Agranulocytosis and Thrombocytopenia in Patient With Blackfan-Diamond Anaemia During Oral Chelator Trial

Hoffbrand, A. V.; Bartlett, A.; Veys, PA; O’Connor, Nathan; Kontoghiorghes, George J.

doi:10.1016/s0140-6736(89)90641-7

Cited by 105 publications

(35 citation statements)

References 2 publications

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“…The indications for commencing iron chelation therapy in Blackfan-Diamond anemia are similar to those in TM. The first report of agranulocytosis with DFP was in an adult patient with Blackfan-Diamond anemia, 47 and the drug should be avoided in this condition. 48 There have been no unexpected side effects in chelating DBA patients with DFX, and it was effective at lowering LIC and serum ferritin, although less so than in myelodysplasia.…”

Section: Congenital Anemiasmentioning

confidence: 99%

How I treat transfusional iron overload

Hoffbrand

Taher

Cappellini

2012

Blood

175

150

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Patients with ␤-thalassemia major (TM) and other refractory anemias requiring regular blood transfusions accumulate iron that damages the liver, endocrine system, and most importantly the heart. The prognosis in TM has improved remarkably over the past 10 years. This improvement has resulted from the development of magnetic resonance imaging (MRI) techniques, especially T2*, to accurately measure cardiac and liver iron, and from the availability of 3 iron-chelating drugs. In this article we describe the use of MRI to determine which adult and pediatric patients need to begin iron chelation therapy and to monitor their progress. We summarize the properties of each of the 3 drugs, deferoxamine (DFO), deferiprone (DFP), and deferasirox (DFX), including their efficacy, patient acceptability, and side effects. We describe when to initiate or intensify therapy, switch to another drug, or use combined therapy. We also discuss the management of refractory anemias other than TM that may require multiple blood transfusions, including sickle cell anemia and myelodysplasia.

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Section: Congenital Anemiasmentioning

confidence: 99%

How I treat transfusional iron overload

Hoffbrand

Taher

Cappellini

2012

Blood

175

150

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“…20 In the patient, a 28-year-old female, neutrophils reappeared after 17 days and normalized after 36 days. In addition, a 19-year-old male patient that developed neutropenia that was stable for one year but later progressed into a fatal aplastic anemia has been reported from Venezuela at a medical meeting.…”

Section: Case Reportmentioning

confidence: 92%

Fatal agranulocytosis after deferiprone therapy in a child with Diamond-Blackfan anemia

Henter

Karlén

2007

Blood

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A 10-year-old girl with steroid-resistent Diamond-Blackfan anemia (DBA) developed agranulocytosis 9 weeks after chelation with deferiprone was initiated (45 mg/kg daily, 60% of recommended dose) in addition to her ordinary deferoxamine therapy. The blood counts, checked weekly, dropped markedly between weeks 8 and 9. She rapidly developed a septicemia and was admitted with high fever (40.9°C), white blood cell count 0.4 ؋ 10 9 / L, absolute neutrophil count 0.1 ؋ 10 9 /L and platelets 114 ؋ 10 9 /L. She was administered broad spectrum antibiotics, G-CSF (10 microgram/kg daily) and corticosteroids but remained neutropenic and died 6 weeks after admission. Bone marrow examination day 23 revealed areas with low cellularity (around 30%), but also areas with infiltrates of T cells; granulopoiesis and erythropoiesis were scarce. We conclude that weekly neutrophil monitoring is not sufficient to avoid fatal agranulocytosis. We suggest that deferiprone not be prescribed to DBA patients unless the clinical indications are particularly strong, and that the risk of agranulocytosis in thalassemia patients be carefully considered. (Blood. 2007;109:5157-5159)

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“…While fortunately not observed as commonly as arthro pathy, the most serious adverse effect associated with the administration of deferiprone has been severe neutropenia or agranulocytosis, first reported in 1989 [68] by investiga tors in the United Kingdom and now reported from at least four centers in which trials of deferiprone are ongoing [69][70][71][72]. To date, the number of patients in whom this compli cation has been observed include eight patients whose courses have been published or presented [72], While three of these have had a bone marrow failure syndrome such as myelodysplasia or Diamond-Blackfan anemia, the remain der have thalassemia syndromes with no previous history of bone marrow suppression.…”

Section: Agranulocytosis and Neutropeniamentioning

confidence: 99%

Long-Term Therapy with Deferiprone

Olivieri

1996

Acta Haematol

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Data from several trials have provided direct and supportive evidence for the efficacy of deferiprone in the treatment of iron overload in thalassemia major. Deferiprone has been shown to induce sustained decreases in body iron to concentrations associated with survival free from the complications of iron overload in deferoxamine (DFO)-treated patients. Despite this evidence of efficacy, the risk of agranulocytosis mandates a careful evaluation in patients willing and able to use DFO. The incidence of agranulocytosis associated with deferiprone is under study in a prospective multicenter trial in Canada, Italy and the United States, under corporate sponsorship (Apotex Research, Canada). The results of this study should determine the risk associated with the use of this agent, and may provide the data required for an FDA decision regarding licensing of this agent for the treatment of iron overload, a goal supported by investigators worldwide.

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Agranulocytosis and Thrombocytopenia in Patient With Blackfan-Diamond Anaemia During Oral Chelator Trial

Cited by 105 publications

References 2 publications

How I treat transfusional iron overload

How I treat transfusional iron overload

Fatal agranulocytosis after deferiprone therapy in a child with Diamond-Blackfan anemia

Long-Term Therapy with Deferiprone

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