Outcome of Treatment with Thalidomide in Transfusion Dependent Thalassemia Patients: A Prospective Study in a Thalassemia Center, Dhaka, Bangladesh

Maksuda Begum; Mir Hasan Md. Moslem; Nurun Nahar Fatema Begum; Md. Zobaidur Rahman

doi:doi:10.11648/j.ajp.20200603.11

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Outcome of Treatment with Thalidomide in Transfusion Dependent Thalassemia Patients: A Prospective Study in a Thalassemia Center, Dhaka, Bangladesh

Maksuda Begum, Mir Hasan Md. Moslem, Nurun Nahar Fatema Begum, Md. Zobaidur Rahman

Published in American Journal of Pediatrics (Volume 6, Issue 3)

Received: 15 March 2020 Accepted: 27 March 2020 Published: 30 April 2020

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Abstract

The aim of the study was to assess the outcome of thalidomide in patients with Thalassemia. Method: We identified the outcome of thalidomide in 51 patients with thalassemia needing blood transfusion. We enlisted thalassemia patients, who received treatment with thalidomide between 01 July 2017 and 29 Feb 2020 in Combined Military Hospital, Dhaka. Thalidomide was used in a reduced dose (2 mg/ kg to 5 mg/kg). Thalidomide had been stopped in those patients who developed complications. The following factors were recorded: age and sex, hemoglobin (Hb) levels, ferritin level, blood transfusion interval before and after treatment with thalidomide. Furthermore, we documented blood transfusion dependence and the complications subsequently. All data were studied using SPSS v 24.0 (SPSS Inc. Chicago, IL, U.S.A.). For patients, a paired sample t-test was used to compare the changes in the mean Hb level, ferritin level and the difference in blood transfusion interval before and after treatment with thalidomide. P < 0.05 was considered statistically significant. Results: The study included 51 patients comprised of 23 females (45.1%) and 28 males (54.9%) with an age ranging from 3 to 24 years (9.75±4.11 years). Amongst them 18% (n=9) were beta-thalassemia and 82% (n=42) were Hb E beta-thalassemia. Data of Hb and ferritin levels before and after treatment were recorded in all 51 patients. Ferritin levels reduced to 20.52% (3258.11±2291.91 ng/ml to 2589.65± 2072.74 ng/ml). Hb level increased by 19.26% (7.04±0.53 g/dL to 8.39±1.44g/dL). We found 18 (35.3%) patients with no requirement of blood transfusion after the treatment. 63% (n=32) of the patients showed no adverse effect of thalidomide after the treatment. 16% (n=8) of the patients showed high ALT (Alaline Aminotransferase) and 6% (n=3) showed excessive sleepiness. Other effects included- high TSH, acute urticaria, restlessness, edema, facial puffiness, palpitation, and vomiting. Conclusion: Treatment with thalidomide considerably improved Hb level, increased blood transfusion interval even resulted to non-dependence on blood transfusion and reduced ferritin levels. More studies are necessary to clarify the possible use of thalidomide in the treatment of thalassemia.

Published in	American Journal of Pediatrics (Volume 6, Issue 3)
DOI	10.11648/j.ajp.20200603.11
Page(s)	168-171
Creative Commons	This is an Open Access article, distributed under the terms of the Creative Commons Attribution 4.0 International License (http://creativecommons.org/licenses/by/4.0/), which permits unrestricted use, distribution and reproduction in any medium or format, provided the original work is properly cited.
Copyright	Copyright © The Author(s), 2020. Published by Science Publishing Group

