Childhood Endocrine Tumors - The Basic

MD Anderson Cancer Center
Date: 08/15/2011

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Lisa Garvin: Welcome to Cancer Newsline, a weekly Podcast series from the University of Texas, MD Anderson Cancer Center. Cancer Newsline helps you stay current with the news on cancer research, diagnosis, treatment, and prevention, providing the latest information or reducing your family's cancer risk. I'm you're host, Lisa Garvin. And today, we're going to be talking about endocrine tumors in children and our guest today is associate professor and deputy chair of the Endocrine Neoplasia and Hormonal Disorder's Department at MD Anderson, Dr. Steven Waguespack, welcome.

Dr. Steven Waguespack:  Thank you Lisa. It's a pleasure to be here.

Lisa Garvin:  Let's talk about, what are endocrine tumors.

Dr. Steven Waguespack:  Well, endocrine tumors can be either benign or malignant growths that occur in any of the endocrine organs in the body ranging from the pituitary to the thyroids and parathyroid glands; as well as the adrenal glands; and the pancreas, the endocrine cells within the pancreas. We tend not to consider endocrine tumors in the same area as like germ cell tumors and tumors that arise in the gonads, although theoretically those could be endocrine tumors. That's not currently what we focus on.

Lisa Garvin: So endocrine glands are basically, they produce hormones so they regulate things like growth and so forth.

Dr. Steven Waguespack:  Exactly and endocrine hormone is it's a hormone or substance that produced by a gland and then travels thought the blood stream and has an action at the distant site. And so that what an endocrine tissue does.

Lisa Garvin:  What sorts of endocrine tumors do we see in children?

Dr. Steven Waguespack:   Well, the primary tumors that we take care of our thyroid tumors. Again, these are either benign or malignant thyroid tumors and the thyroid is composed of two different populations of cells. There are the follicular thyroid cells that actually are the cells that make thyroid hormone and those are cells that concentrate iodine and those cells can give rise to benign adenomas or keloid, nodules, and in addition, it gives rise to what we called the differentiated thyroid carcinomas, the major one being papillary thyroid carcinoma in childhood. And then separately within the thyroid, there is another subgroup of tiny cells called C-cells that live among the thyroid follicles. And those cells can give rise to another type of cancer called medullary thyroid carcinoma. And that is typically seen within a hereditary setting in a syndrome called multiple endocrine neoplasia type two.

Lisa Garvin:  So the second most common type of endocrine tumor is the pituitary gland. And let's talk about the pituitary gland's role in development as well.

Dr. Steven Waguespack:   So, that is the second most common type of tumor we would see. And the pituitary is what's called the master gland. It sits at the base of the brain and it secretes multiple hormones that are important for growth and development as it relates to the child. And so these tumors can either be tumors that secrete excessive amounts of hormones and cause a certain clinical syndrome such as Cushing's syndrome, when there is too much cortisol; or gigantism, when there's too much growth hormone, although the most common one we would see is a prolactin hypersecreting tumor that can cause girls to stop having periods and to have breast mild production, and so forth. And then there is another type of tumor that doesn't make any hormones and those are called "nonfunctioning tumors." So we do tend to see those diagnosed in childhood, very rarely does that occur. And in addition, that actual most common tumor that affects the region of the pituitary that we also help to take care of is something called the craniopharyngioma which is actually not a pituitary derived tumor, it's a tumor that grows from a vestigial remnant of when the pituitary developed as a fetus. So that is typically what we cover when we deal with children with pituitary tumors.

Lisa Garvin:  How do patients come to you? It seems to me that it will be hard to diagnose something like this. I mean, is there often a delayed diagnosis because the symptoms aren't really clear until the child gets older?

Dr. Steven Waguespack:   Exactly, I mean this--because these are rare situations than they often--it takes years for them to fully be recognized. And so a child for example who has a functioning pituitary tumor might present with excessive weight gain and at the same time, lack of linear growth. And that's very concerning for something called the Cushing's syndrome which is usually Cushing's disease due to a pituitary tumor. So, it takes the primary care provider to actually recognize some of these subtle changes that might occur in a child that might lead to this diagnosis. What we're seeing more of these days is a lot of children are actually getting more MRIs for headaches or other neurological signs or symptoms and then incidentally discovered is something in the pituitary. That doesn't always turn out to be something real clinically relevant for the patient but that is another way that children are identified to have these lesions.

