Serum ferritin level and morbidity risk in transfusion-independent patients with  -thalassemia intermedia: the ORIENT study

Musallam, Khaled M.; Cappellini, Maria Domenica; Daar, Shahina; Karimi, Mehran; El‐Beshlawy, Amal; Graziadei, Giovanna; Magestro, Matthew; Wulff, Jerome; Pietri, G.; Taher, Alì

doi:10.3324/haematol.2013.097220

Cited by 58 publications

(60 citation statements)

References 9 publications

(15 reference statements)

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“…The increased number of DFP-treated patients who were initially classified as having a higher serum ferritin level (>800 lg/l) and transitioned to the lower serum ferritin level classification (>300 and <800 lg/l; 300 lg/l; Supporting Information Table 6) suggest that this treatment may be able to decrease morbidity [17]. Musallam et al (2014) suggested that the mean overall serum ferritin level is higher in patients with at least one morbidity [17].…”

Section: Discussionmentioning

confidence: 99%

“…Musallam et al (2014) suggested that the mean overall serum ferritin level is higher in patients with at least one morbidity [17]. Moreover, the same authors have suggested that the cumulative incidence of multiple morbidities is 59.3% in patients with a serum ferritin level 800 lg/l, 5.9% in patients with > 300 and < 800 lg/ml serum ferritin, and 0% in patients with 300 lg/l serum ferritin (Supporting Information Table 6) [17].…”

Section: Discussionmentioning

confidence: 99%

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Deferiprone versus deferoxamine in thalassemia intermedia: Results from a 5‐year long‐term Italian multicenter randomized clinical trial

et al. 2015

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In patients with thalassemia intermedia (TI), such as beta-TI, alpha-thalassemia (mainly HbH disease and mild/moderate forms of HbE/beta-thalassemia), iron overload is an important challenge in terms of diagnosis, monitoring, and treatment. Moreover, to date, the only possible chelators available are deferoxamine, deferasirox, and deferiprone. Here, we report the first 5-year long-term randomized clinical trial comparing the effectiveness of deferiprone versus deferoxamine in patients with TI. Body iron burden, which was determined by measuring serum ferritin levels in the same patient over 5 years and analyzed according to the generalized linear mixed model (GLMM), showed a linear decrease over time in the mean serum ferritin levels in both treatment groups (P-value 5 0.035). The overall period of observation was 235.2 person-years for the deferiprone patients compared with 214.3 person-years for the deferoxamine patients. The results of the log-rank test suggested that the deferiprone treatment did not affect survival compared with the deferoxamine treatment (P-value 5 0.360). The major adverse events observed included gastrointestinal symptoms and joint pain or arthralgia. Neutropenia and agranulocytosis were also detected, suggesting needing of strict hematological control. In conclusion, long-term iron chelation therapy with deferiprone is associated with an efficacy and safety similar to that of deferoxamine, suggesting that this drug is an alternative option in cases in which deferoxamine and deferasirox are contraindicated.

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Section: Discussionmentioning

confidence: 99%

Section: Discussionmentioning

confidence: 99%

Deferiprone versus deferoxamine in thalassemia intermedia: Results from a 5‐year long‐term Italian multicenter randomized clinical trial

et al. 2015

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“…4 Levels <9 to 10 g/dL (which indicate considerable ineffective erythropoiesis in nontransfusion-dependent patients and suboptimal transfusion therapy in transfusion-dependent patients) showed highest thrombosis risk. 5,7 Advancing age prolongs the exposure of patients to these risk factors, and higher rates of thrombosis have been reported in older beta-thalassemia patients (>35 years). 5,6 Iron overload (serum ferritin ≥800-1000 μg/L) in these patients has also been associated with high thrombosis rates, in view of the damaging effects of toxic iron species on the endothelium and vasculature, as well as to secondary development of multiorgan failure (heart, liver, and endocrine dysfunction).…”

Section: Supporting Informationmentioning

confidence: 99%

Nationwide outcomes of nontrauma splenectomy

et al. 2013

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“…Iron overload, which is responsible for many of the complications, develops slowly in these patients, producing cumulative effects. Indeed, several studies have established the positive correlation between iron overload indices and advancing age (29,37,38). Nonetheless, the three main complications encountered in the present study, apart from iron overload, were gallstones in five patients, moyamoya and hypothyroidism in one patient each.…”

Section: Discussionmentioning

confidence: 69%

Clinical and Molecular Characteristics of Non-Transfusion-Dependent Thalassemia in Kuwait

et al. 2015

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Although not regularly transfused, patients with non-transfusion-dependent thalassemia (NTDT) are prone to iron overload and its complications. Their molecular, phenotypical and laboratory characteristics vary in different populations and there is a need to document local prevailing patterns. We have reviewed the records of our patients with NTDT in Kuwait and documented their clinical and molecular characteristics in addition to iron status [serum ferritin and liver magnetic resonance imaging (MRI) T2*], management and complications. There were 41 patients, made up of 20 with β-thalassemia intermedia (β-TI), 18 with Hb H (β4) disease and three with Hb E (HBB: c.79G > A)-β-thalassemia (Hb E-β-thal); their ages ranged from 3 to 36 years (mean 12.5 ± 7.7). While 18 (43.9%) had been transfused at least once, only three (7.3%) had been transfused on multiple occasions. Three patients had serum ferritin >500 ng/mL; while four of 38 had mild or moderate liver iron overload. Seven (35.0%) of the β-TI patients were managed with hydroxyurea (HU) with good response. Other complications included five patients with gallstones and one each of hypothyroidism and moyamoya. The most common mutations among the β-TI patients were IVS-II-1 (G > A) and IVS-I-6 (T > C), while among the Hb H patients, the Saudi α2-globin gene polyadenylation (polyA) (AATAAA > AATAAG) mutation was responsible for all cases either as homozygotes (61.1%) or compound heterozygotes with the α-thal-2 (-α(3.7)) allele (33.3%). Although the pattern of NTDT in Kuwaiti patients is generally mild, there is a need to follow them to adulthood as the complications are cumulative and more prevalent in this group.

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Serum ferritin level and morbidity risk in transfusion-independent patients with -thalassemia intermedia: the ORIENT study

Cited by 58 publications

References 9 publications

Deferiprone versus deferoxamine in thalassemia intermedia: Results from a 5‐year long‐term Italian multicenter randomized clinical trial

Deferiprone versus deferoxamine in thalassemia intermedia: Results from a 5‐year long‐term Italian multicenter randomized clinical trial

Nationwide outcomes of nontrauma splenectomy

Clinical and Molecular Characteristics of Non-Transfusion-Dependent Thalassemia in Kuwait

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