same kinds of silences I experienced. I did that through an organization I cofounded, “Bodies Like Ours,” and through the CARES Foundation, a non-profit foundation dedicated to helping people with CAH.

From 2004 until 2009, I served as a member of the CARES Board of Directors. I was the first, and remain the only, adult with virilizing CAH ever to serve in that capacity. It was my hope and my goal to bring a patient’s voice to the discussions regarding the standards of experimental care directed at making CAH girls “more normal” through surgeries and prenatal dexamethasone.

Before I talk about what the authors of the target article just don’t appear to understand, I want to make sure everyone appreciates that the interest in designing our bodies and tweaking our brains does nothing to address CAH’s life-threatening components. Giving dexamethasone prenatally does nothing to cure the medical condition that is CAH.  Children treated in utero with dex are still born with CAH.

CAH results in a hormonal imbalance that can, if not properly managed, make a person critically ill or even die. The parents who have children with the “classic,” salt-losing form of CAH suffer a constant background noise of anxiety over the possibility that their children may go into adrenal crisis. What parent in such a circumstance wouldn’t? This worry makes these parents especially vulnerable to every possibility of “treatment” they are offered. Many parents have difficulty understanding that not every intervention is necessary to keep their children healthy and safe. They don’t understand, for example, that there is no evidence that a big clitoris represents any danger to a girl and I don’t believe most understand the risks involved with either surgery or dex.

To their credit, the CARES Foundation has done much to educate parents about the complexities of CAH and about treatment options. CARES helps parents navigate the challenges of communicating their needs to care providers. CARES has worked effectively to make sure that newborn screening nationwide means fewer children needlessly suffer or die from CAH.

But overly close relationships between CARES and certain endocrinologists and surgeons might mean that addressing the real needs of the patient while reducing parents’ anxiety doesn’t happen through honest and forthright discussion of issues in a way that informs and empowers parents. Instead, at CARES I saw much old school “doctor knows best” reasoning and biases that serve physicians and their careers. 

I question the club-like atmosphere of CARES, one in which the doctors on the advisory board are the same doctors CARES praises as the best and recognizes with lifetime achievement awards, while referring the membership to this very small pool of experts. Many parents do not understand that, in addition to accessing important support by coming to CARES, by coming they could also be channeled into a recruitment system for research. Yes, that research may ultimately benefit those families. But it also means that research and clinical care are getting mixed up in ways that make it hard for parents to sort out what they are getting into when they sign up with a doctor CARES strongly recommends.

For example, in one section of the CARES website, prenatal dexamethasone is discussed by Dr. Svetlana Lajic as experimental.  According to Dr. Lajic, “treatment should only be given within the frames of a clinical study in order to ascertain accurate follow-up of the mother, the fetus and the growing child.” (See http://www.caresfoundation.org/productcart/pc/prenatal_treatment_cah.html ) But on another page, written in language far more accessible to the average parent, we find what looks like CARES’ endorsement of Dr. Maria New’s clinic. Posted in 2003, this other page doesn’t make dex sound experimental at all. Parents are assured that

Dr. New maintains contact with all children treated prenatally, and has found no adverse developmental consequences. Thus, with nearly 20 years’ experience, the treatment appears to be safe for mother and child. [...] The benefits to families of classically affected girls cannot be underestimated. (see http://www.caresfoundation.org/productcart/pc/news_letter/winter02-03_page_9.htm)

I watched for many years as CARES brought in experts like Dr. New to tout particular therapies like prenatal dex, while the people worrying about these therapies’ necessity, safety, or efficacy never seemed to convey their concerns or the experimental nature of the therapies. When there was mention of the criticisms of people like Dr. Walter Miller of UCSF, the suggestion was made that these were merely personal attacks on Dr. New rather than legitimate fears about the administration of dex.  It seems clear that CARES and Dr. New did not tell these families that one set of doctors after another characterized this therapy as risky and experimental.

I have seen in these years how prenatal dex has been promoted as a way to avoid “masculinization” of girls’ brains. We all understood what that was code for. Girls with “masculine” brains might like the sorts of things boys like—including girls. A number of parents looked to Dr. New and her Cornell team to reassure them that, with dex, their girls wouldn’t end up tomboys and lesbians.

I watched Dr. New suggest that we should trust her because she is herself a mother. In one videotape of a November 14, 2001 presentation to CARES at Weill Medical College of Cornell University, we see her apparently telling the audience that she herself is a carrier for CAH.(1) The meaning is clear: I am one of you, and I would use prenatal dex. All this alliance formation and coded talk doesn’t seem to me a good atmosphere for informed consent. It feels coercive.

To make it worse, some doctors seem to be intimidated by Dr. New at the meetings I attend. They appear afraid to question her. At the international meeting held in Miami this past January, one doctor asked Dr. New about the informed consent process for her treatments of prenatal dex, and she simply refused to answer the question.(2) I could only wonder, if she is permitted to ignore her colleagues’ questions about informed consent in a professional meeting, how impossible parents might find it to get honest answers to questions about the safety of the drugs they were taking.

I was frankly shocked to learn from the authors of the target article that Dr. New now says she only wrote one prescription for prenatal dexamethasone. So why has she positioned herself for years as being the go-to person for this treatment? This looks a lot like a shell game of responsibility.

The target article also says we shouldn’t worry about dex treatment because it doesn’t start until the ninth week of pregnancy. But Dr. New has stated repeatedly, “Treatment begins as soon as pregnancy is confirmed.” (See, for example, http://www.newchf.org/testing.php.) I am also unsure why the target article’s authors think that this drug is “benign.” The authors dismiss studies that suggest serious risk, but many of the physicians with whom I’ve talked at conferences think there is plenty of reason for concern about prenatal dex.(3)

There have been no adequate checks and balances in this system to protect these women and their children, because there has been no FDA approval for this use and, so far as we can ascertain, attending physicians have not obtained IRB oversight when women have been given this drug. Dr. New apparently recruits families to her clinic through CARES, treats them, and finally tries to study them. Truly independent researchers should be called upon to study those women and children exposed to dex (including those who turned out not to be girls with CAH) so that we don’t have to rely on Dr. New to find out what the outcomes really were.

We only have to look at recent history to understand that best intentions have sometimes done much more harm than healing. I am grateful to Drs. Dreger and Feder for calling on their colleagues to finally sound an alarm about prenatal dexamethasone for CAH. I am grateful to all of the bioethicists who signed the letters of concern. I hope our government will find out what happened to these women and advise them all that they may have been part of an experiment without being told. If we cannot count on our support groups, doctors, and hospital ethics boards to protect us, then hopefully we can rely on professional bioethicists to do so. If that makes them “transgressive,” I hope a lot more of them will transgress. Someone needs to protect girls and women like me, and their mothers, too.

 

Citations:

1. 1.[Untitled presentation to CARES Foundation by Maria New at Weill Medical College of Cornell University.] Video cassette on file with author. 2001.
   

2. 2.New, M. I. 2001. Prenatal Diagnosis of Congenital Adrenal Hyperplasia: Long Range Outcome of Prenatal Treatment. At the Second World Congress on Hormonal and Genetic Basis of Sexual Differential Disorders and Hot Topics in Endocrinology, January 15-17, 2010. Eden Roc Hotel, Miami Beach, Florida.
   

3. 3.See, for example, Miller WL. Prenatal treatment of classic CAH with dexamethasone (con). Endocrine News (Tri-Point Series) 2008:16-8. See also Frias J LL, Oberfield SE, Pang S, and Silverstein J. For the AAP Ad Hoc Writing Committee, in reply to Maria New. Pediatrics 2001;107:805.