Improvement in Hematopoiesis after Iron Chelation Therapy with Deferasirox in Patients with Aplastic Anemia

Lee, Sung‐Eun; Yahng, Seung‐Ah; Cho, Byung-Sik; Eom, Ki‐Seong; Kim, Yoo-Jin; Lee, Seok; Min, Chang‐Ki; Kim, Hee-Je; Cho, Seok-Goo; Kim, Dong-Wook; Min, Woo-Sung; Park, Chong-Won; Lee, Jong Wook

doi:10.1159/000342772

Cited by 28 publications

(23 citation statements)

References 52 publications

Supporting

Mentioning

Contrasting

Order By: Relevance

“…The patient, however, reported no other drug intake, and we consider this to be further evidence of improved hematopoiesis with ICT leading to sustained transfusion independence as described in several case reports in patients with primary myelofibrosis [2,5,12,13,14,15,18]. Restoration of trilineage hematopoiesis with ICT with deferasirox was also described in several patients with aplastic anemia [12,23,24]. However, no other similar histological improvement in a patient with advanced myelofibrosis, as in the patient described here, has been reported before.…”

Section: Discussionsupporting

confidence: 58%

Transfusion Independency and Histological Remission in a Patient with Advanced Primary Myelofibrosis Receiving Iron-Chelation Therapy with Deferasirox

et al. 2016

View full text Add to dashboard Cite

Background: Iron overload is a common problem in patients with primary myelofibrosis and anemia due to transfusion dependency. This results in organ damage and toxic effects on hematopoietic cells in the bone marrow. At present, iron chelation therapy is not recommended in patients with myeloproliferative syndromes. Case Report: We describe a very interesting development in a patient with primary myelofibrosis receiving iron chelation. Transfusion independency and a nearly complete histological remission of the underlying disease occurred within a few weeks of therapy. In addition, a change in molecular genetic findings was observed. Initially a JAK2 and a U2AF1 mutation were detected in the core biopsy. During and after therapy the U2AF1 mutation progressed, whereas the JAK2 mutation could no longer be verified. Conclusion: The improvement in hematopoiesis might results from reduction of oxidative stress on hematopoietic progenitor cells or other unclear deferasirox-mediated effects, whereas the reason for the change in molecular genetic findings is unclear. It appears that deferasirox might have a modulating effect on JAK2-kinase mutations. However, further investigation of selective molecular suppression properties of deferasirox are warranted.

show abstract

Section: Discussionsupporting

confidence: 58%

Transfusion Independency and Histological Remission in a Patient with Advanced Primary Myelofibrosis Receiving Iron-Chelation Therapy with Deferasirox

et al. 2016

View full text Add to dashboard Cite

show abstract

“…In fact, one report demonstrated that iron chelation therapy improved haematopoiesis in transfusiondependent patients with thymoma-associated PRCA, although the mechanism was uncertain (Kojima et al, 2013). A similar effect of iron chelation therapy on haematopoiesis has been described in aplastic anaemia patients (Oliva et al, 2010;Lee et al, 2013).…”

Section: Months Aōer Diagnosis Survivalmentioning

confidence: 89%

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

et al. 2015

View full text Add to dashboard Cite

SummaryImmunosuppressive therapy has been employed as the initial treatment for acquired chronic pure red cell aplasia (PRCA), such as idiopathic, thymoma-associated, or large granular lymphocyte (LGL) leukaemia-associated PRCA, which is thought to be immune-mediated. To explore the overall long-term outcome following immunosuppression and to identify the risk factors for death in these disorders, we conducted nationwide surveys in Japan 2004 and 2006, and identified a total of 185 patients with acquired chronic PRCA, including 72 idiopathic, 41 thymoma-associated and 14LGL leukaemia-associated cases of PRCA for whom data was available. The present study evaluated 127 patients with these three subsets of PRCA. The median overall survival has not yet been reached in idiopathic PRCA. The estimated median overall survival times in patients with thymomaassociated and LGL leukaemia-associated PRCA were 142Á1 and 147Á8 months, respectively. Twenty-two deaths were reported, and the response to induction therapy and relapse of anaemia were found to be associated with death. The major causes of death were infection in seven patients and organ failure in another seven patients. The results suggest that maintenance therapy and the management of infectious complications are crucial for improving the prognosis of chronic PRCA.

show abstract

“…Obviously, this rationale can be applied only in the context of a well‐designed clinical trial. The initial published experiences in various diseases clearly support larger clinical trials.…”

Section: Management Of Iron Overload During Transplantmentioning

confidence: 81%

Management of iron overload before, during, and after hematopoietic stem cell transplantation for thalassemia major

Angelucci¹,

Pilo²

2016

Annals of the New York Academy of Sciences

View full text Add to dashboard Cite

Solid evidence has established the negative impact of high iron burden and related tissue damage on the outcome of hemopoietic stem cell transplantation for thalassemia major. Recent improvements in our knowledge of iron metabolism have been focused on elevated non-transferrin-bound iron and labile plasma iron levels in the peritransplantation period as potential contributors to tissue toxicity and subsequent adverse transplant outcome. As mouse models have shown, iron overload can injure bone marrow hematopoiesis by increasing reactive oxygen species. The Pesaro experience, conducted in the deferoxamine-only era, clearly defined three iron-related factors (liver fibrosis, hepatomegaly, and quality of lifelong chelation) as significantly affecting transplant outcome. The detrimental effect of iron has only been clarified in recent years. Active interventional strategies are ongoing. Although successful hematopoietic stem cell transplantation clinically resolves the thalassemia marrow defect, patients still remain carriers of iron overload and of all the clinical complications acquired during prior years of transfusion therapy. Therefore, adequate "iron diagnosis" and management is mandatory after hemopoietic stem cell transplantation. In transplanted thalassemia patients, body iron should be returned to within the normal range. Phlebotomy is the gold standard to reduce iron burden; though deferoxamine is a proven, acceptable alternative, clinical investigations on deferasirox are ongoing.

show abstract

Improvement in Hematopoiesis after Iron Chelation Therapy with Deferasirox in Patients with Aplastic Anemia

Cited by 28 publications

References 52 publications

Transfusion Independency and Histological Remission in a Patient with Advanced Primary Myelofibrosis Receiving Iron-Chelation Therapy with Deferasirox

Transfusion Independency and Histological Remission in a Patient with Advanced Primary Myelofibrosis Receiving Iron-Chelation Therapy with Deferasirox

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

Management of iron overload before, during, and after hematopoietic stem cell transplantation for thalassemia major

Contact Info

Product

Resources

About