Safety Of Deferiprone Research Articles

Safety of Deferiprone in Patients with Myelodysplastic Syndromes: Results from the Deferiprone US Safety Registry and a Compassionate Use Program

Background: Myelodysplastic syndromes (MDS) are a heterogenous group of hematopoietic stem cell malignancies characterized by ineffective blood cell production. Treatment can include frequent blood transfusions, which may lead to iron overload and subsequent complications. Several treatment guidelines recommend iron chelation therapy (ICT) for lower-risk patients with MDS based on transfusion burden, ferritin level, or iron overload imaging studies. However, ICT options are limited for patients with MDS because deferoxamine is administered parenterally and requires a burdensome infusion regimen that may reduce treatment adherence. Deferasirox is administered orally and requires closely monitoring a patient's renal and hepatic function; deferasirox is contraindicated in patients with significant renal impairment (estimated glomerular filtration rate <40 mL/min/1.73m2), a frequent comorbidity in the primarily geriatric population of patients with MDS. Deferiprone (DFP) is an oral ICT approved for transfusional iron overload in thalassemia, sickle cell disease, and other anemias. Although DFP has no contraindication or dosing adjustment based on renal function, it requires close patient monitoring and laboratory testing, since its use is associated with severe neutropenia (absolute neutrophil count <0.5x109/L) in 1-2% of patients. While existing studies have evaluated the effects of DFP in patients with MDS, limited data on its safety are available for this patient population. Objective: To evaluate the DFP safety profile in patients with MDS from a US safety registry and a compassionate use program. Methods: The DFP US safety registry was established to meet FDA postmarketing requirements following DFP approval in the US in 2011. The registry assessed all reports of adverse events (AEs) in all patients, including those with MDS, from 2011 to 2021. The compassionate use program included patients in the US and Canada from 1996 to 2015. All AEs were assessed in all patients with MDS who received this chelator. In both the safety registry and the compassionate use program, DFP was administered at doses that ranged from 75 - 99 mg/kg/day. AEs were assessed irrespective of a causal relationship with DFP. Results: The US safety registry included 115 adults with MDS (mean ± SD age: 77.7 ± 9.0 y; 61.7% males). Mean ± SD DFP exposure was 1.1 ± 1.4 y (range: 0-7.9 y), with 59 (51.3%) patients receiving DFP for > 6 mo and 34 (29.6%) for > 12 mo. Reasons for discontinuation (> 1 patient) included death (n=30), physician direction (n=26), adverse reaction (n=16), hospice care (n=12), nonresponsive patient (n=6), insurance issues (n=4), and switching to another chelator/formulation (n=3). Observed AEs are summarized in Table 1. The most common AEs were diarrhea (n=34), nausea (n=32), vomiting (n=26), and fatigue (n=26). No deaths were attributed to DFP. In 3 patients, neutropenia was reported in temporal association with fatal outcome, but none were attributed to DFP. Four patients (3.5%) reported severe neutropenia/agranulocytosis: 2 events resolved; 1 case outcome was not reported; and 1 case was ongoing at last follow-up. The compassionate use program included 15 adults with MDS (mean ± SD age: 67.9 ± 10.4 y; 60% males). Mean ± SD DFP exposure was 1.2 ± 2.3 y, with a median (range) of 0.7 y (0-9.1 y). Reported AEs are summarized in Table 2. The most common AEs were nausea and decreased neutrophil count (n=3 each). There were 2 cases of severe neutropenia/agranulocytosis; both resolved. Conclusions: The findings of the safety registry and a compassionate use program suggest the DFP safety profile in MDS is potentially consistent with that of thalassemia, sickle cell disease, and other anemias. No unexpected AEs and no new safety concerns were found. The frequency of the most serious AE associated with DFP, severe neutropenia/agranulocytosis, was 3.5% within this population; of reported outcomes, all resolved. In the compassionate use program, 13.3% of patients reported agranulocytosis, which is higher than what has been shown in patient populations without MDS (2%); however, it is not clear if this percentage is higher than other ICT agents used in MDS, due to the patients' primary disease, or the relatively small sample size. While acknowledging these limitations and observational nature of the data, DFP may be a viable ICT for patients with lower-risk MDS. Figure 1View largeDownload PPTFigure 1View largeDownload PPT Close modal