Lisa Garvin:  Now, depending on the endocrine gland involved, are the symptoms fairly different for these diseases?

Dr. Steven Waguespack:  Exactly. So, you know, for example, thyroid coming back to the most common tumor we treat. So a thyroid cancer for example would not be necessarily identified based upon pain or any symptoms the child is having. Usually, the most common route to diagnosis is a painless enlargement of the thyroid or in the youngest children, a lymph node that enlarges and never goes away and eventually it's picked up if that is a metastatic papillary carcinoma. The pituitary again varies on how that comes to diagnosis from an asymptomatic child to a severely symptomatic child who might have a large tumor that's causing headaches and visual changes.

Lisa Garvin:  I guess the age range of these tumors we--do we see them like youngest infants and through adolescents?

Dr. Steven Waguespack:   The thyroid tumors we actually have seen quite a few young children. So the youngest child we've ever taken care of here with thyroid cancer was age 2 at the time of presentation. So very unusual. If you look at the incidents of thyroid cancer in childhood, it's less than 0.5 cases per million per year in the less than age 5 age group. So usually the diagnosis is made in teenagers, it can occur in the youngest children, again presenting usually with the painless lumps in the neck. Pituitary, we can see a diagnosis of craniopharyngioma often at an earlier age when the child presents with signs of increased intracranial pressure. Pituitary tumors would be quite rare at that age group. We've had a couple but usually it's more of the teenage age group that would be picked up with that.

Lisa Garvin: What is in--Of course we can't generalized across the tumors, but generally with endocrine issues, surgery is the primary approach, is that correct?

Dr. Steven Waguespack:   Generally so. So, for pituitary tumors for example, usually surgery is considered first. There is one type of tumor; the prolactin secreting tumor where there actually is a medication that we can use that is effective in treating that without surgery. But most of the time for other functioning tumors, for tumors that are large and causing problems for the child, surgery is the optimal management approach. Thyroid cancer, thyroid tumors in general are primarily treated with surgery and for the cancer spectrum of thyroid neoplasia, surgery is the best hope for cure. Benign thyroid nodules are either treated with surgery or observation. In terms of the thyroid cancer, the differentiated thyroid carcinomas, after surgery, we use adjuvant therapy with radioactive iodine in some cases to help treat residual or metastatic disease. The other endocrine glands that we haven't mention yet, the parathyroids, if there is tumor that develops there, that is primarily surgical disease.

Lisa Garvin:  And those are in the neck, is that correct?

Dr. Steven Waguespack:   Exactly. Therefore, parathyroid glands that live kind of on either side of the thyroid and when we see that in childhood, we think of another genetic syndrome called multiple endocrine neoplasia type one, where hyperparathyroidism, excessive parathyroid production would be the earliest manifestation of that disease. In terms of the pancreas, we really don't see many endocrine tumors in the pancreas during childhood. And if we did, we think of certain syndromes like immune one again, Von Hippel-Lindau disease. The adrenal gland really are the rarest tumors, the most common adrenal tumor actually is neuroblastoma which is taken cared of by pediatric oncology. But from an endocrine standpoint, there are tumors that can arise from the outer adrenal cortex or the inner adrenal medulla, the cortex tumors are called adrenal cortical tumors, the inner tumors are called pheochromocytomas. And so those are rare, very relevant with some of the familial syndromes as well. For the cortical tumors, we would think of something called Li-Fraumeni syndrome in a child who presents with an adrenal cortical tumor which may present with early pubertal changes or Cushing's in some cases. The pheochromocytoma presents with high blood pressure and the kind of fight or flight response. And those tumors are associated primarily with Von Hippel-Lindau disease in childhood but we also consider other syndromes such as the familial paraganglioma syndromes and of course we consider MEN 2 as well although it's less likely those children present during childhood with that type of tumor. They would tend to present first with medullary thyroid carcinoma.

Lisa Garvin:  And let's talk about hereditary syndromes such Von Hippel-Lindau. These are things that are inherited. Is genetic testing--When you find this in the family, is genetic testing important?

