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June 25, 2008 (芝加哥) — 一篇新研究显示，基因确认DYT1 张力失常的26位病患，进行苍白球脑部深层刺激术(DBS)之后，其动作征兆获得至少3年的改善，有2位病患的改善甚至长达10年。
主要作者、法国Montpellier大学医学院与Gui de Chauliac医院的Laura Cif博士表示，结果显示，治疗后1年的改善和治疗后第3、5、6年的结果并无显著不同。
Cif博士向Medscape Neurology & Neurosurgery表示，我们的结果显示，DBS对于DYT1张力失常病患有持续的效果；相较于其他文献，追踪10年是很长的时间。
她在由动作异常学会赞助的巴金森氏症与动作异常第12届国际研讨会中发表研究结果。
【改善动作技巧】
根据作者发表的壁报，在手术植入电极之前，病患的Burke-Fahn-Marsden肌张力不全失能评量表 (Dystonia Rating Scale)平均动作分数为57.51(分数120表示最严重)，在术后3年和5年显著降低到15.15 (P < .0001)和6.25 (P < .0001)。
持续参与研究的26名病患(16名女性与10名男性)，平均发作年纪为8.1岁，平均手术年纪为21.1岁；一名病患为成人，手术前的平均张力失常身体部位为6.2，Cif 博士表示这属于全身型。
Cif 博士表示，所有的病患最初有双侧单一电极植入于苍白球内核(internal globus pallidus)后侧，没有人需要重新放置；她解释，如果稍后要放置第2对电极，下肢为主要患部的病患，放置在原本植入物的前方，若是上肢，就放在原本植入物的后方；她向Medscape表示，没有病患停止治疗，但是有2人在骨折后需要重放。
Cif 博士表示，在术后6和12个月评估肌张力不全失能评量表的动作与失能分数，之后每年评估，直到10年；两位神经科医师对这些病患进行检查，通常以非盲目方式进行。
【张力失常改善】
作者发现，虽然长期以DBS治疗，但张力失常改善缓慢。
Cif 博士在访问中表示，即使我们在这些病患身上看到明显的改善，但在我们关掉神经刺激器时可以发现仍有一些潜在疾病有一些恶化，有些一开始受到DBS完整控制的动作征兆复发。
有些病患在术后4年出现疾病恶化，但她指出，对于整体病患族群来说，失去的效果没有统计上的显著意义。
不过，因为最初的不完全反应(< 50%)或者后续的张力失常恶化，其中有8名病患需要植入第2对电极(平均在第1次手术之后5年)；这几位病患和其他18位只接受1对电极的病患相比较，5年时，恶化的动作分数(P = .01)和失能分数(P = .025)都显著较高；此外，这8位病患中仅4位在植入第2对电极之后获得改善。
【并非治愈】
尽管如此，这项结果依旧令一名听众感到鼓舞，克里夫兰大学医院Case医学中心DBS计划主任、Benjamin Walter医师表示，DBS虽不是治愈，但是长期治疗效果并未显著减少这一点，的确相当令人振奋。
他建议，进行一个盲目独立神经学评估，来帮助确认此研究的效果；不过，他表示，回顾作者的研究对象在术前、术后的动作影片之后，的确有戏剧性的改善。
Cif 医师和Walter医师都表示没有相关资金上的往来。
巴金森氏症与动作异常第12届国际研讨会：摘要484。发表于2008年6月24日。
Study Shows Deep-Brain Stimulation Effectively Treats Generalized Dystonia Long Term
By Kathleen Louden
Medscape Medical News
June 25, 2008 (Chicago, Illinois) — Twenty-six patients with genetically confirmed DYT1 dystonia had improvement in their motor signs with pallidal deep-brain stimulation (DBS) for at least 3 years and for as long as 10 years in 2 of the patients, a new study showed.
Results showing improvement at 1 year postoperatively did not significantly differ from outcomes at 3, 5, and 6 years after the start of treatment, said lead author Laura Cif, MD, PhD, from the Hospital Gui de Chauliac and the University of Montpellier School of Medicine, in France.
"Our results show there is a persistent efficacy of DBS in patients with DYT1 dystonia. The 10-year follow-up is a long time compared with other reports in the literature," Dr. Cif told Medscape Neurology & Neurosurgery.
She presented the results here at the 12th International Congress of Parkinson’s Disease and Movement Disorders, sponsored by the Movement Disorder Society.
Improved motor skills
Before surgery to implant the electrodes, the patients' mean motor score on the Burke-Fahn-Marsden Dystonia Rating Scale was 57.51 (with a score of 120 being most severe), according to the authors' poster presentation. There was a statistically significant decrease postoperatively to 15.15 (P < .0001) at 3 years and 16.25 (P < .0001) at 5 years.
The 26 consecutively recruited patients (16 females and 10 males) had a mean age at onset of disease of 8.1 years and a mean age at surgery of 21.1 years. One patient was an older adult. The mean number of body regions affected by dystonia before surgery was 6.2, indicating generalized disease, Dr. Cif said.
All patients initially had a bilateral single electrode lead implanted in the posterior lateral aspect of the internal globus pallidus, and no repositioning was required, Dr. Cif noted. If a second electrode pair was implanted later, she explained, it was placed in front of the original implant if the patient's lower limbs were primarily involved and behind the initial lead if the arms were affected. No patient had to discontinue stimulation, but 2 leads had to be replaced after they fractured, she told Medscape.
Motor and disability scores on the Dystonia Rating Scale were evaluated preoperatively, at 6 and 12 months postoperatively, and every year after that for up to 10 years. Two neurologists examined the patients, usually in a nonblinded fashion, Dr. Cif said.
Progression of dystonia
Despite long-term treatment with DBS, the dystonia slowly progressed, however, the authors found.
"Even if we have a significant improvement in these patients, there is some progression of the underlying disease, which we can see when we turn off the neurostimulator," Dr. Cif said in an interview. "Sometimes motor signs that were completely controlled under DBS recur."
Some patients had worsening of their disease about 4 years postoperatively, but she added that the loss of efficacy was not statistically significant for the whole patient population.
A subgroup of 8 patients, however, required implantation of a second pair of electrode leads (at a mean of 5 years after the first surgery) because of initial incomplete response (< 50%) or subsequent worsening of dystonia. This group, compared with the 18 patients who received only a single pair of electrodes, had significantly worse motor scores (P = .01) and disability scores (P = .025) at 5 years, results showed. In addition, only 4 of the 8 patients improved after receiving their second electrode pair.
"Not a cure"
Still, the results were encouraging to 1 attendee who reviewed the poster presentation. "DBS is not a cure, but a long-term therapy whose efficacy doesn't wane significantly is very exciting," said Benjamin Walter, MD, medical director of the DBS program at University Hospitals Case Medical Center, in Cleveland, Ohio.
A blinded, independent neurologic evaluation would have added to the validity of the study results, he commented. However, after viewing the authors' videotape of study patients moving before and after surgery, he said, "There's still dramatic improvement."
Neither Dr. Cif nor Dr. Walter reported any relevant financial disclosures.
12th International Congress of Parkinson’s Disease and Movement Disorders: Abstract 484. Presented June 24, 2008.