RANDOMIZED CONTROLLED TRIAL OF THE EFFICACY AND SAFETY OF DEFERIPRONE: SUBGROUP ANALYSIS OF PEDIATRIC PATIENTS IN IRON-OVERLOADED PATIENTS WITH SICKLE CELL DISEASE AND OTHER ANEMIAS

Deferiprone (DFP), an oral iron chelator, has recently been approved as the first-line treatment for transfusional iron overload in pediatric and adult patients with Sickle Cell Disease (SCD) and other anemias. The FIRST study (NCT02041299) reported that DFP is noninferior to Deferoxamine (DFO) in patients with SCD and iron overload (as assessed by Liver Iron Concentration [LIC]) and has an acceptable safety profile. Here, we report a subgroup analysis of FIRST to assess whether the efficacy and safety of DFP are comparable to DFO in children with SCD. In this multicenter, 2-arm, randomized, open-label study, eligible patients were randomized 2:1 to receive DFP or DFO for 12 months. The subgroup analysis included children (2–16 years of age) with SCD or another rare anemia who were treated for transfusional iron overload. The primary efficacy endpoint was the change in LIC from baseline to month 12. Safety was assessed by Adverse Events (AEs) in the study period. The safety analysis was composed of 128 (DFP, n = 86; DFO, n = 42) children; most (DFP, 76%; DFO, 81%) had a primary diagnosis of SCD (HbS). Mean age (SD) in the DFP and DFO arms were 9.9 (3.7) and 10.9 (3.0) years (p = 0.09), respectively. 5 children in the DFP arm withdrew due to AEs and 19 (DFP, n = 14; DFO, n = 5) for other reasons. Children treated with DFP or DFO showed no difference in the number of AEs (p = 0.77; including neutropenia [p = 0.30]), severe AEs (p = 0.10), serious AEs (p = 0.16), or withdrawals due to AEs (p = 0.17). Overall incidence of nonserious AEs possibly related to treatment was higher with DFP than DFO (59% vs. 33%; p = 0.01). Transient elevation of liver enzymes was significantly higher with DFP (p = 0.03). 1 child developed agranulocytosis during parvovirus infection, which resolved the following day. In the efficacy population, there was no significant difference in mean (SD) LIC decrease from baseline to 12 months with DFP compared to DFO (-3.39 ± 4.24 mg/g vs. -2.99 ± 3.16 mg/g, respectively; p = 0.57). This pediatric subgroup analysis corroborates previous findings that DFP is comparable to DFO in safely reducing LIC in children with transfusion-dependent anemias. No new safety concerns were observed in children that have not been previously noted in other populations. The present findings on DFP may benefit children with SCD and their healthcare providers when considering effective iron chelation therapy that may also address treatment-adherence concerns.

