We sought to assess adherence to hydroxyurea among patients with scd and investigate associations between adherence and clinical and economic outcomes. The johns hopkins university evidencebased practice center, baltimore, md investigators. Green, md associate dean for clinical research operation associate professor of pediatrics, division of hematology columbia university medical center october 27, 2011. It was used for a wide variety of malignant conditions initially, but it has been used in sickle cell disease now for more than 25 years. Pdf update on the use of hydroxyurea therapy in sickle cell disease. We conducted a study using an electronic medication containermonitorreminder device glowcap to track adherence and determine whether use of. Sickle cell disease scd is a group of blood disorders typically inherited from a persons parents. It was first tested in sickle cell disease in 1984. Jude children s research hospital researchers report that maximizing the dose of hydroxyurea increased levels of fetal hemoglobin and reduced the odds of hospitalizations for young sickle cell anemia patients memphis, tennessee, november 9, 2017.
Strive dc is a free mentoring and advocacy program for teens that are living with sickle cell disease. Is hydroxyurea the only treatment for sickle cell disease. The nhs sickle cell and thalassaemia screening programme has a helpful parents guide to managing sickle cell disease pdf, 3. Hydroxyurea has been shown to induce the production of hbf, initially in nonhuman primates, and now in more than fifty patients with sickle cell anemia. Hydroxyurea, a myelosuppressive agent, is the only effective drug proven to reduce the frequency of painful episodes. If you have questions after reading this handbook, talk to your health care team. Hydroxyurea treatment and young children with sickle cell. However, the exact mechanism leading to the improved blood flow is incomplete. Attitudes of primary care physicians toward sickle cell. Additional mechanisms of action for hbf induction have been proposed for hydroxyurea, including free radical formation, iron chelation. Five years of experience with hydroxyurea in children and young adults with sickle cell disease.
An 18yearold woman with sickle cell anemia presents with recurrent painful crises, and treatment with hydroxyurea is recommended. In sicklecell disease it increases hemoglobin and decreases the number of attacks. Medline, embase, toxline, and the cumulative index to nursing and allied health. Hydroxyurea, a key component of care for many patients with sickle cell disease, was the drug most frequently discussed during this meeting. Department of health and human services 540 gaither road rockville, md 20850. While laboratory and clinical benefits of hydroxyurea for patients with sickle cell disease scd are well. But in over 20 years of hydroxyurea use in sickle cell disease patients, there have been no reported cases of this medicine causing cancer. Hydroxyurea was first developed as a chemotherapy medication in the 1960s. Concurrent use of folic acid decreases the risk of neural tube defects.
Hydroxyurea for the treatment of sickle cell disease ahrq. It also can reduce hospital stays for sickle cell disease. People who need a lot of blood transfusions may also need to take medicine called chelation therapy. Hydroxyurea for the treatment of sickle cell disease. The mean dose of hydroxyurea used by the patients was 12.
We partnered with community care of north carolina ccnc to. Hydroxyurea can help people with sickle cell disease live longer. Hydroxyurea is the only approved medication for the treatment of sickle cell disease in adults. Newborn screening is done on tiny samples of blood taken from your babys heel 24 to 36 hours after birth. Approval was based on data from an openlabel singlearm trial, the european sickle cell disease cohort study escort hu, nct02516579, of 405 pediatric patients with sickle cell disease from 218. Hydroxyurea for sickle cell disease american academy of pediatrics.
The sickle cell disease scd training and mentoring program stamp. Patients with sickle cell disease treated with hydroxyurea had a mean age of 25. Sickle cell disease scd is a potentially devastating condition that is caused by an autosomal. Sickle cell disease scd family fact sheet what is a positive newborn screen. The use of hydroxyurea hu can improve the clinical course of sickle cell disease. Hydroxyurea hu, a proven therapy for individuals with sickle cell disease scd, is known to improve blood flow. What are the indications for hydroxyurea in patients with. Learn about side effects, warnings, dosage, and more. Stamp is a pilot program designed to train primary care providers on the basics of scd care the goal of stamp is to identify motivated primary care providers and equip them with the appropriate. The majority of patients with sickle cell disease respond to the drug with a more than twofold increase in hbf levels. The effects of hydroxyurea on pulmonary complications in. Optimizing hydroxyurea treatment for sickle cell disease.