Keywords

Thalidomide, Transfusion, Thalassemia

References

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[4]	Management of Haemoglobin Disorders (2008) Joint WHO-TIF meeting on management of hemoglobin disorders [Monograph Online] Geneva, World Health Organization.
[5]	Tripette J, Alexy T, Hardy-Dessources MD, Mougenel D, Beltan E, et al. (2009) Red blood cell aggregation, aggregate strength and oxygen transport potential of blood are abnormal in both homozygous sickle cell anemia and sickle-hemoglobin C disease. Haematologica 94: 1060-1065.
[6]	Weiss MJ, dos Santos CO (2009) Chaperoning erythropoiesis. Blood 113: 2136-2144.
[7]	Lemonne N, Charlot K, Waltz X, Ballas SK, Lamarre Y, et al. (2015) Hydroxyurea treatment does not increase blood viscosity and improves red blood cell rheology in sickle cell anemia. Haematologica 100: e383-386.
[8]	Fard AD, Hosseini SA, Shahjahani M, Salari F, Jaseb K (2013) Evaluation of Novel Fetal Hemoglobin Inducer Drugs in Treatment of β-Hemoglobinopathy Disorders. Int J Hematol Oncol Stem Cell Res 7: 47-54.
[9]	Ramanan. V, Kelkar. K. Sci Forschen Journal of Blood Disorders and Medicine Role of Thalidomide in Treatment of Beta. 2017: 8-10.
[10]	Fibach E, Rachmilewitz EA. Pathophysiology and treatment of patients with beta-thalassemia - an update. F1000Research. 2017; 6 (0). doi: 10.12688/f1000research.12688.1).
[11]	Aerbajinai W, Zhu J, Gao Z, et al. Thalidomide induces γ-globin gene expression through increased reactive oxygen species-mediated p38 MAPK signaling and histone H4 acetylation in adult erythropoiesis.
[12]	Moutouh-de Parseval LA, Verhelle D, Glezer E, et al. Pomalidomide and lenalidomide regulate erythropoiesis and fetal haemoglobin production in human CD34+ cells. J Clin Invest. 2008; 118: 248–58.
[13]	Fozza C, Pardini S, Giannico DB, et al. Dramatic erythroid response to low-dose thalidomide in two patients with transfusion independent thalassemia and severe post-transfusional alloimmune hemolysis. Am J Hematol. 2015; 90 (7): E141.
[14]	Jalali Far MA, Dehghani Fard A, Hajizamani S, et al. Thalidomide is more efficient than sodium butyrate in enhancing GATA-1 and EKLF gene expression in erythroid progenitors derived from HSCs with β-globin gene mutation. Int J Hematol Oncol Stem Cell Res. 2016; 10 (1): 37-4.
[15]	Gunaseelan. S and Anand Prakash. Thalidomide-induced Stroke in a Child With Thalassemia. Journal of Pediatric Hematology / Oncology: olters Kluwer Health, Inc. Date: Nov 1, 2017.
[16]	Chen, J, Zhu, W, Cai, N, Bu, S, Li, J, Huang, L. Thalidomide induces haematologic responses in patients with β-thalassaemia. Eur J Haematol. 2017; 99: 437–441. https://doi.org/10.1111/ejh.12955).

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Maksuda Begum, Mir Hasan Md. Moslem, Nurun Nahar Fatema Begum, Md. Zobaidur Rahman. (2020). Outcome of Treatment with Thalidomide in Transfusion Dependent Thalassemia Patients: A Prospective Study in a Thalassemia Center, Dhaka, Bangladesh. American Journal of Pediatrics, 6(3), 168-171. https://doi.org/10.11648/j.ajp.20200603.11

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Maksuda Begum; Mir Hasan Md. Moslem; Nurun Nahar Fatema Begum; Md. Zobaidur Rahman. Outcome of Treatment with Thalidomide in Transfusion Dependent Thalassemia Patients: A Prospective Study in a Thalassemia Center, Dhaka, Bangladesh. Am. J. Pediatr. 2020, 6(3), 168-171. doi: 10.11648/j.ajp.20200603.11

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Maksuda Begum, Mir Hasan Md. Moslem, Nurun Nahar Fatema Begum, Md. Zobaidur Rahman. Outcome of Treatment with Thalidomide in Transfusion Dependent Thalassemia Patients: A Prospective Study in a Thalassemia Center, Dhaka, Bangladesh. Am J Pediatr. 2020;6(3):168-171. doi: 10.11648/j.ajp.20200603.11

