COMPARISON OF DIFFERENT TREATMENT MODALITIES OF CHELATION THERAPY IN BETA THALASSEMIA MAJOR PATIENTS

Authors

  • Ghazal Usman Shaikh Zayed Hospital Lahore-Pakistan
  • Mahfooz ur Rehman Punjab Institute of Cardiology Lahore-Pakistan
  • Shifa Khalil Shaikh Zayed Hospital Lahore-Pakistan
  • Muhammad Sarmad Hafeez Information Technology University Lahore-Pakistan

Keywords:

Thalassemia, iron chelating, agents, Deferoxamine, Drug therapy, Combination

Abstract

Background: Thalassemia has high prevalence and carrier rate of 8 – 10% in Pakistan, repeated blood  transfusions lead to iron  deposition  in  organs. In this Prospective study we have compared  the efficacy  of three  chelation  regimens being used in  our country. It has been conducted at PBTS, Fatmid  Foundation and Children   Hospital  Lahore. Methods:  60 thalassemia  major  patients, were divided  into  3  groups according  to  their  mode  of chelation.  Patients in group  I  were on oral  iron  chelator  deferiprone, 7  days  per  week. Thalassemics  in  group  II  were on parenteral  iron  chelator  deferoxamine  given  subcutaneously  for  4  days in a week, and group  III  patients  were on  combination  therapy, deferiprone for 5 days & deferoxamine  given  twice  weekly.  The  assessment  of  chelation  was  done  by  measurement  of  serum  ferritin and  24  hour urinary  iron  excretion  at  the  start  of study  and then  after  six  months  of  follow  up.  To  assess  the  hepatic  iron,  hepatic  MRI  were  also  performed.  Results:   Ferritin  levels were  maximally  decreased  in  group  II,  followed  by  group  III, with  no  significant  reduction in group  I. However  statistically  significant  difference  in  mean  urinary  iron  excretion (increased) was  seen  in  group III. The  hepatic  iron  was  very  high  in  all  three  groups  as  shown  by  the  hepatic  MRI. Conclusion: Combination  chelation  therapy  is  the  most  effective  chelation  therapy  in  iron  overloaded patients. It helps  improve  the  compliance and increases urinary iron excretion. Patients on DFX  have  lowest  degree  of  hepatic  siderosis   even  though  it is  considerably  higher  than  normal  population.

Author Biographies

Ghazal Usman, Shaikh Zayed Hospital Lahore-Pakistan

 

Mahfooz ur Rehman, Punjab Institute of Cardiology Lahore-Pakistan

 

Shifa Khalil, Shaikh Zayed Hospital Lahore-Pakistan

 

Muhammad Sarmad Hafeez, Information Technology University Lahore-Pakistan

Project Manager

References

Waheed N, Ali S, Butt MA. Comparison of deferiprone and deferrioxamine for treatment of transfusional iron overload in children with beta thalassemia major. J Ayub Med Coll Abbottabad 2014;26(3):297–300.

Settin AA, Al-Haggar MM, Neamatallah M, Al-Said AM, Hafez MM. Detection of beta thalassemia mutations using primer specific amplification compared to revised dot blot hybridization technique in Egyptian cases. Hematol 2006;9(3):401–9.

Angastinosis M, Modell B. Global epidemiology of hemoglobin disorders. Ann N Y Acad Sci 1998;850:251–69.

Angulo IL, Covas DT, Carneiro AA, Baffa O, Elias Junior J, Vilela G. Determination of iron overload in thalassemia by hepatic MRI and ferritin. Rev Bras Hematol E Hemoter 2008;30:449–52.

Ceci A, Felisi M, De Sanctis V, De Mattia D. Pharmacotherapy of iron overload in thalassemia patients. Expert Opin Pharmacother 2003;4:1763–74.

Alústiza Echeverría JM, Castiella Eguzkiza A, Zapata Morcillo E, Jáuregui Garmendia L, Gabilondo Aguirregabiria A, Paloc C. Quantification of liver iron concentration using 1-Telsa MRI. Radiologica, 2000;50(4):303–7.

De Sanctis, Katz, M, Vullo C, Bagni B, Ughi M, Wonke B. Effect of different treatment regimes on linear growth and final height in beta thalassemia major. Clin Endocrinol (Oxf) 1994;40(6):791–8.

De Sanctis V, Angola G, Wonke B. The development of Diabetes Mellitus in chronic liver disease in ;long term chelated β thalassemia patients. Postgrad Med J 19986;62(731):831–6.

Gandon Y, Guyades D, Heautot JF. Hemachromatosis: diagnosis and quantification of liver iron with gradient echo MRI imaging. Radiology 1994;193(2):533–8.

Kontoghires GJ, Pattichi K, Hadjigariel M, Kolnagou A. Transfusional iron overload and chelation therapy with desferrioxamine and deferiprone. Transfus Sci 2000;23(3):211–23.

Nadeem I, Khalid H, Muhammad Y, Samina A. Ferritin levels in patients of beta thalassaemia major. Intl J Pathol 2004;2(2):71–4.

Weatherall D, Hoffbrand AV. Hemoglobin and its Inherited disorders. In: Hoffbrand AV, Tuddenham E, Catovsky D, editors. 5th edition. Postgraduate Hematology. Blackwell Publishing Oxford. 2005; p.44–58.

Rahman M, Lodhi Y. Prospects and future of conservative management of beta thalassemia major in a developing country. Pak J Med Sci 2004;20:105–12.

Hoffbrand AV, Cohen, AR, Hershko C. Role of Deferiprone in chelation therapy for transfusional iron overload. Blood 2003;102(1):17–24.

Agarwal MB, Gupte SS, Viswanathan C, Vasandani D, Ramanathan J, Desai N, et al. Long term assessment of efficacy and safety of L1, an oral iron chelator, in transfusion dependent thalassemia: Indian trail. Br J Hematol 1992;82(2):460–6.

Brogan-pignatti C, Cappellini MD, De Stefano P, Del Vecchio GC, Forni GL, Gamberini MR, et al. Cardiac morbidity and mortality in Desferrioxamine and Deferiprone treated patients with thalassemia major. Blood 2006;107(9):3733–7.

Wonke B, Wright C, Hoffbrand AV. Combined therapy with desferrioxamine and deferiprone. Br J Hematol 1988;103(2):361–4.

Gomber S, Saxena R, Madan N. Comparative efficacy desferrioxamine, deferiprone and in combination on iron chelation in thalassemia children, Indian Pediatr 2004;41(1):21–7.

Origa R, Bina P, Agus A, Crobu G, Defraia E, Dessì C, et al. Combined therapy with deferiprone & desferrioxamine in thalassemia major. Hematologica 2005;90(10):1309–14.

Oliveri NF, Brittenham GM. Iron chelation therapy and treatment of thalassemia. Blood 1997;89(3):739–61.

Wood J, Enriquez C, Ghure N, Tyzke M, Carson S, Nelson M, et al. MRI R1 and R2 mapping accurately estimates hepatic iron concentration in transfusion dependent thalassemia and sickle cell patients. Blood 2005;106(4):1460–5.

Ejaz MS, Baloch S, Arif F. Efficacy and adverse effects of oral chelating therapy (deferasirox) in multitransfused Pakistani children with beta thalassemia major. Pak J Med Sci 2015;31(3):621–5.

Sajid R, Ghani F, Adil S, Khurshid M. Oral iron chelation therapy with deferiprone in patients with thalassemia major. J Pak Med Assoc 2009;59(6):388–90.

Published

2024-02-05