CLEVELAND, Ohio (Ivanhoe Newswire) - It's not magic, it's medicine that's making tumors disappear in babies. Now, an old drug is being used in a new way to make these tumors go away.
Ten percent of newborns will develop hemangioma, a benign tumor, like Myla Hershey. Neither her parents nor her doctors noticed anything at first.
"When she was born we couldn't see it at all," Jennifer Hershey, Myla's mom, told Ivanhoe.
However, at three months, "it was like a big slab of meat on her forehead and it was actually weighing her eyebrow down," Jennifer said.
Until now, doctors would treat the tumors with steroids, chemotherapy, and surgery, but doctors in France were treating a heart patient with a common beta blocker. The side effect was that it also caused the child's hemangioma to fade away.
"It affects these infantile hemangiomas by killing the hemangioma cells and restricting the blood vessels that feed them," Alex Golden, MD, Pediatric Cardiologist at Cleveland Clinic Children's Hospital, told Ivanhoe.
Dr. Golden is now using it to treat babies in the United States.
"The day we start the medication, the hemangiomas stop growing," Dr. Golden said.
Myla took three liquid doses of the beta blocker a day and within just a few days, her mom started to see the tumor disappear.
"It started to fade first, get lighter, and to shrink," Jennifer said.Submit
Now, after a year on the medicine Myla has just a little reminder left and doctors said it will not come back.
Right now, doctors are only using the beta blockers on infants; 100-percent of the tumors in the last 75 patients stopped growing or got smaller. Doctors don't yet know if the treatment will have the same effect on older children and adults.
BACKGROUND: A birth mark that looks like a nodule of extra blood vessels in the skin or a bright red patch is called a hemangioma. It grows during a baby's first year of life and recedes over time. It starts out as a flat red mark; it can be present anywhere on the body, but is most often found on the face, scalp, or back of the neck. During the child's first year, the red mark will become a spongy mass that protrudes from the skin. It will grow rapidly at first to two to three inches in diameter. Then, it will stop growing and enters a rest phase. Eventually, it will begin to slowly disappear. A child usually only has one mark, but some children have more than one, especially if they are a twin. It is usually benign and is not associated with other medical conditions. For most cases, the hemangioma doesn't require treatment. When a child who had it reaches the age of ten, there is usually little visible trace of the birthmark. (Source:www.mayoclinic.com)
TREATMENT: Most hemangiomas never need any form of treatment. However, treatment can be controversial. Some parents feel that treatment is necessary because the marks can be disfiguring and may cause psychological or social problems. Doctors may be hesitant to treat a hemangioma that is not causing physical problems because they usually fade gradually without treatment, and because it could have potential side effects. If the growth interferes with a child's vision or causes other problems, treatment options may include laser surgery or corticosteroid medications. Lasers can stop its growth. However, the risks can include infection, bleeding, scarring, pain, and changes in skin color. Corticosteroids can be given by mouth, injected, or applied to the skin. They are used to stop the growth of the hemangioma and sometimes reverse it. The risks can include high blood sugar, high blood pressure, poor growth, and clouding of the normally clear lens of the eye. Some experimental treatments include interferon alfa, beta blockers, and topical immune suppressants. (Source: www.mayoclinic.com)
NEW TECHNOLOGY: French researchers at Bordeaux Children's Hospital in France were the first to discover that propranolol (a blood pressure medication from a class of medication called beta blockers) can be used to treat hemangiomas. They were using the blood pressure medication to treat heart conditions in two infants who also had a hemangioma. They said they noticed that the size and color of the hemangioma was reduced within a day in both babies. "After informed consent was obtained from the parents, propranolol was given to nine additional patients who had severe or disfiguring infant hemangiomas. In all cases, 24 hours after the onset of treatment, we observed a change in color from intense red to purple, associated with a palpable softening of the lesion," Dr. Christine Leaute-Labreze, from the department of Pediatric Dermatology at Bordeaux Children's Hospital in France, was quoted as saying. (Source:http://health.usnews.com/health-news/family-health/articles/2008/06/11/beta-blocker-may-shrink-infant-hemangiomas)
Dr. Alex Golden, Pediatric Cardiologist and Staff Member of the Cleveland Clinic Children's Hospital, is now using this method to treat babies in the United States. He has seen 100 percent success rates. There have not been any side effects to the patients who are on the medication. The medication is a liquid and is taken two to three times a day. Results are typically seen in a couple of days, but Dr. Golden says as soon as they start medication, the hemangiomas stop growing. In one to two weeks the tumor begins to shrink. After 12 weeks, it is completely shrunk. (Source: www.clevelandclinic.org)
Alex Golden, MD, Pediatric Cardiologist and staff physician at the Cleveland Clinic Children's Hospital, talks about vascular anomalies in children and a new treatment that could help them without serious side effects.