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@article{10.11648/j.ajp.20200603.11,
  author = {Maksuda Begum and Mir Hasan Md. Moslem and Nurun Nahar Fatema Begum and Md. Zobaidur Rahman},
  title = {Outcome of Treatment with Thalidomide in Transfusion Dependent Thalassemia Patients: A Prospective Study in a Thalassemia Center, Dhaka, Bangladesh},
  journal = {American Journal of Pediatrics},
  volume = {6},
  number = {3},
  pages = {168-171},
  doi = {10.11648/j.ajp.20200603.11},
  url = {https://doi.org/10.11648/j.ajp.20200603.11},
  eprint = {https://article.sciencepublishinggroup.com/pdf/10.11648.j.ajp.20200603.11},
  abstract = {The aim of the study was to assess the outcome of thalidomide in patients with Thalassemia. Method: We identified the outcome of thalidomide in 51 patients with thalassemia needing blood transfusion. We enlisted thalassemia patients, who received treatment with thalidomide between 01 July 2017 and 29 Feb 2020 in Combined Military Hospital, Dhaka. Thalidomide was used in a reduced dose (2 mg/ kg to 5 mg/kg). Thalidomide had been stopped in those patients who developed complications. The following factors were recorded: age and sex, hemoglobin (Hb) levels, ferritin level, blood transfusion interval before and after treatment with thalidomide. Furthermore, we documented blood transfusion dependence and the complications subsequently. All data were studied using SPSS v 24.0 (SPSS Inc. Chicago, IL, U.S.A.). For patients, a paired sample t-test was used to compare the changes in the mean Hb level, ferritin level and the difference in blood transfusion interval before and after treatment with thalidomide. P < 0.05 was considered statistically significant. Results: The study included 51 patients comprised of 23 females (45.1%) and 28 males (54.9%) with an age ranging from 3 to 24 years (9.75±4.11 years). Amongst them 18% (n=9) were beta-thalassemia and 82% (n=42) were Hb E beta-thalassemia. Data of Hb and ferritin levels before and after treatment were recorded in all 51 patients. Ferritin levels reduced to 20.52% (3258.11±2291.91 ng/ml to 2589.65± 2072.74 ng/ml). Hb level increased by 19.26% (7.04±0.53 g/dL to 8.39±1.44g/dL). We found 18 (35.3%) patients with no requirement of blood transfusion after the treatment. 63% (n=32) of the patients showed no adverse effect of thalidomide after the treatment. 16% (n=8) of the patients showed high ALT (Alaline Aminotransferase) and 6% (n=3) showed excessive sleepiness. Other effects included- high TSH, acute urticaria, restlessness, edema, facial puffiness, palpitation, and vomiting. Conclusion: Treatment with thalidomide considerably improved Hb level, increased blood transfusion interval even resulted to non-dependence on blood transfusion and reduced ferritin levels. More studies are necessary to clarify the possible use of thalidomide in the treatment of thalassemia.},
 year = {2020}
}

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TY - JOUR
T1 - Outcome of Treatment with Thalidomide in Transfusion Dependent Thalassemia Patients: A Prospective Study in a Thalassemia Center, Dhaka, Bangladesh
AU - Maksuda Begum
AU - Mir Hasan Md. Moslem
AU - Nurun Nahar Fatema Begum
AU - Md. Zobaidur Rahman
Y1 - 2020/04/30
PY - 2020
N1 - https://doi.org/10.11648/j.ajp.20200603.11
DO - 10.11648/j.ajp.20200603.11
T2 - American Journal of Pediatrics
JF - American Journal of Pediatrics
JO - American Journal of Pediatrics
SP - 168
EP - 171
PB - Science Publishing Group
SN - 2472-0909
UR - https://doi.org/10.11648/j.ajp.20200603.11
AB - The aim of the study was to assess the outcome of thalidomide in patients with Thalassemia. Method: We identified the outcome of thalidomide in 51 patients with thalassemia needing blood transfusion. We enlisted thalassemia patients, who received treatment with thalidomide between 01 July 2017 and 29 Feb 2020 in Combined Military Hospital, Dhaka. Thalidomide was used in a reduced dose (2 mg/ kg to 5 mg/kg). Thalidomide had been stopped in those patients who developed complications. The following factors were recorded: age and sex, hemoglobin (Hb) levels, ferritin level, blood transfusion interval before and after treatment with thalidomide. Furthermore, we documented blood transfusion dependence and the complications subsequently. All data were studied using SPSS v 24.0 (SPSS Inc. Chicago, IL, U.S.A.). For patients, a paired sample t-test was used to compare the changes in the mean Hb level, ferritin level and the difference in blood transfusion interval before and after treatment with thalidomide. P < 0.05 was considered statistically significant. Results: The study included 51 patients comprised of 23 females (45.1%) and 28 males (54.9%) with an age ranging from 3 to 24 years (9.75±4.11 years). Amongst them 18% (n=9) were beta-thalassemia and 82% (n=42) were Hb E beta-thalassemia. Data of Hb and ferritin levels before and after treatment were recorded in all 51 patients. Ferritin levels reduced to 20.52% (3258.11±2291.91 ng/ml to 2589.65± 2072.74 ng/ml). Hb level increased by 19.26% (7.04±0.53 g/dL to 8.39±1.44g/dL). We found 18 (35.3%) patients with no requirement of blood transfusion after the treatment. 63% (n=32) of the patients showed no adverse effect of thalidomide after the treatment. 16% (n=8) of the patients showed high ALT (Alaline Aminotransferase) and 6% (n=3) showed excessive sleepiness. Other effects included- high TSH, acute urticaria, restlessness, edema, facial puffiness, palpitation, and vomiting. Conclusion: Treatment with thalidomide considerably improved Hb level, increased blood transfusion interval even resulted to non-dependence on blood transfusion and reduced ferritin levels. More studies are necessary to clarify the possible use of thalidomide in the treatment of thalassemia.
VL - 6
IS - 3
ER -