It usually decreases the rate of painful episodes by 50 %. Gastrointestinal tract symptoms were no more common among people who were receiving hydroxyurea for sickle cell disease than among those not receiving hydroxyurea. This leads to a rigid, sicklelike shape under certain circumstances. Hydroxyurea adherence and associated outcomes among. Hydroxyurea hu is a fda and emaapproved drug that earned an important place in the treatment of patients with severe sickle cell anemia sca by showing its efficacy in many studies.
Provider barriers to hydroxyurea use in adults with sickle cell disease. Sickle cell disease can even shorten your life but hydroxyurea can help you live longer. How i use hydroxyurea to treat young patients with sickle. Update on the use of hydroxyurea therapy in sickle cell disease trisha e. Increasing hydroxyurea dose helps to keep young sickle cell patients out of the hospital st. Nih stateofthescience conference statement on hydroxyurea treatment for sickle cell disease nih consensus and stateofthescience statements volume 25, number 1 february 2527, 2008 national institutes of health office of the director. It raises the level of hbf and the haemoglobin level. Sickle cell anemia and hydroxyurea verywell health.
Review of hydroxyurea treatment for sickle cell disease. Hydroxyurea causes a shift toward the production of red cells con. Hydroxyurea can reduce the number of painful events and acute chest syndrome episodes. Hemoglobin f augmentation is another approach to treat sickle cell disease scd. The sickle cell disease scd training and mentoring. Pdf hydroxyurea hydroxycarbamide for sickle cell disease. The journal pediatrics has published the first study to look at the costs and health outcomes costeffectiveness of hydroxyurea hu treatment in young children with sickle cell disease scd.
Sickle cell disease scd is a complex chronic disease requiring multidisciplinary care that involves primary care physicians pcps working with a hematologist or scd specialists. Sickle cell disease symptoms, diagnosis, treatment and recent developments sickle cell diseases in the present occupied and frenzied timetable, where individuals dont possess energy for thinking about their wellbeing which can make heaps of diseases them which influences their body and debilitate their wellbeing however in some cases they get going in their life in such a way, that. Hydroxyurea for the treatment of adults with sickle cell. Hydroxyurea is the only fda approved medication that has been shown to mitigate the effects of sickle cell disease and potentially prolong survival, yet it is underutilized by the patient population. People with normal hemoglobin have mostly hemoglobin a in their red blood cells. Sickle cell disease scd is a chronic genetic disorder caused. This resultant free ferrous hemoglobin likely consumes. Problems in sickle cell disease typically begin around 5 to 6. Hydroxyurea for children with sickle cell anemia in sub. Hydroxyurea is usually prescribed by a hematologist, using rigorous selection criteria. However, several features of hu treatment remain unclear, including the predictability of drug response and determination of adequate doses, considering positive responses and minimal side effects. Hydroxyurea safe and effective for sickle cell anemia global.
The quality of life of patients with sickle cell anemia is severely compromised. Hydroxyurea is only one 1 choice of treatment at this time. It results in an abnormality in the oxygencarrying protein haemoglobin found in red blood cells. Hydroxyurea for sickle cell disease in children and for prevention of cerebrovascular events. One of the reasons for this lack of utilization of hydroxyurea is the lack of provider knowledge about the disease and the use of hydroxyurea. Effective control of sickle cell disease with hydroxyurea. Severe sickle cell disease scd remains associated with high morbidity and early mortality. Hydroxycarbamide, also known as hydroxyurea, is a medication used in sicklecell disease, chronic myelogenous leukemia, cervical cancer, and polycythemia vera. The webcast is archived and available for viewing free of charge at.
A previous scientific article external icon from the baby hug study found that 1 year old children with sickle cell anemia randomly selected to receive hu had fewer hospital stays between ages 1 and 3. This handbook was created to help answer common questions about hydroxyurea treatment. Hydroxyurea treatment for sickle cell disease nih consensus. Hydroxyurea treatment for sickle cell disease thescientificworldjournal 2002 2, 17061728 1707 hbs2,3. In a randomized clinical trial comparing hydroxyurea and placebo, hydroxyurea did not appear to affect the development of leg ulcers in people who have sickle cell disease. How i use hydroxyurea to treat young patients with sickle cell anemia. Resources for families comprehensive sickle cell disease.
