Adil, Salman Naseem

Shaikh, Muhammad Shariq

English

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eCommons@AKUDepartment of Pathology and Laboratory Medicine Medical College, PakistanJuly 2015Iron chelation in patients with transfusiondependent thalassemia: an insight on response todeferasiroxMuhammad Shariq ShaikhAga Khan University, muhammad.shariq@aku.eduSalman Naseem AdilFollow this and additional works at: http://ecommons.aku.edu/pakistan_fhs_mc_pathol_microbiolPart of the Microbiology Commons, and the Pathology CommonsRecommended CitationShaikh, M. S., Adil, S. (2015). Iron chelation in patients with transfusion dependent thalassemia: an insight on response to deferasirox.JCPSP: Journal of the College of Physicians and Surgeons Pakistan, 25(7), 553-554.Available at: http://ecommons.aku.edu/pakistan_fhs_mc_pathol_microbiol/467Sir,Situated in South Asia, Pakistan is a home to over 180million populations. With a carrier rate of 5% to 13% inthe country,1 an estimated 5000 to 9000 children areborn annually with beta-thalassemia major. Bonemarrow transplantation is expensive and restricted to acouple of large cities, therefore, majority of children aretreated with regular blood transfusions.Excess iron from transfusions deposits in liver, heart andendocrine glands leading to organ failures if notcontrolled and eliminated by chelation therapy.2Deferoxamine, the parent drug in use for last fourdecades requires parenteral administration whereas,deferiprone although orally active and well proven to becardioprotective,3 requires thrice daily administrationand may cause idiosyncratic reactions like erosivearthritis and agranulocytosis.3 These facts are thereasons for non-compliance and hence poor ironchelation. After the availability of deferasirox in Pakistanin 2008, physicians and patients considered it as ananswer to their miseries. It is orally active and hasbioavailability and half-life suitable for once dailydosing.4 The most common side effects reported withdeferasirox are gastro-intestinal events, skin rash andmild dose-dependent increase in creatinine and hepatictransaminase levels.Our institute has a day-care centre which offers bloodtransfusion facilities to over 200 thalassemia majorpatients. Several thalassemics were started ondeferasirox however, many of them discontinued itbecause of its questionable efficacy. Serum ferritin levelsdid not improve substantially in several patients. As itwas a significant observation, we evaluated the efficacyof deferasirox in these patients. Thalassemics wereincluded who reported a good compliance with themedicine and were free from any liver disease. Baselineserum ferritin levels (before starting deferasirox), dose ofdeferasirox, last available ferritin levels and duration oftreatment were inquired and confirmed from medicalrecords. For assessment of hepatic and renal functions,Alanine Aminotransferase (ALT) and serum creatininelevels respectively were performed before starting thedrug and thence repeated at a variable time period of1 - 6 months. Complete blood counts were evaluated forcytopenias before each transfusion (at 3 - 4 weeksinterval). Furthermore, patients were examined forauditory, ocular, cardiac and gastrointestinal disturban-ces at their visits to outpatient clinic or in the day-care.Due to unavailability of more sophisticated methods likeT2*MRI and Superconducting Quantum InterferenceDevice (SQUID), serum ferritin level was used to assessreduction in iron overload. Parents/guardians of allpatients were counselled in detail regarding possiblebenefits and adverse effects of deferasirox. All thepatients were provided liberty to stop or change themedicine at any time.A total of 63 patients were included for analysis. Themean age was 12.2 ± 7.2 years with male: female ratioof 1:1. The mean serum ferritin level before startingdeferasirox was 5254.5 ± 3540.4 ng/ml (range 1276 -19265) whereas it was 4701.8 ± 2989.8 ng/ml (range538 - 17200) at the time of final analysis. The applicationof paired-samples T-test showed a difference of -552.7 ±1677.2 ng/ml (p=0.01). The mean duration of themedicinal intake was 15.2 ± 15.9 months (range 5 - 65months) and the mean dose of deferasirox was 22.9 ±7.5 mg/kg/day. Ten (16%) patients reported gastro-intestinal disturbance mostly in first few weeks of startingthe drug. No patient developed skin rash. Hepatic, renal,ocular, auditory and cardiac abnormalities were notobserved in any patient.High serum ferritin level in the study cohort reflects thepoor iron chelation in majority of thalassemics in ourcountry.5 Nevertheless in current analysis, deferasiroxresulted in decrease in ferritin levels with continued use(p=0.01) without any major untoward adverse effect. Insearch of an ideal iron chelator, the availability ofdeferasirox generated a hope for proper managementand hence, better lifestyle for thalassemia majorpatients. Based on this observation and its concordancewith the literature,4 we recommend the continued use ofdeferasirox in patients with thalassemia majorparticularly in those who are non-compliant with otherregimens.REFERENCES1. Shariq M, Moiz B, Zaidi N, Azhar WB, Iqbal W, Humaira A,et al. Pre-marital screening for beta-thalassaemia in Pakistan:an insight. J Med Screen 2014; 21:163-4.2. Farmaki K, Tzoumari I, Pappa C. Oral chelators in transfusion-dependent thalassemia major patients may prevent or reverseiron overload complications. Blood Cells Mol Dis 2011; 47:33-40.3. Jamuar SS, Lai AHM. Safety and efficacy of iron chelationtherapy with deferiprone in patients with transfusion-dependent thalassemia. Ther Adv Hematol 2012; 3:299-307.Journal of the College of Physicians and Surgeons Pakistan 2015, Vol. 25 (7): 553-554 553LETTER TO THE EDITORIron Chelation in Patients withTransfusion DependentThalassemia: An Insight onResponse to Deferasirox4. Cappellini MD, Bejaoui M, Agaoglu L, Canatan D, Capra M,Cohen A, et al. Iron chelation with deferasirox in adult andpediatric patients with thalassemia major: efficacy and safetyduring 5 years' follow-up. Blood 2011; 118:884-93.5. Riaz H, Riaz T, Khan MU, Aziz S, Ullah F, Rehman A, et al.Serum ferritin levels, socio-demographic factors anddesferrioxamine therapy in multi-transfused thalassemia majorpatients at a government tertiary care hospital of Karachi,Pakistan. BMC Res Notes 2011; 4:287.Letter to the editor554 Journal of the College of Physicians and Surgeons Pakistan 2015, Vol. 25 (7): 553-554Muhammad Shariq Shaikh and Salman Naseem AdilDepartment of Pathology and Microbiology, The Aga KhanUniversity Hospital, Karachi.Correspondence: Dr. Muhammad Shariq Shaikh, BungalowA-1/D, Block - E, Unit No. 6, Latifabad, Hyderabad, Sindh.E-mail: muhammad.shariq@aku.eduReceived: September 11, 2014;   Accepted: February 11, 2015.

Iron chelation in patients with transfusion dependent thalassemia: an insight on response to deferasirox

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Iron chelation in patients with transfusion dependent thalassemia: an insight on response to deferasirox

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