Biomaterial Database

High-dose intravenous corticosteroid treatment for patients with Diamond-Blackfan syndrome resistant or refractory to conventional treatment. 1988

S Ozsoylu

Department of Pediatrics, Faculty of Medicine, Hacettepe University, Ankara, Turkey.

Five out of eight patients with congenital pure red cell aplasia (PRCA), who became refractory or did not respond to conventional prednisone treatment (2 mg/kg), responded to high-dose intravenous methylprednisolone administration. The drug was given daily 30 mg/kg for 3 days, 20 mg/kg for 4 days, then subsequently 10, 5, and 2 mg/kg for a week, followed by 1 mg/kg until the hemoglobin level reached 12 g/dl. In the other three patients, although normoblasts appeared in the bone marrow, hemoglobin elevation was not observed. These three patients finally responded fully to a dose of 100 mg/kg intravenously. Therefore, we believe that resistance to corticosteroids could be overcome by increasing the dose in patients with Diamond-Blackfan syndrome. Four of these patients have been off the treatment for 2-13 months. Seven recurrences occurred in three patients 6-9 months following discontinuation of the treatment. However, all again responded to the same regimen.

UI	MeSH Term	Description	Entries
D007223	Infant	A child between 1 and 23 months of age.	Infants
D007275	Injections, Intravenous	Injections made into a vein for therapeutic or experimental purposes.	Intravenous Injections,Injection, Intravenous,Intravenous Injection
D008297	Male		Males
D008775	Methylprednisolone	A PREDNISOLONE derivative with similar anti-inflammatory action.	6-Methylprednisolone,Medrol,Metipred,Urbason,6 Methylprednisolone
D011241	Prednisone	A synthetic anti-inflammatory glucocorticoid derived from CORTISONE. It is biologically inert and converted to PREDNISOLONE in the liver.	Dehydrocortisone,delta-Cortisone,Apo-Prednisone,Cortan,Cortancyl,Cutason,Dacortin,Decortin,Decortisyl,Deltasone,Encorton,Encortone,Enkortolon,Kortancyl,Liquid Pred,Meticorten,Orasone,Panafcort,Panasol,Predni Tablinen,Prednidib,Predniment,Prednison Acsis,Prednison Galen,Prednison Hexal,Pronisone,Rectodelt,Sone,Sterapred,Ultracorten,Winpred,Acsis, Prednison
D012010	Red-Cell Aplasia, Pure	Suppression of erythropoiesis with little or no abnormality of leukocyte or platelet production.	Aplasia Pure Red Cell,Erythrocyte Aplasia,Pure Red-Cell Aplasia,Aplasia, Erythrocyte,Aplasia, Pure Red-Cell,Aplasias, Erythrocyte,Erythrocyte Aplasias,Pure Red Cell Aplasia,Pure Red-Cell Aplasias,Red Cell Aplasia, Pure,Red-Cell Aplasias, Pure
D001827	Body Height	The distance from the sole to the crown of the head with body standing on a flat surface and fully extended.	Body Heights,Height, Body,Heights, Body
D002675	Child, Preschool	A child between the ages of 2 and 5.	Children, Preschool,Preschool Child,Preschool Children
D004351	Drug Resistance	Diminished or failed response of an organism, disease or tissue to the intended effectiveness of a chemical or drug. It should be differentiated from DRUG TOLERANCE which is the progressive diminution of the susceptibility of a human or animal to the effects of a drug, as a result of continued administration.	Resistance, Drug
D004920	Erythropoiesis	The production of red blood cells (ERYTHROCYTES). In humans, erythrocytes are produced by the YOLK SAC in the first trimester; by the liver in the second trimester; by the BONE MARROW in the third trimester and after birth. In normal individuals, the erythrocyte count in the peripheral blood remains relatively constant implying a balance between the rate of erythrocyte production and rate of destruction.	Erythropoieses