Successful treatment of Diamond‐Blackfan anemia with metoclopramide

Abstract

Diamond-Blackfan anemia (DBA) is a congenital anemia characterized by a low reticulocyte count, the absence or severe reduction of hemoglobin-containing cells in the bone marrow, and normal megakaryocytic and granulocytic differentiation. Although the anemia may initially respond to corticosteroid therapy, many patients require lifelong red blood cell (RBC) transfusion, leading to infectious complications and iron overload. Metoclopramide has recently been used to treat DBA. Treatment with metoclopramide induces the release of prolactin from the pituitary and stimulates erythropoiesis. For these reasons, we used metoclopramide to treat a 20-year-old man with DBA refractory to low and high doses of corticosteroids, cyclosporin A, and tacrolimus (FK506). The hemoglobin and hematocrit slowly increased, and he has remained asymptomatic and transfusion-independent for 8 months. Metoclopramide therapy should be considered in patients with refractory DBA before treatment-related complications develop. Am.

Pregnancy and lactation modulate maternal splenic growth and development of the erythroid lineage in the rat and mouse

“…Prolactin (PRL) has been implicated as a regulator of haematopoiesis Lowenstein 1964, 1965;Bellone et al 1997;Abkowitz et al 2002;Akiyama et al 2005). Early studies demonstrated that PRL stimulates erythropoiesis in nonpregnant, pregnant and lactating mice Lowenstein 1964, 1965).…”

Diagnosing and treating Diamond Blackfan anaemia: results of an international clinical consensus conference

“…Review ª 2008 The Authors (Totterman et al, 1984;Williams et al, 1987;Seip & Zanussi, 1988;Leonard et al, 1989;Splain & Berman, 1992;Monteserin et al, 1993;Alessandri et al, 2000;Bobey et al, 2003) or metoclopramide (Abkowitz et al, 2002;Akiyama et al, 2005;Leblanc et al, 2007), but currently these responders cannot be identified a priori. There have been two reports on the use of valproic acid (Jabr et al, 2004;Jabr & Taher, 2006) and leucine with good outcomes, however these are case reports and warrant further study before any conclusions can be drawn.…”

“…These drugs appear to be largely ineffective and there is currently no evidence that any of these has a major role in the management of DBA (Geller et al , 1975; Dunbar et al , 1991; Fiorillo et al , 1991; Niemeyer et al , 1991; Gomez‐Almaguer & Gonzalez‐Llano, 1992;Sumimoto et al , 1992; Bejaoui et al , 1993; Gillio et al , 1993a, 1993b;Bastion et al , 1994; Brown et al , 1994; Olivieri et al , 1994; Ozsoylu, 1994; Ball et al , 1995; Bernini et al , 1995; Buchanan, 2001; Alter, 2003 ). Specific patients may respond to cyclosporine ( Totterman et al , 1984; Williams et al , 1987; Seip & Zanussi, 1988; Leonard et al , 1989; Splain & Berman, 1992; Monteserin et al , 1993; Alessandri et al , 2000; Bobey et al , 2003) or metoclopramide (Abkowitz et al , 2002; Akiyama et al , 2005; Leblanc et al , 2007), but currently these responders cannot be identified a priori . There have been two reports on the use of valproic acid (Jabr et al , 2004; Jabr & Taher, 2006) and leucine (Pospisilova et al , 2007) with good outcomes, however these are case reports and warrant further study before any conclusions can be drawn.…”

How I treat Diamond-Blackfan anemia

“…Unfortunately, other studies in the United States and Europe did not confirm these responses but showed an overall 10% response rate. 66,67 Most recently, leucine and lenalidomide have been contemplated for use in DBA. Recently, leucine has been tried in one patient with DBA and reported to have attained a remission.…”