We are talking about tumors that appear in infants, right?
Dr. Golden: Yes, these are benign tumors; they are not malignant. It is not cancer, but they are growths that start out either very tiny or not present at all when the child is born and then grow to be variable in size, but can be quite large or damaging depending on the location.
What is the proper name of them?
Dr. Golden: Infantile hemangioma.
What I thought was amazing with Myla is there was nothing there when she was born. Then within a couple of weeks, it was the size of a golf ball; correct?
Dr. Golden: Yes. When they start growing, they grow very rapidly and it can be quite scary for parents.
What causes it?
Dr. Golden: We still do not know exactly what causes the infantile hemangiomas. There are multiple theories, but none of them has emerged as the correct answer.
What happens if it goes untreated?
Dr. Golden: Some of them stop growing on their own fairly early, but the ones that get larger or are in very difficult or dangerous places, particularly on the face or other areas, can cause quite a bit of destruction in the meantime. They will grow for a period of time, usually somewhere before the first birthday, at which point they stop and then can start to slowly go away, but there is a pretty significant subset of them that do not go away completely and that is where the propranolol treatment comes in.
You said it causes damage?
Dr. Golden: It can. For instance, a hemangioma on the nose can destroy the nasal tissues and cause deformation of those that could be permanent. There are infantile hemangiomas that occur near the eyes and can affect vision. If it is near the airway it can affect breathing and obviously, as in many cases that we have seen it can be cosmetically a very important problem for kids too.
Jaime has a friend whose baby had one and it was in the same place as Myla. Is that a very common place?
Dr. Golden: It is quite common and in fact, it is even much more common in premature babies. So with younger and younger deliveries and our neonatal intensive care units getting better at taking care of little premature babies, we see more hemangiomas coming from that population as well.
Can babies be born with this lesion too?
Dr. Golden: Sometimes there is some evidence when the baby is born. Usually it is tiny or almost nothing is there at birth and then really starts to take off within the first month or two of life and some of them can grow extremely rapidly.
Is there a reason why they grow so much more after birth?
Dr. Golden: I do not know the answer to that and that is not a completely known fact. When the current treatment for this, propranolol, was discovered, it was really by accident. Some doctors in Bordeaux, France serendipitously found out that it worked on hemangiomas when they were using it to treat one child with heart disease who happened to have a hemangioma as well and found that the hemangioma went away. They tried it again and pretty soon they had done a series of this, published it, and the rest of the world found out about it in 2009.
Traditionally you would treat this with radiation and steroids?
Dr. Golden: Steroids is the mainstay treatment, yes. In the past there have been chemotherapy agents that have been used sometimes, but up until this time the mainstay treatment has been high dose oral steroid therapy which has many risks.
As a parent I would think that is the last thing I want to put into my newborn is high dose steroids.
Dr. Golden: Yes, that is right.
What could be the risks for that?
Dr. Golden: There are all sorts of dysregulation of endocrine systems and hormone production. There are weight changes, weight to fat distribution changes, high blood pressure, and heart disease as a result of the high blood pressure among other problems.
So by treating this traditionally, you could be affecting your child's life forever?
Dr. Golden: Absolutely, and we find it is not as effective as the beta blockade, which is the propranolol treatment we are using now.
So the beta blockade, can you explain that?
