Deferasirox treatment improved hematopoiesis and led to complete remission in a patient with pure red cell aplasia

Abstract

A 64-year-old woman developed pure red cell aplasia (PRCA) 4 years after thymectomy for thymoma. During anti-thymocyte globulin treatment, the patient developed cytomegalovirus pneumonia and was thus unable to continue immunosuppressive therapy and became transfusion dependent. Deferasirox was started for treatment with iron overload when serum ferritin increased to >1000 ng/mL. Seven months after initiation of deferasirox treatment, serum ferritin level decreased the normal range and the patient has remained transfusion independent thereafter. Deferasirox was discontinued when serum ferritin level decreased below 500 ng/mL, and she has maintained in complete remission over the last 15 months. Hypotheses have been raised regarding the improvement of hematopoiesis by deferasirox treatment, but the mechanism whereby this might be achieved remains unclear. Deferasirox treatment may be clinically beneficial both by reducing iron overload and by improving hematopoiesis in patients with PRCA.

The clinical characteristics and therapy response of patients with acquired pure red cell aplasia

“…Hirokawa reported that treatment of post-transfusion iron overload may contribute to the improvement of the outcome of acquired PRCA [3]. Kojima reported that iron chelation therapy improved hematopoiesis and led to CR in one PRCA patient [22]. Our research is consistent with previous studies, confirms that iron overload is associated with poor prognosis of PRCA.…”

Long‐term outcome of patients with acquired chronic pure red cell aplasia (PRCA) following immunosuppressive therapy: a final report of the nationwide cohort study in 2004/2006 by the Japan PRCA collaborative study group

“…These findings suggest that management of infectious complications and treatment of post‐transfusion iron overload may contribute to the improvement of the treatment outcome of acquired chronic PRCA patients receiving immunosuppressive therapy. In fact, one report demonstrated that iron chelation therapy improved haematopoiesis in transfusion‐dependent patients with thymoma‐associated PRCA, although the mechanism was uncertain (Kojima et al , ). A similar effect of iron chelation therapy on haematopoiesis has been described in aplastic anaemia patients (Oliva et al , ; Lee et al , ).…”

Iron as the Key Modulator of Hepcidin Expression in Erythroid Antibody-Mediated Hypoplasia

“…It seems, therefore, that the increased iron concentration is the key modulator of hepcidin synthesis in this type of erythroid hypoplasia. In fact, it has been recently reported that the reduction of serum iron by iron chelation therapy promoted an improvement in erythropoiesis in PRCA, though the mechanism whereby this is achieved is still unclear [ 46 ].…”