Featured Research

from universities, journals, and other organizations

New Potential Therapy For Sickle Cell Disease; Inhaled Gas May Offer A Novel Treatment Approach

Date:
March 5, 2003
Source:
Children's Hospital Boston
Summary:
A study from Children's Hospital Boston gives intriguing preliminary evidence that inhaling the gas nitric oxide may relieve the vaso-occlusive pain crises suffered by patients with sickle-cell disease.

Boston -- A study from Children's Hospital Boston gives intriguing preliminary evidence that inhaling the gas nitric oxide may relieve the vaso-occlusive pain crises suffered by patients with sickle-cell disease. Appearing in the March 5 edition of JAMA, this is the first published study of inhaled nitric oxide for treatment of these intensely painful, debilitating episodes in children. Pain crises often begin in infancy, and by adolescence account for 90 percent of hospitalizations in children with sickle-cell disease. Over time, vaso-occlusion (blood vessel blockage) can lead to chronic, multi-system organ damage and ultimately shorten a patient's life.

Current treatments (analgesics, fluids) are directed at easing the symptoms of vaso-occlusive crisis and are of only limited effectiveness, said Debra Weiner, MD, PhD, assistant in Emergency Medicine at Children's Hospital Boston and the study's leader. Rather than targeting symptoms, Weiner and her team, including senior investigator and director of Children's Hematology Lab, Carlo Brugnara, MD, were interested in targeting disease processes that underlie vaso-occlusion itself. A key factor in vaso-occlusion is now thought to be a shortage of nitric oxide, a gas produced by cells throughout the body. Nitric oxide dilates blood vessels to help maintain good blood flow, prevents abnormal clotting, and controls inflammation.

The study is based on the idea that providing extra nitric oxide could compensate for this shortage. In a randomized clinical trial, the Children's Hospital Boston team gave 20 young patients with severe vaso-occlusive crisis, aged 10 to 21, either nitric oxide or placebo (room air only) through a face mask for four hours. All patients received routine treatment with fluids and morphine. Respiratory therapists gave treatments; neither Weiner nor the patient knew which treatment was being given. Patients scored their degree of pain on a validated visual scale going from 0 to 10 centimeters. All patients were carefully monitored.

Overall, the 10 patients receiving nitric oxide had a greater decrease in hourly pain scores as compared with the placebo group. This difference was near statistical significance at 3 hours and achieved statistical significance when pain was looked at over the full 4 hours of inhalation. The nitric oxide group also used about a third less morphine over six hours, showed a trend toward shorter hospitalization, and scored well on all measures of safety.

Weiner emphasized that the study was small and that the gas was given for only four hours, which probably was not long enough for nitric oxide to be fully effective. "The fact that we saw differences under these circumstances is very encouraging," Weiner said. "If nitric oxide works, it will be a long-overdue breakthrough for these patients."

Nitric oxide is already used, sometimes at home, to treat respiratory disease and pulmonary hypertension. It is also used in cardiac and surgical settings to prevent reperfusion injury (cellular injury that occurs when blocked blood flow is restored).

Sickle-cell disease affects millions of people worldwide and about 70,000 Americans, primarily people of African descent. About 8 percent of African Americans are carriers, and 1 in 500 African-American newborns have the disease, making it the most common genetic disease in this population.

Although the genetic defect has been known for 50 years, research is still unraveling the complex pathophysiology of sickle-cell disease. The defective gene results in production of abnormal hemoglobin, the oxygen-carrying protein in red blood cells. In low-oxygen conditions, the defective hemoglobin molecules bind together, causing the cell's shape to distort. This "sickling" results in impaired circulation and inadequate delivery of oxygen to tissues. Sickled cells are more easily destroyed than normally shaped red blood cells, and recent work from the National Institutes of Health found that free hemoglobin, released when the cells are destroyed, rapidly scavenges and destroys nitric oxide, making it unavailable for use by the body.

"The idea is that if you give ample nitric oxide to overcome this deficiency, and give it for long enough, you may be able to reverse or stop the vaso-occlusive crisis," Weiner said. "We don't know that nitric oxide works yet, but the results are encouraging and warrant further investigation." Her team plans further studies.

###

The Children's Hospital Boston study was funded by an Orphan Products Development grant from the Food and Drug Administration and by the Children's Hospital Boston's General Clinical Research Center National Institutes of Health grant. Pulmonox Medical Corporation (Tofield, Alberta, Canada) donated the nitric oxide.

