New study compares steroid regimens in boys with Duchenne Muscular Dystrophy

Duchenne muscular dystrophy (DMD) affects 1 in 3,600 boys and is caused by a mutation in the dystrophin gene, resulting in progressive muscle weakness.

Affected male children are normal at birth, but develop signs of muscle weakness before age 6, usually first affecting the legs and pelvis, causing difficulty getting up from the floor or a sitting position, and difficulty climbing stairs. Untreated boys are usually wheelchair dependent by age 12.

Ongoing research is leading towards pre-symptomatic diagnosis of DMD, and there has been some progress in genetic therapy for affected boys identified in these early stages of the disease.

However, so far corticosteroid therapy is the only treatment that has been shown to increase muscle strength in boys already affected with symptoms of DMD.

Steroids can cause side effects, and there is wide variability whether doctors doctors prescribe prednisone or deflazacort, as well as the dosing, duration of steroid use or even whether steroids are prescribed at all.

A new study aims to answer some of these questions.

The study is looking to enroll boys aged 4-7 with genetically confirmed DMD who have not taken steroids before.

They will be included in the study for 3-5 years and receive either:
1. Prednisone 0.75mg/Kg/d, or
2. Prednisone 0.75mg/Kg/d 10days on then 10 days off, or
3. Deflazacort 0.9mg/Kg/d

They will need hospital visits every 3 months for the first 6 months, then every 6 months thereafter.

This study is available locally at Penn Sate Hershey Medical Center – parents or physicians of interested patients should contact Beth Stephens at 717-531 0003 extension 283395 or by email for more information.

AAN Video – Epilepsy: A Guide for Patients and Families

Monmouth Stroke Service Success Story: Great outcome after emergent carotid endartercomy

Case presentation prepared by Drs N. Nachimuthu and M Chan, Residents, Dept of Internal Medicine, Monmouth Medical Center

Introduction:

When feasible, administration of tissue plasminogen activator (tPA) is the standard of care for treatment of acute ischemic stroke to improve outcomes. Treated patents may be found on subsequent work up to have significant stenosis of one or both carotid arteries. Carotid endarterectomy (CEA) has been shown to be more effective than medical therapy for preventing subsequent strokes in patients with symptomatic stenosis. However, the timing of CEA after ischemic stroke with or without administration of tPA remains controversial, particularly in patients with critical stenosis or unstable symptoms.

To better illustrate this dilemma, we present the case of a 43 year old male who presented with symptoms of acute stroke, was given tPA within the recommended time frame, but was subsequently found to have high grade carotid stenosis and fluctuating symptoms. We follow with a review and discussion of recent literature showing that in select cases, CEA can be done early with no increase in perioperative complications or adverse events.

Case report:

A 43 year old man presented to our Emergency Room after he was found to be restless in bed by his wife at 12:30am on the day of admission. He was also unable to express himself and was noted to have had a right sided facial droop. He was last observed to be asymptomatic 1 hour and 45 mins earlier when he was getting ready for bed at 10:45pm.

The patient arrived at the ER at 1:15am and a code stroke was immediately called. Initial examination revealed aphasia, disorientation, and right-sided facial droop, with a NIH stroke score of 5. There was no motor weakness and the rest of the neurologic exam was unremarkable. Vital signs were stable and within normal limits. A stat CT scan was done which did not show any hemorrhage or findings of ischemia:

After the CT scan, the patient initially showed some improvement in speech and orientation with the NIH stroke score dropping to 2. It seemed that treatment with tPA might not be necessary.

However, at 2am the patient’s symptoms again worsened acutely. Repeat NIH stroke score was 6 at 1:50am. tPA was given at 2:10am, 3 hours and 25 mins from last known normal.

Following tPA administration, the patient seemed to be improving again and was admitted to the ICU for close observation. However, a few hours later, at 6am, the patient again worsened. He had new right sided weakness and worsening of his aphasia and right facial droop. Given the fluctuating course of the patient’s symptoms he underwent a repeat stat CT of the head to rule out a bleed. This was negative. A CT angiogram of the head and neck was done at the same time, and this showed severe stenosis (almost total occlusion) of the left internal carotid artery:

At this point, our multidisciplinary stroke team suggested that he undergo emergent carotid endarterectomy to prevent further deterioration of his neurologic status. This was a controversial decision, but after discussing the risks and benefits of the procedure, the patient consented and a carotid endarterectomy was done urgently and completed at 12:05am on the second hospital day, or 21 hours and 55 mins from administration of tPA.

