Sickle cell anemia drug safe and effective for infants and toddlers, adds treatment option
New research shows a drug commonly used to treat sickle cell anemia in adults reduces bouts of acute pain and a pneumonia-like illness, cuts hospitalization time and eases other symptoms of the disease in young patients. Results of the randomized, double-blind trial mark a dramatic advance in treatment of children with the inherited blood disorder.
"These results show that hydroxyurea has the potential to dramatically improve the quality of life for an entire generation of patients with sickle cell disease," said Winfred Wang, M.D., the study's principal investigator and a member of the St. Jude Children's Research Hospital Department of Hematology. He said the findings mean hydroxyurea should now be considered for treatment of all infant and toddlers with sickle cell anemia in hopes of preventing or delaying disease complications. The research will be published in the May 14 issue of the British medical journal The Lancet.
St. Jude researchers led the six-year Pediatric Hydroxyurea Phase III Clinical Trial, known as Baby HUG. The federally funded study involved 193 infants and toddlers enrolled at 13 participating U.S. medical centers and a coordinating center.
About 100,000 Americans have sickle cell anemia, a chronic disorder associated with a range of health problems, including an increased risk of strokes and premature death. Sickle cell anemia is the most common genetic disorder affecting Americans of African descent, but the disease also strikes persons of other racial and ethnic backgrounds.
Sickle cell anemia is caused by a gene mutation that leaves the red blood cells of sickle cell patients prone to assuming the stiff, crescent shape for which the disease is named. The misshapen cells can clog blood vessels, triggering pain crises, strokes and organ damage, including kidney failure. The drug works in part by increasing production of fetal hemoglobin, which counteracts the effects of the sickle hemoglobin. Fetal hemoglobin is the main hemoglobin produced by all newborns, but production normally decreases dramatically within a few months after birth.
While life expectancy for sickle cell anemia patients has improved in recent decades thanks in part to better supportive care, Wang said hydroxyurea is the first drug proven to reduce the incidence of a wide range of symptoms in extremely young sickle cell patients regardless of disease severity. The drug is inexpensive and easy to administer. The drug has been used for more than 15 years as a treatment for sickle cell disease with no evidence of serious side effects. Hydroxyurea began as a potential cancer treatment, but won U.S. Food and Drug Administration approval for use in adults with severe sickle cell disease. Baby HUG is the largest trial of hydroxyurea in much younger patients.
The Baby HUG trial was launched in 2003 after promising preliminary results regarding the drug's safety and effectiveness in extremely young children. The research included patients who were ages 9 to 18 months when they began the study. Participants were randomly assigned to receive either a standard dose of hydroxyurea or a placebo every day for two years. Neither the families nor the caregivers knew which children received hydroxyurea.
An analysis of 179 patients who completed at least 18 months of the study found children in the placebo group had nearly twice as many acute pain episodes, were three times more likely to suffer a pneumonia-like illness known as acute chest syndrome and five times more likely to develop painful swelling of the hands and feet called dactylitis. They were also slightly more likely to be hospitalized or need blood transfusions to ease sickle cell symptoms.
The most common side effect reported in this study was a mild-to-moderate drop in the white blood cells known as neutrophils, which occurred more often in children receiving hydroxyurea. Low neutrophil counts can be associated with an increased risk of infection, but there was no evidence of this in the Baby HUG trial.
Although results of kidney and spleen function tests were not significantly different between the Baby HUG treatment groups, Wang said other measures suggested that hydroxyurea might protect those organs as well as the brain and lungs from the chronic damage that leaves sickle cell anemia patients at increased risk for premature death. A follow-up study to Baby HUG is underway, focusing on possible long-term benefits from continued treatment with higher doses of hydroxyurea.