S115: LONG-TERM EFFICACY AND SAFETY OF DEFERIPRONE FOR PATIENTS WITH SICKLE CELL DISEASE OR OTHER ANEMIAS

Background: FIRST-EXT was a prospective, multicenter, single-arm, open-label extension of the FIRST study in patients with sickle cell disease (SCD) and other transfusion-dependent anemias. In FIRST, patients were randomly assigned 2:1 to receive oral deferiprone (DFP) or parenteral deferoxamine (DFO) for 12 months. The noninferiority of DFP versus DFO was previously reported.1 Here we report the efficacy and safety of DFP in FIRST-EXT. Aims: To evaluate the long-term efficacy and safety of DFP in iron-overloaded patients with SCD or other anemias. Methods: Patients who completed FIRST could enter FIRST-EXT for up to 2 years. Patients previously treated with DFP continued on DFP (DFP-DFP), while those previously treated with DFO were switched to DFP (DFO-DFP). Baseline was defined as the start of FIRST for DFP-DFP patients, and the start of FIRST-EXT for DFO-DFP patients. Efficacy endpoints were yearly changes from baseline in liver iron concentration (LIC), cardiac MRI T2*, and serum ferritin (SF). We also report adverse drug reactions (ADRs), defined as adverse events at least possibly related to DFP. All patients provided informed consent or assent. Results: Patients (N=134: 89 DFP-DFP; 45 DFP-DFO) were 60.4% male, with a mean (SD) age of 16.2 (8.6) years. Most (85.8%) had SCD; 14.2% had other anemias. At baseline, all patients had elevated (≥1.8 mg/g dry weight [dw]) LIC; all but one had elevated (females >300 µg/L, males >400 µg/L) SF; and all had cardiac MRI T2* in the normal range (≥20 ms). A significant, progressive decline was seen in LIC, with mean (SD) changes from baseline to years 1, 2, and 3 of -2.64 (4.64), -3.91 (6.38), and -6.64 (7.72) mg/g dw, respectively (P<0.01 for all). A decline was also seen in SF, with mean (SD) changes from baseline to years 1, 2, and 3 of -1 (1986), -771 (2171), and -1016 (3617) µg/L, respectively (P<0.05 for years 2 and 3). Cardiac MRI T2* values changed little from baseline. The most frequent ADRs were neutropenia (9.0%), decreased neutrophil count (9.0%), and abdominal pain (7.5%); 2 patients (1.5%) experienced agranulocytosis. One patient withdrew due to ADRs of thrombocytopenia and neutropenia, which resolved. Another patient withdrew due to generalized edema and died for reasons unknown 17 days after withdrawal from the study. Conclusions: DFP long-term use (≤3 years) was effective in controlling body iron load in patients with SCD and other anemias. There were no new safety concerns.

Deferiprone vs deferoxamine for transfusional iron overload in SCD and other anemias: a randomized, open-label noninferiority study

Many people with sickle cell disease (SCD) or other anemias require chronic blood transfusions, which often causes iron overload that requires chelation therapy. The iron chelator deferiprone is frequently used in individuals with thalassemia syndromes, but data in patients with SCD are limited. This open-label study assessed the efficacy and safety of deferiprone in patients with SCD or other anemias receiving chronic transfusion therapy. A total of 228 patients (mean age: 16.9 [range, 3-59] years; 46.9% female) were randomized to receive either oral deferiprone (n = 152) or subcutaneous deferoxamine (n = 76). The primary endpoint was change from baseline at 12 months in liver iron concentration (LIC), assessed by R2* magnetic resonance imaging (MRI). The least squares mean (standard error) change in LIC was −4.04 (0.48) mg/g dry weight for deferiprone vs −4.45 (0.57) mg/g dry weight for deferoxamine, with noninferiority of deferiprone to deferoxamine demonstrated by analysis of covariance (least squares mean difference 0.40 [0.56]; 96.01% confidence interval, −0.76 to 1.57). Noninferiority of deferiprone was also shown for both cardiac T2* MRI and serum ferritin. Rates of overall adverse events (AEs), treatment-related AEs, serious AEs, and AEs leading to withdrawal did not differ significantly between the groups. AEs related to deferiprone treatment included abdominal pain (17.1% of patients), vomiting (14.5%), pyrexia (9.2%), increased alanine transferase (9.2%) and aspartate transferase levels (9.2%), neutropenia (2.6%), and agranulocytosis (0.7%). The efficacy and safety profiles of deferiprone were acceptable and consistent with those seen in patients with transfusion-dependent thalassemia. This trial study was registered at www://clinicaltrials.gov as #NCT02041299.

