Rechallenging to Hydroxycarbamide Post Thalidomide Treatment and Response in a Non Transfusion-Dependent Patient, is it Possible?

  • Paolo RicchiEmail author
  • Silvia Costantini
  • Anna Spasiano
  • Patrizia Cinque
  • Tiziana Di Matola
  • Massimiliano Ammirabile
  • Aldo Filosa

Dear Editor

The management of several patients with non transfusion dependent thalassaemia (NTDT) committed to receive regular blood transfusions may be cumbersome because of the occurrence of severe post-transfusion reactions and/or the development of irregular antibodies. We recently reported the case of an untransfusable patient that, because of the inefficacy of the long-term treatment with hydroxicarbamide (HU), was started on continuous low doses of thalidomide as off label therapy, obtaining the improvement in haemoglobin level and in a part of NTDT related complications [1]. Here we report the follow up data related to the interruption of thalidomide treatment, due to side effects, and to the re-challenging to HU.

Case Report

LMC is a 53-year-old female patient attending to our Thalassaemia Unit since she was 4 years old. She was 3‐years‐old when homozygous β‐thalassaemia for β° 39 nonsense C → T mutation with a baseline haemoglobin almost completely (95%) represented by HbF...



  1. 1.
    Ricchi P, Costantini S, Spasiano A, De Dominicis G, Di Matola T, Cinque P, Ammirabile M, Marsella M, Filosa A (2016) The long-term and extensive efficacy of low dose thalidomide in a case of an untransfusable patient with non-transfusion-dependent thalassemia. Blood Cells Mol Dis 57:9–97CrossRefGoogle Scholar
  2. 2.
    Ricchi P, Meloni A, Costantini S, Spasiano A, Di Matola T, Pepe A, Cinque P, Filosa A (2017) Soluble form of transferrin receptor-1 level is associated with the age at first diagnosis and the risk of therapeutic intervention and iron overloading in patients with non-transfusion-dependent thalassemia. Ann Hematol 96:6–1541CrossRefGoogle Scholar
  3. 3.
    Ricchi P, Ammirabile M, Costantini S, Spasiano A, Di Matola T, Verna R, Pepe A, Cinque P, Saporito C, Filosa A, Pagano L (2016) Soluble form of transferrin receptor as a biomarker of overall morbidity in patients with non-transfusion-dependent thalassaemia: a cross-sectional study. Blood Transfus. 14:40–538Google Scholar
  4. 4.
    Mancuso A, Maggio A, Renda D, Di Marzo R, Rigano P (2006) Treatment with hydroxycarbamide for intermedia thalassaemia: decrease of efficacy in some patients during long-term follow up. Br J Haematol 133:6–105CrossRefGoogle Scholar
  5. 5.
    Rivella S (2015) β-thalassemias: paradigmatic diseases for scientific discoveries and development of innovative therapies. Haematologica 100:30–418CrossRefGoogle Scholar
  6. 6.
    Lukacs NW, Strieter RM, Lincoln PM, Brownell E, Pullen DM, Schock HJ, Chensue SW, Taub DD, Kunkel SL (1996) Stem cell factor (c-kit ligand) influences eosinophil recruitment and histamine levels in allergic airway inflammation. J Immunol 156:3945–3951Google Scholar
  7. 7.
    Ricchi P, Ammirabile M, Costantini S, Cinque P, Lanza AG, Spasiano A, Di Matola T, Di Costanzo G, Pagano L, Prossomariti L (2010) The impact of previous or concomitant IFN therapy on deferiprone-induced agranulocytosis and neutropenia: a retrospective study. Expert Opin Drug Saf 9:875–881CrossRefGoogle Scholar

Copyright information

© Indian Society of Hematology and Blood Transfusion 2019

Authors and Affiliations

  1. 1.UOSD Malattie Rare del Globulo RossoAzienda Ospedaliera di Rilievo Nazionale “A.Cardarelli”NaplesItaly
  2. 2.UOC Biochimica ClinicaAORN Ospedali dei ColliNaplesItaly
  3. 3.Laboratory of Clinical Chemistry and MicrobiologyFondazione IRCCS Ca’ Granda Ospedale Maggiore PoliclinicoMilanItaly

Personalised recommendations