Provided by St. Jude Children's Research Hospital
- Study targets stroke prevention in children with sickle cell anemia Oct 28, 2009 | not rated yet | 0
- Two studies provide insight into stroke risk and prevention in young sickle cell anemia patients Dec 06, 2010 | not rated yet | 0
- Sickle cell disease pain can occur daily and is much more severe than previously thought Jan 14, 2008 | not rated yet | 0
- Researchers find new genetic target for sickle cell disease therapy Dec 04, 2008 | not rated yet | 0
- Stroke prevention study in children with sickle cell anemia, iron overload stopped early Jun 04, 2010 | not rated yet | 0
- Motion perception revisited: High Phi effect challenges established motion perception assumptions Apr 23, 2013 | 3 / 5 (2) | 2
- Anything you can do I can do better: Neuromolecular foundations of the superiority illusion (Update) Apr 02, 2013 | 4.5 / 5 (11) | 5
- The visual system as economist: Neural resource allocation in visual adaptation Mar 30, 2013 | 5 / 5 (2) | 9
- Separate lives: Neuronal and organismal lifespans decoupled Mar 27, 2013 | 4.9 / 5 (8) | 0
- Sizing things up: The evolutionary neurobiology of scale invariance Feb 28, 2013 | 4.8 / 5 (10) | 14
How can there be villous adenoma in colon, if there are no villi there
5 hours ago As title suggest. Thanks :smile:
How can there be a term called "intestinal metaplasia" of stomach
May 21, 2013 Hello everyone, Ok Stomach's normal epithelium is simple columnar, now in intestinal type of adenocarcinoma of stomach it undergoes "intestinal...
Pressure-volume curve: Elastic Recoil Pressure don't make sense
May 18, 2013 From pressure-volume curve of the lung and chest wall (attached photo), I don't understand why would the elastic recoil pressure of the lung is...
If you became brain-dead, would you want them to pull the plug?
May 17, 2013 I'd want the rest of me to stay alive. Sure it's a lousy way to live but it beats being all-the-way dead. Maybe if I make it 20 years they'll...
MRI bill question
May 15, 2013 Dear PFers, The hospital gave us a $12k bill for one MRI (head with contrast). The people I talked to at the hospital tell me that they do not...
Ratio of Hydrogen of Oxygen in Dessicated Animal Protein
May 13, 2013 As an experiment, for the past few months I've been consuming at least one portion of Jell-O or unflavored Knox gelatin per day. I'm 64, in very...
- More from Physics Forums - Medical Sciences
More news stories
The new 13-valent pneumococcal conjugate vaccine (PCV13) appears to be as safe as the previous version used prior to 2010, the 7-valent pneumococcal conjugate vaccine (PCV7), according to a Kaiser Permanente study published ...
Medications 2 hours ago | not rated yet | 0
The University of Gothenburg Vaccine Research Institute (GUVAX) announces successful results in a placebo controlled phase I study of an oral, inactivated Escherichia coli diarrhea vaccine.
Medications 4 hours ago | not rated yet | 0
An experimental sleeping pill from US drug company Merck is effective at helping people fall and stay asleep, according to reviewers at the US Food and Drug Administration, which could soon approve the new drug.
Medications 19 hours ago | 4.2 / 5 (5) | 0
Transparent information on the evidence supporting global recommendations on paediatric medicines should be easily accessible in order to help policy makers decides on what drugs to include in their national drug lists, according ...
Medications 20 hours ago | not rated yet | 0
Regardless of pain, social class or age, a woman is more likely to be prescribed pain-relieving drugs. A study published in Gaceta Sanitaria (Spanish health scientific journal) affirms that this phenomenon is inf ...
Medications May 21, 2013 | not rated yet | 0
Researchers from Queen Mary, University of London have led the largest sequencing study of human disease to date, investigating the genetic basis of six autoimmune diseases.
5 minutes ago | not rated yet | 0 |
Swiss scientists reveal the mechanism responsible for aging hidden deep within mitochondria—and dramatically slow it down in worms by administering antibiotics to the young.
5 minutes ago | not rated yet | 0 |
The human gut is loaded with commensal bacteria – "good" microbes that, among other functions, help the body digest food. The gastrointestinal tract contains literally trillions of such cells, and yet the ...
2 minutes ago | not rated yet | 0 |
Scientists have reversed behavioral and brain abnormalities in adult mice that resemble some features of schizophrenia by restoring normal expression to a suspect gene that is over-expressed in humans with ...
1 hour ago | not rated yet | 0 |
Researchers at Johns Hopkins have unraveled the molecular foundations of cocaine's effects on the brain, and identified a compound that blocks cravings for the drug in cocaine-addicted mice. The compound, already proven safe ...
1 hour ago | not rated yet | 0 |
University of Granada scientists have patented a new treatment for acne that is based on completely natural substances and is much more effective than artificial formulas because it does not create resistance ...
1 hour ago | not rated yet | 0