Dr. Steven Waguespack:  Absolutely. I mean the first thing that we think of when we see a child with an endocrine tumor is why did this child get this particular tumor? For the main tumor, we see papillary carcinoma. There is no clear genetic answer for that. But for a lot of the others, there is. And so for most of the children we see, we actually get our--we have a wonderful genetic counseling group here and we have our genetic consular, see the patients and we do offer them genetic testing based upon the type of tumor, the clinical presentation as well as the family background. And we're doing some research. You know, we have looked at some of these genes for example in children with pituitary tumors to see, you know, how many of those children actually might have underlying MEN 1 and this is there early presentation of that disease.

Lisa Garvin:  I know that in some cases, you have a certain percentage chance that you're gonna pass this genetic mutation to your children. Can you quantify that with some of these tumors? Say, you know, if your father has this, you have a 50 percent chance of--have you been able to do that?

Dr. Steven Waguespack:  Exactly. So, if we can identify mutation in one of these genes that is know to cause these syndromes, then, these are dominant syndromes. Meaning there is a 50 percent chance that each child would be at risk for inheriting that gene defect. Unfortunately, lot of the tumors, we don't have in underlying genetic defect that's identified. And so that's a little harder to quantify. Of course, parents are always worried to death that, you know, when one child has, for example thyroid cancer, papillary carcinoma, what is the risk for the other children? But in general to see familial cases of papillary carcinoma, it's quite rare, usually less than 3 percent of the time. And hopefully, in the future through further research, you know, we may ultimately identify some predisposing genes.

Lisa Garvin:  And let's talk about--We were talking earlier about how endocrine tumors in and of themselves may not be fatal except in certain rare cases, but they can caused long term consequences for the child.

Dr. Steven Waguespack:   Exactly. So, you know, the most fatal endocrine tumors actually, adrenal cortical carcinoma, that fortunately again is very rare. Thyroid cancer usually has an excellent prognosis when diagnosed during childhood. And particularly in the MEN syndromes, we have ways of identifying children in a more presymptomatic nature so that we can actually act by removing the thyroid before the malignancy becomes a concern. Pituitary tumors can affect the quality of life in children. Very rarely are these tumors malignant. But certainly can impact the child in terms of growth and development because the other normal pituitary hormones can be negatively impacted by the treatment for the primary tumor. And as we had talked before the session about thyroid cancer in particular, that's a very difficult situation sometimes because we have very sensitive tumor markers that tell us that a child still has cancer cells in their bodies. But we don't--we can't readily identify it. And so these children are monitored for years with the parents knowing that they still have cancer cells. But thyroid cancer fortunately is often in an indolent disease. We're okay monitoring. We know the life expectancies quite prolonged. But that is something unique about children and thyroid cancer that doesn't quite exist for other solid tumor that are diagnosed during childhood.

Lisa Garvin: We do treat benign disease at MD Anderson, but I wonder if people would say, "Why would I need to go to a cancer center? Is it important to be seen by specialist to treat endocrine tumors?"

Dr. Steven Waguespack:  Absolutely. No one physician or team is an island unto themselves when it comes to the treatment of these tumors. And, you know, MD Anderson has a long history of multidisciplinary approaches to cancer and tumor care. And in endocrine tumors, you know, it requires the expertise of not only the endocrinologist but the surgeon who treats these tumors, the radiation specialist who will help us radiate some of these tumors, the pathologist who is well versed in the types of tumors and how to make pathological diagnosis. Not to mentioned all of the radiology specialists that help us in the diagnosis and followup of these tumors and our genetic counseling team. So, I think it's very critical and that has actually shown as it looks at surgeons that children do much better when treated by high volume surgeons, meaning the surgeons that actually do this for a living, removing the thyroid, removing the parathyroids. They have fewer complication rates. They have lower length of stay in the hospital. And so I strongly encourage, you know, whenever a child is diagnosed with endocrine tumor, the first thing I always suggest is that the parents take a deep breath and know it's gonna be okay and to spent sometime researching to find the right center with the multidisciplinary expertise to help take of their child.

Lisa Garvin:  Thank you very much for being with us today.

Dr. Steven Waguespack:  Well, thank you. It's been a pleasure.

Lisa Garvin: If you have any questions about anything you've heard today on Cancer Newsline, contact and askMDAnderson at 1-877-MDA-6789, or online at [Background music] Thank you for listening to this episode of Cancer Newsline. Tune in next week for the next podcast in our series.

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