TY - JOUR
T1 - Clinical characteristics predict response to antithymocyte globulin in paroxysmal nocturnal haemoglobinuria
AU - Paquette, Ronald L.
AU - Yoshimura, Ryan
AU - Veiseh, Charles
AU - Kunkel, Lori
AU - Gajewski, James
AU - Rosen, Peter J.
PY - 1997
Y1 - 1997
N2 - Seven patients with paroxysmal nocturnal haemoglobinuria (PNH) were treated with antithymocyte globulin (ATG). Each patient received ATG (20 mg/kg/d) for 8 d and prednisone to prevent or control serum sickness. Three patients experienced a sustained improvement in at least one peripheral blood cytopenia, including one patient who had a complete trilineage response. Several pretreatment clinical features appeared to be associated with response to ATG. All responding patients had hypoproliferative features including depressed platelet counts (<30 x 109/l), and a minor degree of chronic haemolysis as indicated by relatively low reticulocyte counts (<100 x 109/l), lactate dehydrogenase (<1000 U/l) and total bilirubin (<17 μmol/l) levels. Responding patients continued to have chronic low-grade haemolysis after their response to immunosuppression that was similar to that observed prior to treatment. The non-responding patients had a classic haemolytic form of PNH characterized by elevated reticulocyte counts (>100 x 109/l), lactate dehydrogenase (>2000 U/l) and total bilirubin (>17 μmol/l) levels. The impaired haemopoiesis that occurs in hypoproliferative PNH may respond to ATG treatment, but the haemolytic component of the disease, and hence the PNH clone, is not altered by immunosuppressive therapy.
AB - Seven patients with paroxysmal nocturnal haemoglobinuria (PNH) were treated with antithymocyte globulin (ATG). Each patient received ATG (20 mg/kg/d) for 8 d and prednisone to prevent or control serum sickness. Three patients experienced a sustained improvement in at least one peripheral blood cytopenia, including one patient who had a complete trilineage response. Several pretreatment clinical features appeared to be associated with response to ATG. All responding patients had hypoproliferative features including depressed platelet counts (<30 x 109/l), and a minor degree of chronic haemolysis as indicated by relatively low reticulocyte counts (<100 x 109/l), lactate dehydrogenase (<1000 U/l) and total bilirubin (<17 μmol/l) levels. Responding patients continued to have chronic low-grade haemolysis after their response to immunosuppression that was similar to that observed prior to treatment. The non-responding patients had a classic haemolytic form of PNH characterized by elevated reticulocyte counts (>100 x 109/l), lactate dehydrogenase (>2000 U/l) and total bilirubin (>17 μmol/l) levels. The impaired haemopoiesis that occurs in hypoproliferative PNH may respond to ATG treatment, but the haemolytic component of the disease, and hence the PNH clone, is not altered by immunosuppressive therapy.
KW - antithymocyte globulin
KW - aplastic anaemia
KW - immunosuppressive therapy
KW - paroxysmal nocturnal haemoglobinuria
UR - http://www.scopus.com/inward/record.url?scp=0031032545&partnerID=8YFLogxK
UR - http://www.scopus.com/inward/citedby.url?scp=0031032545&partnerID=8YFLogxK
U2 - 10.1046/j.1365-2141.1997.d01-1984.x
DO - 10.1046/j.1365-2141.1997.d01-1984.x
M3 - Article
C2 - 9012693
AN - SCOPUS:0031032545
SN - 0007-1048
VL - 96
SP - 92
EP - 97
JO - British Journal of Haematology
JF - British Journal of Haematology
IS - 1
ER -