Editorial Comment Acta Haematol 2015;134:231–232 DOI: 10.1159/000431215

Received: April 23, 2015 Accepted after revision: May 6, 2015 Published online: June 26, 2015

Another Step Forward in Iron Chelation Therapy Emanuele Angelucci  Hematology and Transplant Center, ‘Armando Businco’ Regional Reference Cancer Center, Cagliari, Italy

© 2015 S. Karger AG, Basel 0001–5792/15/1344–0231$39.50/0 E-Mail [email protected] www.karger.com/aha

Even if one can postulate that best practice in myelodysplastic syndrome transfusion-dependent patients is to prevent rather than treat bulky iron deposition, this study has demonstrated that liver iron debulking is feasible by deferasirox. As a limitation, it should be underlined that the present study population is younger than the general myelodysplastic syndrome population. It is unknown if the same treatment is feasible in older patients. Moreover, the authors tested deferasirox safety and efficacy in the previously untested Japanese population. The results were similar to other reports demonstrating that there is no dissimilarity as concerns deferasirox safety and efficacy in this ethnic group compared to previously studied populations. This is a new and important observation. Finally, ophthalmologic complications were specifically studied and reported. This is an old safety concern coming from previous deferoxamine hyperchelation experience in thalassemia patients in whom ocular toxicity was described. Although no specific study has been carried out in the modern era of oral chelators, the current clinical practice is deeply influenced by this old observation. It is mostly in older patients that this warning is still a major concern. The present study, which reports data on 50 patients, provides encouraging data to address this long-standing safety issue. This study, reporting a large, very well studied transfusion-dependent adult population, provides important information contributing to improving our daily clinical practice.

Emanuele Angelucci, MD Hematology and Transplant Center ‘Armando Businco’ Regional Reference Cancer Center IT–09121 Cagliari (Italy) E-Mail emnang @ tin.it

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Iron chelation outside the well-defined field of transfusion-dependent thalassemia is ground for debate in the medical and scientific community. Although the most important question concerns the impact of chelation therapy on the survival of patients with myelodysplastic syndrome, there are other aspects that require clarification. In this issue of Acta Haematologica, Kohgo et al. [1] report an open-label, multicenter (31 centers across 5 countries), large, single-arm phase II trial to investigate deferasirox efficacy and safety in patients with myelodysplastic syndrome, aplastic anemia and other rare anemias. The authors used an adjusted dosing strategy considering transfusional iron intake and applied the only validated magnetic resonance imaging method (Ferriscan) [2] to quantitate the liver iron concentration and measure the iron removal efficacy. The sophisticated high-technology MRI method was uniformly applied to a large number of patients before starting iron chelation after 1 and 2 years of treatment. This is a relevant improvement compared to previous studies in which the chelation efficacy had been studied by serum ferritin only [3, 4]. The results provide clinically meaningful information for tailoring iron chelating therapy with deferasirox, emphasizing the potentiality of deferasirox in reducing bulky liver iron deposition. The mean absolute changes in liver iron concentration were –10.9 mg/g dry weight after 1 year and –13.5 after 2 years (baseline 24.5 mg/g). This high-quality data, together with accurate transfusional iron input estimates, could permit to quantitate the total body iron reduction by the chelation therapy [5].

References

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3 Angelucci E, Santini V, Di Tucci AA, Quaresmini G, Finelli C, Volpe A, Quarta G, Rivellini F, Sanpaolo G, Cilloni D, Salvi F, Caocci G, Molteni A, Vallisa D, Voso MT, Fenu S, Borin L, Latte G, Alimena G, Storti S, Piciocchi A, Fazi P, Vignetti M, Tura S: Deferasirox for transfusion-dependent patients with myelodysplastic syndromes: safety, efficacy, and beyond (GIMEMA MDS0306 Trial). Eur J Haematol 2014;92:527–536.

Acta Haematol 2015;134:231–232 DOI: 10.1159/000431215

4 Gattermann N, Finelli C, Della Porta M, Fenaux P, Ganser A, Guerci-Bresler A, Schmid M, Taylor K, Vassilief D, Habr D, Domokos G, Roubert B, Rose C; EPIC study investigators: Deferasirox in iron-overloaded patients with transfusion-dependent myelodysplastic syndromes: results from the large 1-year EPIC study. Leuk Res 2010;34:1143–1150. 5 Angelucci E, Brittenham GM, McLaren CE, Ripalti M, Baronciani D, Giardini C, Galimberti M, Polchi P, Lucarelli G: Hepatic iron concentration and total body iron stores in thalassemia major. N Engl J Med 2000; 343: 327–331.

Angelucci

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1 Kohgo Y, Urabe A, Kilinç Y, Agaoglu L, Warzocha K, Miyamura K, Lim CL, Glaser S, Wang C, Wiktor-Jedrzejczak W: Deferasirox decreases liver iron concentration in ironoverloaded patients with myelodysplastic syndromes, aplastic anemia and other rare anemias. Acta Haematol 2015; 134: 233–242. 2 St Pierre TG, Clark PR, Chua-Anusorn W, Fleming AJ, Jeffrey GP, Olynyk JK, Pootrakul P, Robins E, Lindeman R: Noninvasive measurement and imaging of liver iron concentrations using proton magnetic resonance. Blood 2005;105:855–861.

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