Intraoperatively, the patient was found to have left internal carotid artery narrowing secondary to hemorrhagic plaque and dissection:

The patient experienced no intraoperative complications. Post-operatively, he was started on Lovenox at 1mg/kg every 12 hours. He did develop a hematoma on the site of the CEA, and Lovenox and antiplatelets were immediately stopped. Aspirin at 325mg daily was resumed after a day and Plavix 75mg daily was resumed the next day, after the hematoma had shown signs of resolution. The patient experienced no further complications.

He underwent a repeat CT scan after surgery, more than a day from symptom onset, which did show an evolving left hemispheric stroke:

However, clinically, in the hours following CEA, his right sided weakness improved.

By the time the patient was discharged to a rehabilitation facility, he had only mild aphasia, a residual right facial droop, but no motor deficits.

Discussion:

Many vascular surgeons suggest waiting 6-8 weeks after acute stroke before considering CEA, because of fear or bleeding or extension of cerebral infarction during the surgery. However, this delay can lead to recurrent stroke or complete occlusion of the carotid artery.  Moreover, more recent studies have shown that urgent early CEA can be performed on patients with evolving symptoms without additional risks.

We feel that our patient’s near complete recovery was the direct result of early CEA, done despite the recent stroke and potential hemorrhagic complications associated with the use of tPA.

These types of complicated medical decisions can only be made after discussion between neurologists, intensivists and vascular surgeons in a multidisciplinary stroke center.

Click here to find out more about Monmouth’s Certified Stroke Center.

“Brainstorm”, Rehabilitation for Brain Tumor Patients, May 22 at 5pm

Click here to register by email

Click here to find out more about the Brainstorm seminars

Click here to find out more about Monmouth’s David S.  Zocchi Brain Tumor Center

A less invasive surgical treatment for spinal stenosis?

…..The jury is still out!….

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First, the problem:

Lumbar spinal stenosis is an abnormal narrowing (stenosis) of the spinal canal that can lead to compression of the cauda equina, resulting in pain, numbness, and weakness in the legs.

Affected patients complain of back pain, leg pain and weakness, worse with prolonged standing and walking, better with sitting or flexing the spin forward (such as pushing a supermarket trolley).

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Next, the solution(s):

This stenosis (or narrowing) is caused by degenerative bone spurs, disc disease and ligamental hypetrophy.  While many patients try conservative measures including physical therapy and epidural injections, spinal stenosis is a mechanical problem, and usually necessitates a mechanical (surgical) solution to alleviate symptoms.  Consider the analogy of a blocked sewer pipe – you can try pouring chemicals down the sink, but ultimately you know you are going to need to call a plumber  (Rota-Rooter)!

Traditional surgical management calls for a lumbar laminectomy to decompress the spinal canal and alleviate symptoms.

In some cases,  laminectomy can leave the spine unstable necessitating a spinal fusion using bone garft, rods and screws (“hardware”) done at the same time, and this major surgery can lead to an extended recovery period of months to a year.

Implantation of a interspinous spacer has been proposed as a less-invasive alternative surgical option than spinal fusion:

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Which is better?

A recent study compared the outcomes of these techniques in 99,000 Medicare patients who underwent surgery for spinal stenosis from 2006 to 2009.

6,000 patients who underwent implantation of new interspinous spacers were compared with 76,000 who underwent laminectomy, and 17,000 patients who underwent laminectomy with spinal fusion.

The results showed that patients treated with spacers had the lowest rate of major medical complications: 1.2%, compared to 1.8%with laminectomy and 3.3% with spinal fusion. Patients receiving spacers also spent less time in the hospital: average 1.4 days versus 2.7 days in the decompression group.

However, patients receiving spacers had the highest rate of revision surgery – about 17% of patients receiving spacers needed a second operation on the lumbar spine, compared to only 8.5%  in the laminectomy group and about 10% in the fusion group.

