Deviating from safety guidelines during deferiprone therapy in clinical practice may not be associated with higher risk of agranulocytosis

Mohssen Elalfy, Yasser A. Wali, Mohamad Qari, Ghazi Al Damanhouri, Youssef Al-Tonbary, Dilek Yazman, Zakaria Al Hawsawi, Zeynep Karakas, Yurdanur Kilinc, M. Akif Yesilipek, Mohamed Badr, Usama Elsafy, Mostafa Salama, Yousryeia Abdel Rahman, Shebl Shebl, Anne Stilman, Noemi Toiber Temin, Fernando Tricta

Research output: Contribution to journalArticle

12 Citations (Scopus)

Abstract

Background: A risk associated with the iron chelator deferiprone is the development of neutropenia or agranulocytosis. Accordingly, the product label recommends weekly blood monitoring and immediate interruption of treatment upon detection of an absolute neutrophil count (ANC) 9/L, out of concern that continued therapy might lead to a more severe drop. However, it is uncertain how these recommendations are followed under real-life conditions and, if they are not followed, whether continuation of therapy results in increased incidence of agranulocytosis. Procedure: This non-interventional surveillance program assessed the monitoring of deferiprone therapy in clinical practice. A total of 294 patients with transfusion-dependent anemias received deferiprone, as monotherapy or with another chelator, for up to 1 year. The participating physicians were not given any instructions about treatment and monitoring beyond being referred to the information in the package insert. Results: ANC monitoring was conducted at an average interval of 5±4 weeks, and deferiprone was not always interrupted upon detection of neutropenia. One patient (0.3%) experienced agranulocytosis, and nine others (3%) experienced a total of 11 episodes of neutropenia. All neutropenia episodes resolved; median time to resolution was similar whether or not treatment was interrupted; and no case of neutropenia progressed to agranulocytosis. Conclusions: These data indicate that less frequent ANC monitoring and continuation of deferiprone therapy during neutropenia are not associated with prolonged neutropenia or with progression to agranulocytosis.

Original languageEnglish
Pages (from-to)879-884
Number of pages6
JournalPediatric Blood and Cancer
Volume61
Issue number5
DOIs
Publication statusPublished - 2014

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Agranulocytosis
Neutropenia
Guidelines
Safety
Neutrophils
Chelating Agents
Therapeutics
Product Labeling
deferiprone
Anemia
Iron
Physicians
Incidence

Keywords

  • Agranulocytosis
  • Deferiprone
  • Hemoglobinopathies
  • Neutropenia
  • Red blood cell disorders
  • Thalassemia

ASJC Scopus subject areas

  • Oncology
  • Pediatrics, Perinatology, and Child Health
  • Hematology

Cite this

Deviating from safety guidelines during deferiprone therapy in clinical practice may not be associated with higher risk of agranulocytosis. / Elalfy, Mohssen; Wali, Yasser A.; Qari, Mohamad; Al Damanhouri, Ghazi; Al-Tonbary, Youssef; Yazman, Dilek; Al Hawsawi, Zakaria; Karakas, Zeynep; Kilinc, Yurdanur; Yesilipek, M. Akif; Badr, Mohamed; Elsafy, Usama; Salama, Mostafa; Abdel Rahman, Yousryeia; Shebl, Shebl; Stilman, Anne; Toiber Temin, Noemi; Tricta, Fernando.

In: Pediatric Blood and Cancer, Vol. 61, No. 5, 2014, p. 879-884.