Randomized Controlled Trial of the Efficacy and Safety of Deferiprone: Subgroup Analysis of Pediatric Patients in Iron-Overloaded Patients with Sickle Cell Disease and Other Anemias

Randomized Controlled Trial of the Efficacy and Safety of Deferiprone: Subgroup Analysis of Pediatric Patients in Iron-Overloaded Patients with Sickle Cell Disease and Other Anemias

Safety of Deferiprone in Individuals with Sickle Cell Disease

Background: Deferiprone (Ferriprox®) is an oral iron chelator approved by the US Food and Drug Administration in 2011 for the treatment of transfusional iron overload in patients with thalassemia syndromes with inadequate chelation therapy. It has been used off-label for the treatment of transfusional iron overload in some patients with conditions other than thalassemia. Utilizing a comprehensive deferiprone patient registry, we sought to assess the safety profile of deferiprone use in patients with SCD.Methods: Since 2011, a patient registry has been maintained for all US patients who receive deferiprone, through a comprehensive support program, “Ferriprox Total Care” run by a central pharmacy. At registry entry, patient information is collected, including age, sex, and ICD-10 code for disease classification. The registry also collects all adverse effects (AEs) reported during deferiprone use through a detailed active drug surveillance program. Prior to initial drug shipment, patients are counseled by a pharmacist about the drug, invited to ask questions, and encouraged to contact the program with questions or safety concerns. Thereafter, the patient/family is contacted monthly to inquire about general well-being, which includes questions on dose adjustments, amount of drug left, hospitalizations, and side effects. Any patient reporting an AE is referred to a pharmacist, who collects additional information on the AE. All AEs are de-identified and reported to the Medical Safety department at ApoPharma. For each AE, the pharmacist and/or ApoPharma assigns causality to deferiprone, and the most conservative assessment prevails. This study reviewed all registry data through March 2016 to determine real-world use safety profile of deferiprone in patients with SCD.Results: A total of 291 patients with SCD, 43% male, received deferiprone. The mean age at drug initiation was 29.5 (SD 15.7) years (y) and 79 (27%) were younger than 18y. The mean drug exposure per patient was 1.3y (SD 1; range 0 to 4.1y). At the time of analysis, 117 (40%) remained on drug, 162 (56%) had discontinued treatment, and 12 (4%) were on hold in the registry for various reasons. 10% discontinued for AEs; AEs most commonly associated with discontinuation were nausea (48%), vomiting (31%), and sickle cell crisis (14%). Eighteen percent discontinued due to physician discretion and 10% due to switching to another chelator. Nine (3%) deaths were reported. All four deaths with follow-up information were deemed unrelated to Ferriprox use. Cause of death in the other five cases was not provided. Overall, a total of 275 AEs assessed as at least possibly related to deferiprone occurred in 112 patients (38.5%). These AEs are referred to as adverse drug reactions (ADRs). Gastrointestinal ADRs were most common including nausea (11%), vomiting (7%), and abdominal discomfort (6%). Arthralgia occurred in 6 patients (2%). Elevation in hepatic enzymes was noted in 6 patients (2.1%). In 61 patients (21%) 133 episodes of sickle cell crisis (36.44 events/100 pt years) were reported as serious AEs; five of these episodes were assessed as possibly related to deferiprone. Agranulocytosis (ANC <0.5 x109/L) developed in 2 (0.7%) patients (0.55 per 100 pt years) and milder neutropenia (ANC <1.5 x 109/L, but ≥ 0.5 x 109/L) in 3 (1%; 0.82 per 100 pt years).Conclusions: Deferiprone is frequently prescribed for patients with SCD. Agranulocytosis occurred in 0.7%, comparable to the rate of 1.7% seen in thalassemia. Overall, the adverse effect profile in patients with SCD is consistent with its known safety profile in thalassemia. DisclosuresKwiatkowski:Bluebird Bio: Research Funding; Apopharma: Research Funding; Novartis: Research Funding; Agios: Consultancy, Honoraria; Ionis: Consultancy, Honoraria. Tricta:ApoPharma: Employment. Rozova:ApoPharma: Employment.