However, pcps often lack access to scd specialists and are unaware of scd guidelines or efficacious treatment. Sickle cell disease scd is one of the most common inherited diseases worldwide. The role of hydroxyurea in sickle cell disease halsey 2003. The dosing of hydroxyurea for sickle cell disease is comparable to that in other diseases, although in the case of malignant disease, more drug is often given less frequently such as 80 mgkg every 3 days rather than 1520 mgkg daily. Fda approves hydroxyurea for treatment of pediatric. Hemoglobin helps red blood cells carry oxygen from the lungs to other parts of the body. Hydroxyurea treatment for sickle cell disease adapted. Hydroxyurea for sickle cell disease was webcast live february 2527, 2008. Print this easytoread handout for yourself or your patients to learn more about hydroxyurea, a medicine that can help manage the symptoms and complications of sickle cell disease. Adherence to hydroxyurea hu is a significant modifying factor in sickle cell vasoocclusive pain. In sickle cell anemia, currently, the best medication we have to prevent complications is called hydroxyurea, sometimes abbreviated hu. The most common type is known as sickle cell anaemia sca. However, intravascular haemolysis with binding of free plasma hemoglobin to. Red blood cell transfusions are another treatment choice.
Pdf minimal doses of hydroxyurea for sickle cell disease. Update on the use of hydroxyurea therapy in sickle cell. Our goal was to synthesize the published literature on the efficacy, effectiveness, and toxicity of hydroxyurea in children with sickle cell disease. In addition, a high morbidity rate is related to vascular.
Hydroxyurea is the only approved drug for treatment of sickle cell disease. People with sickle cell disease have mostly sickle or hemoglobin s hb s in their red blood cells. This reduces the amount of iron in their blood to safe levels. Hydroxyurea for the treatment of sickle cell anemia nejm. Hydroxyurea principles sto identify patients who are likely to bene. Hydroxyurea for sickle cell disease a guide for starting treatment hydroxyurea is a medicine proven to prevent pain from sickle cell disease. Pharmacotherapy of sickle cell disease world health organization. The various entities generally called sickle cell disease scd share two. To synthesize the published literature on the efficacy, effectiveness, and toxicity of hydroxyurea when used in adults with sickle cell disease. To assess the effects of hydroxyurea therapy in people with scd all genotypes, of any age. Hydroxyurea oral capsule hydrea, droxia is used to treat certain cancers and sickle cell disease. Higher hydroxyurea dose improves outcomes for young sickle.
Abstract background hydroxyurea is an effective treatment for sickle cell anemia, but few studies have been conducted in subsaharan africa, where the burden is greatest. Hydroxyurea for the treatment of sickle cell disease prepared for. This report synthesizes the available scientific evidence on sickle cell disease and offers guidance to busy primary care clinicians. These promising studies ultimately led to the multicenter study of hydroxyurea in sickle cell anemia msh. Five years of experience with hydroxyurea in children and. Daily administration of hydroxyurea is feasible and safe for children with sickle cell anemia in subsaharan africa, according to new research. The purpose of the report and accompanying quick guide 56364n is to help people living with sickle cell disease receive appropriate care by providing the best sciencebased recommendations to primary care clinicians and other medical professionals. This medication is still underused due to fears of physicians and families and must be optimized. Brandow, 2 wendy lim, 3 and richard lottenberg 4 1 division of pediatric hematologyoncology and department of pathology, oregon health and sciences university, portland, or. Hydroxyurea for the treatment of patients with sickle cell.
Hydroxyurea facts hydroxyurea is a medicine that doctors have used to treat people with sickle cell disease since the 1980s. Free hb scavenges nitric oxide a critical regulatory gas that modulates vascular tone, promoting vascular dysfunction. Areas of the body tested can include the blood, brain, kidneys, heart, and other internal organs. Our sickle cell adolescent transition guidelines are designed for teens with sickle cell disease to help them better understand their health condition. Hydroxyurea is a chemotherapy drug, and some of these types of drugs have been associated with patients developing cancer over many years.
This study evaluates the efficacy and impact of hydroxyurea hu on fetal hemoglobin hbf and other hematological parameters, which result in decreasing the painful crisis and lower hospital admissions. Common side effects include bone marrow suppression, fevers, loss of appetite. So far, research shows that hydroxyurea is as effective for children as for adults. Increase in haemoglobin f, interferes with hbs polymerization.
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