Research output: Contribution to journalArticle

Elalfy, M, Wali, YA, Qari, M, Al Damanhouri, G, Al-Tonbary, Y, Yazman, D, Al Hawsawi, Z, Karakas, Z, Kilinc, Y, Yesilipek, MA, Badr, M, Elsafy, U, Salama, M, Abdel Rahman, Y, Shebl, S, Stilman, A, Toiber Temin, N & Tricta, F 2014, 'Deviating from safety guidelines during deferiprone therapy in clinical practice may not be associated with higher risk of agranulocytosis', Pediatric Blood and Cancer, vol. 61, no. 5, pp. 879-884. https://doi.org/10.1002/pbc.24920
Elalfy, Mohssen ; Wali, Yasser A. ; Qari, Mohamad ; Al Damanhouri, Ghazi ; Al-Tonbary, Youssef ; Yazman, Dilek ; Al Hawsawi, Zakaria ; Karakas, Zeynep ; Kilinc, Yurdanur ; Yesilipek, M. Akif ; Badr, Mohamed ; Elsafy, Usama ; Salama, Mostafa ; Abdel Rahman, Yousryeia ; Shebl, Shebl ; Stilman, Anne ; Toiber Temin, Noemi ; Tricta, Fernando. / Deviating from safety guidelines during deferiprone therapy in clinical practice may not be associated with higher risk of agranulocytosis. In: Pediatric Blood and Cancer. 2014 ; Vol. 61, No. 5. pp. 879-884.
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abstract = "Background: A risk associated with the iron chelator deferiprone is the development of neutropenia or agranulocytosis. Accordingly, the product label recommends weekly blood monitoring and immediate interruption of treatment upon detection of an absolute neutrophil count (ANC) 9/L, out of concern that continued therapy might lead to a more severe drop. However, it is uncertain how these recommendations are followed under real-life conditions and, if they are not followed, whether continuation of therapy results in increased incidence of agranulocytosis. Procedure: This non-interventional surveillance program assessed the monitoring of deferiprone therapy in clinical practice. A total of 294 patients with transfusion-dependent anemias received deferiprone, as monotherapy or with another chelator, for up to 1 year. The participating physicians were not given any instructions about treatment and monitoring beyond being referred to the information in the package insert. Results: ANC monitoring was conducted at an average interval of 5±4 weeks, and deferiprone was not always interrupted upon detection of neutropenia. One patient (0.3{\%}) experienced agranulocytosis, and nine others (3{\%}) experienced a total of 11 episodes of neutropenia. All neutropenia episodes resolved; median time to resolution was similar whether or not treatment was interrupted; and no case of neutropenia progressed to agranulocytosis. Conclusions: These data indicate that less frequent ANC monitoring and continuation of deferiprone therapy during neutropenia are not associated with prolonged neutropenia or with progression to agranulocytosis.",
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AU - Elalfy, Mohssen

AU - Wali, Yasser A.

AU - Qari, Mohamad

AU - Al Damanhouri, Ghazi

AU - Al-Tonbary, Youssef

AU - Yazman, Dilek

AU - Al Hawsawi, Zakaria

AU - Karakas, Zeynep

AU - Kilinc, Yurdanur

AU - Yesilipek, M. Akif

AU - Badr, Mohamed

AU - Elsafy, Usama

AU - Salama, Mostafa

AU - Abdel Rahman, Yousryeia

AU - Shebl, Shebl

AU - Stilman, Anne

AU - Toiber Temin, Noemi

AU - Tricta, Fernando

PY - 2014

Y1 - 2014

N2 - Background: A risk associated with the iron chelator deferiprone is the development of neutropenia or agranulocytosis. Accordingly, the product label recommends weekly blood monitoring and immediate interruption of treatment upon detection of an absolute neutrophil count (ANC) 9/L, out of concern that continued therapy might lead to a more severe drop. However, it is uncertain how these recommendations are followed under real-life conditions and, if they are not followed, whether continuation of therapy results in increased incidence of agranulocytosis. Procedure: This non-interventional surveillance program assessed the monitoring of deferiprone therapy in clinical practice. A total of 294 patients with transfusion-dependent anemias received deferiprone, as monotherapy or with another chelator, for up to 1 year. The participating physicians were not given any instructions about treatment and monitoring beyond being referred to the information in the package insert. Results: ANC monitoring was conducted at an average interval of 5±4 weeks, and deferiprone was not always interrupted upon detection of neutropenia. One patient (0.3%) experienced agranulocytosis, and nine others (3%) experienced a total of 11 episodes of neutropenia. All neutropenia episodes resolved; median time to resolution was similar whether or not treatment was interrupted; and no case of neutropenia progressed to agranulocytosis. Conclusions: These data indicate that less frequent ANC monitoring and continuation of deferiprone therapy during neutropenia are not associated with prolonged neutropenia or with progression to agranulocytosis.

AB - Background: A risk associated with the iron chelator deferiprone is the development of neutropenia or agranulocytosis. Accordingly, the product label recommends weekly blood monitoring and immediate interruption of treatment upon detection of an absolute neutrophil count (ANC) 9/L, out of concern that continued therapy might lead to a more severe drop. However, it is uncertain how these recommendations are followed under real-life conditions and, if they are not followed, whether continuation of therapy results in increased incidence of agranulocytosis. Procedure: This non-interventional surveillance program assessed the monitoring of deferiprone therapy in clinical practice. A total of 294 patients with transfusion-dependent anemias received deferiprone, as monotherapy or with another chelator, for up to 1 year. The participating physicians were not given any instructions about treatment and monitoring beyond being referred to the information in the package insert. Results: ANC monitoring was conducted at an average interval of 5±4 weeks, and deferiprone was not always interrupted upon detection of neutropenia. One patient (0.3%) experienced agranulocytosis, and nine others (3%) experienced a total of 11 episodes of neutropenia. All neutropenia episodes resolved; median time to resolution was similar whether or not treatment was interrupted; and no case of neutropenia progressed to agranulocytosis. Conclusions: These data indicate that less frequent ANC monitoring and continuation of deferiprone therapy during neutropenia are not associated with prolonged neutropenia or with progression to agranulocytosis.

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KW - Hemoglobinopathies

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KW - Red blood cell disorders

KW - Thalassemia

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