Randomized Controlled Trial of the Efficacy and Safety of Deferiprone in Iron-Overloaded Patients with Sickle Cell Disease or Other Anemias

Randomized Controlled Trial of the Efficacy and Safety of Deferiprone in Iron-Overloaded Patients with Sickle Cell Disease or Other Anemias

S144 A MULTICENTRE, RANDOMIZED, NON‐INFERIORITY TRIAL COMPARING THE EFFICACY OF DEFERIPRONE VERSUS DEFERASIROX IN PEDIATRIC PATIENTS AFFECTED BY TRANSFUSION‐DEPENDENT HEMOGLOBINOPATHIES (DEEP‐2 TRIAL)

Background:While the efficacy and safety of deferiprone (DFP) in adult patients with transfusion‐dependent hemoglobinopathies (TDH) has been extensively studied, data in children are sparse.Aims:We report results of DEferiprone Evaluation in Pediatrics‐2 (DEEP‐2), (EudraCT Number 2012‐000353‐31), a Phase III multicentre, randomized, open label study funded by the EU FP7 Research Program, to compare the efficacy of DFP liquid formulation versus deferasirox dispersible tablets (DFX).Methods:DEEP‐2 was a non‐inferiority, 12‐months, active‐controlled trial that included TDH patients, including sickle cell disease (SCD), of 1 month to &lt; 18 years of age. The primary composite efficacy endpoint was treatment success rate based on changes in serum ferritin (SF) levels (all patients) and cardiac iron concentration (MRI‐T2∗) (patients &gt;10 years able to undergo MRI). Predefined criteria (Figure) were used to determine treatment success for the per protocol (PP) population. The secondary endpoints were changes in SF level, cardiac MRI‐T2∗, liver iron concentration‐R2 (LIC‐R2) and safety profile. Non‐inferiority (NI) was based on the 2‐sided 95% confidence interval (CIL, CIU) of the difference in the success rate between the two arms and was established if CIL was greater than −0.125. In the intention‐to‐treat (ITT) analysis, treatment failure was strictly assigned to patients who withdrew from the study, irrespective of the ferritin response observed at the time of the study suspension.Results:21 sites from 7 countries participated in the trial between 3/2014 and 9/2017. 393 patients were randomized (1:1 DFP vs. DFX) and included 117 patients &lt; 6 years of age (30%), 38 with non β‐thalassemia TDH (mainly SCD) and 54 chelation‐naïve. DFP was non‐inferior to DFX based on the PP population (n = 271) while the NI criterion was not met for the ITT population (n = 390) due mainly to more patient withdrawals in the DFP arm, which were not mandated by the protocol (Figure). After 1year treatment (PP population) SF changed from 2468 to 2120 ng/ml in DFP arm and from 2822 to 2328 ng/ml in the DFX arm. Cardiac MRI T2∗ values changed from 31.3 to 32.4ms and from 30.8 to 32.0ms in DFP and DFX arm, respectively. Statistical analysis of the change in SF at the end of treatment also showed NI of DFP for the PP population (δ = −20, CIU = 317 ng/ml; NI criterion: &lt;400 ng/ml), but not for the ITT population when missing SF levels were not analyzed. No significant differences were seen between treatment arms for changes in MRI‐T2∗ and LIC. Overall, there were no statistically significant differences between the two arms in serious adverse events (SAEs) and drug‐related SAEs. 18 (9.3%) DFP and 11 (5.5%) DFX‐treated patients were reported with neutropenia, 3 reversible cases of agranulocytosis were detected in DFP arm, and 2 reversible renal failures in DFX arm.Summary/Conclusion:This FP7‐sponsored trial on chelation therapy in pediatric patients with TDH is the largest in this age group and the only one comparing the two oral chelating agents. The results of the study show that 1) oral iron chelation treatment is effective even in very young ages, 2) treatment with DFP was not inferior to DFX in patients who received 12 months treatment, 3) NI was not shown in the ITT analysis partially due to a different rate of withdrawal not necessarily mandated by the protocol, 4) the overall safety profile of both DFP and DFX was acceptable. The results of the DEEP‐2 trial provide evidence to support the use of DFP in pediatric patients.image