Patients receiving spacers tended to be older and to have other medical problems. With adjustment for these and other factors, patients in the spacer group were more than twice as likely to require repeat surgery.

Hospital payments for spacer surgery were greater for decompression alone, but less than for fusion procedures

Other than the need for repeat surgery (presumably because initial surgery was ineffective), the study did not look at pain control or functional outcome between the groups.  More data is needed.

Nevertheless, so far it looks as if the higher cost and higher re-operation rate with spacers may argue in favor of conventional decompression surgery, except in higher risk older patients with medical problems.

2013 House Staff Research Day at Monmouth Medical

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Save the Date!

June 6, 2013 9am-12noon

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2013 House Staff Research Day and Awards

The annual event, named in honor of Dr Ellen Cosgrove, former faculty member and director of medical education at Monmouth, is an opportunity for graduate medical trainees to present their research projects to the entire medical community at the hospital.

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Posters will be on display in the “Boardwalk” from 9am to 12noon.

Refreshments will be served!

Then, the prize winners for best research projects and posters will be announced in the Borden Auditorium at 12noon.

It’s our graduate medical education and research that sets us apart from the surrounding hospitals.

So, be sure to join us and celebrate the research effort.

A few cents on stroke prevention can save loads of $$$s in future stroke treatment costs

The US currently spends more than $51 billion a year on stroke-related healthcare.

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As we develop new treatments for acute stroke, particularly endovascular therapy, we have to recognize that these treatments are very expensive and the outcomes are still less than ideal. Even after maximal FDA approved treatment, almost 60% of stoke survivors are disabled and unable to work.

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There can be no doubt that stroke prevention is more important and cost effective than acute stroke therapy.

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We know that patients who have had a recent TIA or stroke are high risk for future major stroke.

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Here are some facts and figures that compare the costs and benefits of targeted stroke prevention vs. acute stroke interventions:

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Cost of targeted stroke prevention

1. Atrial fibrillation:
If you have had a recent TIA or stroke and have atrial fibrillation, taking an anticoagulant will reduce your future stroke risk.
Warfarin costs $37/yr
You have to treat 3 patients to prevent 1 stroke per 5 years (at a cost of $560)

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2. Anti platelet drug:
If you have had a prior TIA or stroke, simply taking an aspirin a day will reduce your future stroke risk.
Aspirin costs $5/yr
You have to treat 50 patients to prevent 1 event per 5 years (at a cost of $1250).

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2. Starting a cholesterol lowering agent:
If you have had a recent TIA or stroke, your LDL cholesterol is more than 100, and you take a cholesterol lowering agent, this will reduced your future stroke risk.
Simvastatin costs $40/yr
You have to treat 45 patients to prevent 1 stroke per 5 years (at a cost of $9000)

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3. Blood pressure:
If you have had a recent TIA or stroke, and you are started on a ACEI like ramipril, this will lower your future risk of stroke and MI.
Ramipril costs $75/yr
You have to treat 57 patients to prevent 1 stroke per 5 years (at a cost of $21,000)

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4. Carotid stenosis:
If you have had a recent TIA or stroke caused by a carotid stenosis (with 70% or more narrowing), surgery (carotid endarterectomy) will lower your future stroke risk.
A carotid endarterectomy costs $15,000
You have to treat 2.5 patients to prevent 1 stroke per 5 years (at a cost of $188,000).
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Cost of acute stroke intervention

A single dose of intravenous tissue plasminogen activator (tPA) “clot buster” used to treat acute stroke costs $2500, this is equivalent to  the cost of 75000 aspirin tabs or 25000 simvastatin tabs.

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A single use of the Merci Retrieval device for a patient with acute stroke costs $27,000, this is equivalent to the cost of 750,000 aspirin tablets, 250,000 simvastatin tabs, 10 doses of iv tPA, or 2 carotid endarterectomies.

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Conclusion?

The message here is that acute stroke treatment, costs:

Loads of Money !!

However, targeted stroke prevention for high risk patients is much more cost effective, particularly if it is provided as a well organized out-patient TIA Rapid Evaluation Center.

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In the words of Ben Franklin, an ounce (or a few cents) of prevention is worth a pound (or loads of $$s) of cure.