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| Treatments/Remission |
Specific treatment for DBA and its complications will be determined by your child's physician based on:
Treatment options may include, but are not limited to, the following: -Steroid therapy. This involves taking oral corticosteroids in liquid or pill form. The medicine is usually taken every day or every other day in divided doses. Corticosteroids are different from the more familiar androgenic steroids (like testosterone) that are sometimes taken to increase muscle mass. The goal of the therapy is to increase the hemoglobin, or red blood cells. Side effects of steroids include, but are not limited to: upset stomach, increased appetite, weight gain, unstable blood sugars, increased blood pressure, decreased height, and susceptibility to infection. However, in many patients with DBA the effective dose of corticosteroids is low enough that few or no side effects are observed. -Red Blood Cell transfusions. This requires the insertion of an IV needle or more permanent access device such as an Infuse-A-Port, (ie. Mediport, Central Line, etc.) in order to deliver the blood to the patient. Transfusions take 2-4 hours and are given at hospital or outpatient transfusion centers. Side effects of red blood cell transfusions include, but are not limited to: fever, headache, hives, allergic reaction, infection, and iron overload. Blood transfusions may be repeated every 4-8 weeks, depending on the patient. Blood transfusions are used when anemia is unusually severe or when the response to corticosteroid therapy is inadequate. -Bone Marrow Transplant. At present, this is the only cure for DBA. Bone marrow transplant involves the replacement of diseased bone marrow with another person's healthy bone marrow. The decision to proceed with bone marrow transplant should be discussed with your child's Hematologist and a Bone Marrow Transplant Team. *Click here to see the latest results of Stem Cell Transplants in DBA* Additional treatment alternatives are currently being studied. Pharmaceutical research is practically non-existent, due to the small number of DBA sufferers worldwide. However, pharmaceutical research into related conditions may lead to better treatment for DBA patients, especially related to transfusions and chelation therapy. Experimental Studies Antithymocyte Globulin-ATG (more info. to come) Reglan (more info. to come) Click Here Cyclosporine (more info. to come) Click Here *New* See recommendations on treatment from DBAR Information obtained by Children's Hospital Boston at www.childrenshospital.org, DBA Registry at www.dbar.org and DBA Foundation at www.dbafoundation.org |
| --------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------------- * New Information* Remission A “remission” is defined as a stable hemoglobin adequate for age, maintained for at least six months, without any corticosteroids, transfusions or other therapy. Outcomes of Remission: 61 patients in the DBAR have sustained remission (more than 6 months without treatment). 44 remitted from steroid therapy, 10 from transfusion therapy, 2 with no treatment, 5 unknown. 9 patients relapsed (2 later remitted and 1 died from complications of SCT). The actuarial likelihood of remission is 20% by age 25, with 72% of those happening during the first decade of life. In the DBAR, there are 44 patients to remitted prior to age 10, 15 after age 10, and 2 unknown. The conclusion regarding remission out comes is that although there seems to be equal numbers (proportionally) remitting from either steroid or transfusion therapy, only 2 of the patients remitting from transfusion had failed steroid therapy (1 primary and 1 secondary failure). Steroids do not seem to induce remission (4 remitters were never treated with steroids). Information provided by the Diamond Blackfan Anemia Registry of North America and notes taken by people at Camp Sunshine in 2006 |