Oral deferiprone for iron chelation in people with thalassaemia

Thalassaemia major is a genetic disease characterised by a reduced ability to produce haemoglobin. Management of the resulting anaemia is through red blood cell transfusions.Repeated transfusions result in an excessive accumulation of iron in the body (iron overload), removal of which is achieved through iron chelation therapy. A commonly used iron chelator, deferiprone, has been found to be pharmacologically efficacious. However, important questions exist about the efficacy and safety of deferiprone compared to another iron chelator, desferrioxamine. To summarise data from trials on the clinical efficacy and safety of deferiprone and to compare the clinical efficacy and safety of deferiprone with desferrioxamine for thalassaemia. We searched the Cochrane Cystic fibrosis and Genetic Disorders Group's Haemoglobinopathies trials Register and MEDLINE, EMBASE, CENTRAL (The Cochrane Library), LILACS and other international medical databases, plus registers of ongoing trials and the Transfusion Evidence Library (www.transfusionevidencelibrary.com). We also contacted the manufacturers of deferiprone and desferrioxamine.All searches were updated to 05 March 2013. Randomised controlled trials comparing deferiprone with another iron chelator; or comparing two schedules or doses of deferiprone, in people with transfusion-dependent thalassaemia. Two authors independently assessed trials for risk of bias and extracted data. Missing data were requested from the original investigators. A total of 17 trials involving 1061 participants (range 13 to 213 participants per trial) were included. Of these, 16 trials compared either deferiprone alone with desferrioxamine alone, or a combined therapy of deferiprone and desferrioxamine with either deferiprone alone or desferrioxamine alone; one compared different schedules of deferiprone. There was little consistency between outcomes and limited information to fully assess the risk of bias of most of the included trials.Four trials reported mortality; each reported the death of one individual receiving deferiprone with or without desferrioxamine. One trial reported five further deaths in patients who withdrew from randomised treatment (deferiprone with or without desferrioxamine) and switched to desferrioxamine alone. Seven trials reported cardiac function or liver fibrosis as measures of end organ damage.Earlier trials measuring the cardiac iron load indirectly by magnetic resonance imaging (MRI) T2* signal had suggested deferiprone may reduce cardiac iron more quickly than desferrioxamine. However, a meta-analysis of two trials suggested that left ventricular ejection fraction was significantly reduced in patients who received desferrioxamine alone compared with combination therapy. One trial, which planned five years of follow up, was stopped early due to the beneficial effects of combined treatment compared with deferiprone alone in terms of serum ferritin levels reduction.The results of this and three other trials suggest an advantage of combined therapy over monotherapy to reduce iron stores as measured by serum ferritin. There is, however, no conclusive or consistent evidence for the improved efficacy of combined deferiprone and desferrioxamine therapy over monotherapy from direct or indirect measures of liver iron. Both deferiprone and desferrioxamine produce a significant reduction in iron stores in transfusion-dependent, iron-overloaded people. There is no evidence from randomised controlled trials to suggest that either has a greater reduction of clinically significant end organ damage.Evidence of adverse events were observed in all treatment groups. Occurrence of any adverse event was significantly more likely with deferiprone than desferrioxamine in one trial, RR 2.24 (95% CI 1.19 to 4.23). Meta-analysis of a further two trials showed a significant increased risk of adverse events associated with combined deferiprone and desferrioxamine compared with desferrioxamine alone, RR 3.04 (95% CI 1.18 to 7.83). The most commonly reported adverse event was joint pain, which occurred significantly more frequently in patients receiving deferiprone than desferrioxamine, RR 2.64 (95% CI 1.21 to 5.77). Other common adverse events included gastrointestinal disturbances as well as neutropenia or leucopenia, or both. In the absence of data from randomised controlled trials, there is no evidence to suggest the need for a change in current treatment recommendations; namely that deferiprone is indicated for treating iron overload in people with thalassaemia major when desferrioxamine is contraindicated or inadequate. Intensified desferrioxamine treatment (by either subcutaneous or intravenous route) or use of other oral iron chelators, or both, remains the established treatment to reverse cardiac dysfunction due to iron overload. Indeed, the US Food and Drug Administration (FDA) recently only gave support for deferiprone to be used as a last resort for treating iron overload in thalassaemia, myelodysplasia and sickle cell disease. However, there is evidence that adverse events are increased in patients treated with deferiprone compared with desferrioxamine and in patients treated with combined deferiprone and desferrioxamine compared with desferrioxamine alone. There is an urgent need for adequately-powered, high-quality trials comparing the overall clinical efficacy and long-term outcome of deferiprone with desferrioxamine.

