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Coexistence of normal and clonal haemopoiesis in aplastic anaemia patients treated with immunosuppressive therapy
Author(s) -
Giovanna Piaggio,
Marina Podestà,
A. Pitto,
Mario Sessarego,
O Figari,
Giuseppina Fugazza,
Federica Benvenuto,
Benedetto Bruno,
Maria Teresa Van Lint,
Mauro Truini,
Francesco Frassoni,
Andrea Bacigalupo
Publication year - 1999
Publication title -
british journal of haematology
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.907
H-Index - 186
eISSN - 1365-2141
pISSN - 0007-1048
DOI - 10.1046/j.1365-2141.1999.01729.x
Subject(s) - haematopoiesis , immunology , medicine , hematology , stem cell , biology , genetics
Cytogenetic abnormalities and paroxysmal nocturnal haemoglobinuria (PNH) phenotype are frequent findings in aplastic anaemia patients treated with immunosuppressive therapy (IST). In this study we investigated whether the appearance of clonal haemopoiesis influences patient outcome and survival. 97 patients entered this study and were followed from the onset of the disease for a median follow‐up (FU) of 53 months. 93% are alive, 56% achieved complete remission, 30% partial remission, both transfusion independent, and 14% did not respond. Three groups were identified: (A) patients without evidence of emerging clones (71/97); (B) patients who acquired chromosomal abnormalities (13/97); (C) patients who showed low expression of glycosyl phosphatidylinositol anchored proteins (GPI‐AP) (PNH phenotype) at presentation or later (16/97). Three patients showed both PIG‐AP deficiency and chromosomal abnormalities. The actuarial survival of patients without clonal haemopoiesis ( n = 71) at 6 years was 95%, for patients with chromosomal abnormalities ( n = 13), 88%, and for patients with PIG‐AP deficiency ( n = 16), 89%. There was no difference in the probability of becoming transfusion independent in the three groups (93%, 92% and 88% respectively). This study confirmed that a proportion of severe aplastic anaemia (SAA) patients exhibit clonal markers during the time after IST, often coexisting with cytogenetically or phenotypically normal haemopoiesis. There was no significant clinical impact of these abnormalities on transfusion independence and survival at the median follow‐up of 4 years.