High-dose intravenous methylprednisolone therapy for patients with Diamond-Blackfan anemia refractory to conventional doses of prednisone.
OBJECTIVE
To assess the efficacy and toxicity of very high doses of glucocorticoids in patients with congenital pure red cell aplasia (Diamond-Blackfan anemia) who did not respond to standard doses of prednisone.


METHODS
We prospectively treated eight patients with transfusion-dependent Diamond-Blackfan anemia with high intravenous doses of methylprednisolone. All patients had previously not responded to one or more oral courses of prednisone in standard doses and were dependent on erythrocyte transfusions. Every patient initially received methylprednisolone at a dose of 30 mg/kg per day, followed by slow tapering for 4 weeks, but none responded. All patients then received a second treatment course starting at 100 mg of methylprednisolone per kilogram per day, again followed by slow tapering of the dosage.


RESULTS
Three patients had a complete response that has been sustained for 21+, 31+, and 41+ months, respectively. One patient had a partial response. Toxic effects included a rise in serum alanine aminotransferase activity in all patients, transient diabetes mellitus in one child, and three episodes of bacteremia in two patients with intravenous access devices.


CONCLUSIONS
We conclude that very high doses of methylprednisolone may induce sustained remission in some patients with transfusion-dependent Diamond-Blackfan anemia refractory to standard-dose prednisone therapy.
Bernini
J C
JC
Department of Pediatrics, University of Texas Southwestern Medical Center, Dallas 75235-9063, USA.
Carrillo
J M
JM
Buchanan
G R
GR
D000293:Adolescent; D017668:Age of Onset; D000753:Anemia, Refractory / Q000188:drug therapy*; D000890:Anti-Infective Agents / Q000008:administration & dosage / Q000627:therapeutic use; D002675:Child, Preschool; D002985:Clinical Protocols; D004305:Dose-Response Relationship, Drug; D005199:Fanconi Anemia / Q000188:drug therapy*; D005938:Glucocorticoids / Q000008:administration & dosage* / Q000009:adverse effects / Q000627:therapeutic use*; D006801:Humans; D007223:Infant; D008775:Methylprednisolone / Q000008:administration & dosage* / Q000009:adverse effects / Q000627:therapeutic use*; D011010:Pneumocystis; D016720:Pneumocystis Infections / Q000188:drug therapy / Q000517:prevention & control; D011241:Prednisone / Q000008:administration & dosage / Q000627:therapeutic use; D011446:Prospective Studies; D015662:Trimethoprim, Sulfamethoxazole Drug Combination / Q000008:administration & dosage / Q000627:therapeutic use
D000890:Anti-Infective Agents; D005938:Glucocorticoids; D015662:Trimethoprim, Sulfamethoxazole Drug Combination; D011241:Prednisone; D008775:Methylprednisolone