25 Three years follow-up outcome of deferiprone for pantothenate kinase-associated neurodegeneration (PKAN)

Background The results of our previous one-year-treatment study (Clinicaltrials.gov Identifier: NCT00907283) suggested that deferiprone might be effective in reverse iron deposition and improve neurological manifestations in patients with NBIA. We report the clinical and neuroradiological findings of 3 of 9 pts involved in that study (all with PKAN) that completed 3 year’s treatment. Patients and methods Three patients with genetically confirmed PKAN (2 F; 1 M; age ranging 27–32; disease duration 4–14) received deferiprone solution at 15 mg/kg po bid. All patients underwent brain MRI at baseline and at 12 and 36 months’ follow up. The quantitative assessment of brain iron was performed with T2∗ relaxometry, using a gradient multi-echo T2∗ sequence. UPDRS/III, ICARS, and UDRS were administered at baseline and during follow up. Results The 36-month MRI evaluations showed reduced hypointensity in the globus pallidus (GP) of all the patients; the quantitative assessment confirmed a significant increment in the T2∗ value, and hence a reduction of the iron content in the GP of these patients. Clinical rating scales indicated an improvement in motor symptoms in 2 patients. No evident changes were observed in the other patient, who was the most severely affected. Conclusions The data from our 3-year follow-up study confirm the safety of deferiprone as a chelator agent for iron accumulation. The improvement observed in 2 patients and the clinical stabilization of the third suggests that this drug may be useful in the treatment of those neurological manifestations linked with iron accumulation and neurodegeneration.

Efficacy and safety of deferiprone (Ferriprox), an oral iron-chelating agent, in pediatric patients

BackgroundIron overload is a predictable and life-threatening complication in patients dependent on the regular transfusion of RBCs. The aims of this study were to investigate the efficacy and safety of deferiprone in a variety of pediatric hematologic and/or oncologic patients with a high iron overload.MethodsSeventeen patients (age: 1.1-20.4 years; median: 10.6 years) from 7 hospitals who were treated with deferiprone from 2006 to 2009 were enrolled in this study. Medical records of enrolled patients were reviewed retrospectively.ResultsSerum ferritin levels were 4,677.8±1,130.9 µg/L at baseline compared to 3,363.9±1,149.7 µg/L at the end of deferiprone treatment (P=0.033). Only 1 patient developed neutropenia as a complication.ConclusionDeferiprone treatment is relatively safe for pediatric patients suffering from various hematologic and oncologic diseases that require RBC transfusions as part of treatment. However, the potential development of critical complications such as agranulocytosis and/or neutropenia remains a concern.