Dr. Golden: The name of the medication that we use for these children is propranolol and it is in the beta blocker class of medications. It is what is called a nonselective beta blocker and it affects these infantile hemangiomas by killing the hemangioma cells and also by cinching down and constricting the blood vessels that feed them. We do not know all of the answers about how the medication accomplishes this, but we certainly observe that in basically 100% of these infantile hemangiomas that the day we start the medication, the hemangioma stops growing and begins to shrink quite rapidly.
This is a medication that has been used for decades, mostly in adults but in many children as well, and it has many properties. It blocks beta receptors in the body and by doing that it can lower blood pressure, lower heart rate, and help with cardiac arrhythmias; so most of the patients in the world who receive propranolol get it for those indications. In children we use it a fair amount for rhythm problems, for electrical abnormalities, and heart short circuit type rhythm problems but now this new indication for propranolol has emerged which has really been amazing.
So by giving a child this, does it affect their heart at all?
Dr. Golden: We sometimes see a little bit of lowering of the heart rate and the maximum blood pressure that a child will achieve or maximum heart rate, but essentially we have seen in about 75 patients that we have treated here at the Cleveland Clinic Children's Hospital no important side effects that have caused us to need to stop the medication. The medication can also lower the blood sugar a little bit and we do some careful teaching with families to explain to them to give feedings around the same time as the medication to keep the blood sugar up.
Are there any other risks?
Dr. Golden: No. We do not think there are any long term risks of the medication. We have been using it in children for multiple decades already and know it to be a very safe drug. Now we find that it is amazingly effective, much more effective than the steroids we had been using previously, to treat infantile hemangiomas.
Of the 75 kids you treated, 100 percent have gone away?
Dr. Golden: One hundred percent have stopped growing and 100 percent have improved with treatment, but there is some variability. Some children are much more responsive. There have been patients who when we treat them, they come back in a few weeks and I am really blown away by the shrinkage of the hemangioma. There are others that take more time, but we generally will treat for several months at least and usually past the first birthday, compared to when we initiated treatment, we have seen very significant shrinkage in basically 100% of our patients.
Is Myla rare? Is it rare to have that great of a result?
Dr. Golden: No, not rare. I definitely would put her in a category of excellent responders, but maybe 25 percent or so of our patients have been sort of along her lines.
For Myla, it was just an oral medicine twice a day?
Dr. Golden: Yes. We started out at 3 times a day depending on the age of the patient. We like to meet kids when they are young and small when the hemangioma has not had a lot of time to proliferate and grow very significantly because we think we get a better result if we start early. So if I meet them when they are fairly young, we will start them at 3 times a day dosing. Once they pass the 6 month mark and have a little more meat on their bones, then we will change it to twice a day dosing and that helps a lot with convenience for the family as well.
She was on it for a year; is that normal?
Dr. Golden: Basically it depends on how early we meet them, but we will treat from the time that we meet the children to past the first birthday generally; usually to about the 14 month of age point.
Would it work on adults?
Dr. Golden: No, this is treatment for infantile hemangiomas only at this point and I think we may find in time that research will show there are other patient populations that the medication is effective for. There are other subtypes of hemangiomas that some people have tried using it for, but we do not have enough information yet to know if it will be effective for those other patients. This is a particular treatment for a particular group of patients who come as babies and have these rapidly growing hemangiomas that we really want to stop in their tracks and prevent the damage we know can occur when they grow quickly.
Will Myla's grow back? Is there a risk?
Dr. Golden: No. It would be extremely unusual for it to grow back and the life history of these infantile hemangiomas is it will be not present basically at birth, will crop up at some point, usually in the first couple of months, and grow very rapidly. It will stop growing at about somewhere between 6 and 12 months of age, it is kind of variable, and then will then begin to go away. We know that there is a large fraction of these patients where the hemangioma will not go away completely on its own and even when they do, it takes actually many years for the infantile hemangioma to recede on its own. So the propranolol really accelerates that process and most importantly stops the growth during the growth phase to prevent further damage.
FOR MORE INFORMATION, PLEASE CONTACT:
Alex Golden, MD Pediatric Cardiologist Staff Member Cleveland Clinic Children's Hospital 216-445-7116
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