Children's Hospital Boston is home to the world's largest research enterprise based at a pediatric medical center, where its discoveries have benefited both children and adults for over 100 years. More than 500 scientists, including seven members of the National Academy of Sciences, nine members of the Institute of Medicine and nine members of the Howard Hughes Medical Institute comprise Children's research community. Founded in 1869 as a 20-bed hospital for children, Children's Hospital Boston today it is a 300-bed comprehensive center for pediatric and adolescent health care grounded in the values of excellence in patient care and sensitivity to the complex needs and diversity of children and families. It is also the primary pediatric teaching affiliate of Harvard Medical School. For more information about the hospital visit: http://www.childrenshospital.org .


Story Source:

The above story is based on materials provided by Children's Hospital Boston. Note: Materials may be edited for content and length.


Cite This Page:

Children's Hospital Boston. "New Potential Therapy For Sickle Cell Disease; Inhaled Gas May Offer A Novel Treatment Approach." ScienceDaily. ScienceDaily, 5 March 2003. <www.sciencedaily.com/releases/2003/03/030305081242.htm>.
Children's Hospital Boston. (2003, March 5). New Potential Therapy For Sickle Cell Disease; Inhaled Gas May Offer A Novel Treatment Approach. ScienceDaily. Retrieved July 31, 2014 from www.sciencedaily.com/releases/2003/03/030305081242.htm
Children's Hospital Boston. "New Potential Therapy For Sickle Cell Disease; Inhaled Gas May Offer A Novel Treatment Approach." ScienceDaily. www.sciencedaily.com/releases/2003/03/030305081242.htm (accessed July 31, 2014).

Share This




More Health & Medicine News

Thursday, July 31, 2014

Featured Research

from universities, journals, and other organizations


Featured Videos

from AP, Reuters, AFP, and other news services

Health Insurers' Profits Slide

Health Insurers' Profits Slide

Reuters - Business Video Online (July 30, 2014) Obamacare-related costs were said to be behind the profit plunge at Wellpoint and Humana, but Wellpoint sees the new exchanges boosting its earnings for the full year. Fred Katayama reports. Video provided by Reuters
Powered by NewsLook.com
Concern Grows Over Worsening Ebola Crisis

Concern Grows Over Worsening Ebola Crisis

AFP (July 30, 2014) Pan-African airline ASKY has suspended all flights to and from the capitals of Liberia and Sierra Leone amid the worsening Ebola health crisis, which has so far caused 672 deaths in Guinea, Liberia and Sierra Leone. Duration: 00:43 Video provided by AFP
Powered by NewsLook.com
At Least 20 Chikungunya Cases in New Jersey

At Least 20 Chikungunya Cases in New Jersey

AP (July 30, 2014) At least 20 New Jersey residents have tested positive for chikungunya, a mosquito-borne virus that has spread through the Caribbean. (July 30) Video provided by AP
Powered by NewsLook.com
Xtreme Eating: Your Daily Caloric Intake All On One Plate

Xtreme Eating: Your Daily Caloric Intake All On One Plate

Newsy (July 30, 2014) The Center for Science in the Public Interest released its 2014 list of single meals with whopping calorie counts. Video provided by Newsy
Powered by NewsLook.com

Search ScienceDaily

Number of stories in archives: 140,361

Find with keyword(s):
Enter a keyword or phrase to search ScienceDaily for related topics and research stories.

Save/Print:
Share:

Breaking News:
from the past week

In Other News

... from NewsDaily.com

Science News

Health News

    Environment News

    Technology News



    Save/Print:
    Share:

    Free Subscriptions


    Get the latest science news with ScienceDaily's free email newsletters, updated daily and weekly. Or view hourly updated newsfeeds in your RSS reader:

    Get Social & Mobile


    Keep up to date with the latest news from ScienceDaily via social networks and mobile apps:

    Have Feedback?


    Tell us what you think of ScienceDaily -- we welcome both positive and negative comments. Have any problems using the site? Questions?
    Mobile: iPhone Android Web
    Follow: Facebook Twitter Google+
    Subscribe: RSS Feeds Email Newsletters
    Latest Headlines Health & Medicine Mind & Brain Space & Time Matter & Energy Computers & Math Plants & Animals Earth & Climate Fossils & Ruins