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Author Information

Maksuda Begum

Department of Pediatrics, Combined Military Hospital, Dhaka, Bangladesh
Mir Hasan Md. Moslem

Department of Pediatrics, Combined Military Hospital, Dhaka, Bangladesh
Nurun Nahar Fatema Begum

Department of Pediatrics, Combined Military Hospital, Dhaka, Bangladesh
Md. Zobaidur Rahman

Armed Forces Medical Institute, Dhaka Cantonment, Dhaka, Bangladesh

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Maksuda Begum, Mir Hasan Md. Moslem, Nurun Nahar Fatema Begum, Md. Zobaidur Rahman. (2020). Outcome of Treatment with Thalidomide in Transfusion Dependent Thalassemia Patients: A Prospective Study in a Thalassemia Center, Dhaka, Bangladesh. American Journal of Pediatrics, 6(3), 168-171. https://doi.org/10.11648/j.ajp.20200603.11

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Maksuda Begum; Mir Hasan Md. Moslem; Nurun Nahar Fatema Begum; Md. Zobaidur Rahman. Outcome of Treatment with Thalidomide in Transfusion Dependent Thalassemia Patients: A Prospective Study in a Thalassemia Center, Dhaka, Bangladesh. Am. J. Pediatr. 2020, 6(3), 168-171. doi: 10.11648/j.ajp.20200603.11

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AMA Style

Maksuda Begum, Mir Hasan Md. Moslem, Nurun Nahar Fatema Begum, Md. Zobaidur Rahman. Outcome of Treatment with Thalidomide in Transfusion Dependent Thalassemia Patients: A Prospective Study in a Thalassemia Center, Dhaka, Bangladesh. Am J Pediatr. 2020;6(3):168-171. doi: 10.11648/j.ajp.20200603.11

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@article{10.11648/j.ajp.20200603.11,
  author = {Maksuda Begum and Mir Hasan Md. Moslem and Nurun Nahar Fatema Begum and Md. Zobaidur Rahman},
  title = {Outcome of Treatment with Thalidomide in Transfusion Dependent Thalassemia Patients: A Prospective Study in a Thalassemia Center, Dhaka, Bangladesh},
  journal = {American Journal of Pediatrics},
  volume = {6},
  number = {3},
  pages = {168-171},
  doi = {10.11648/j.ajp.20200603.11},
  url = {https://doi.org/10.11648/j.ajp.20200603.11},
  eprint = {https://article.sciencepublishinggroup.com/pdf/10.11648.j.ajp.20200603.11},
  abstract = {The aim of the study was to assess the outcome of thalidomide in patients with Thalassemia. Method: We identified the outcome of thalidomide in 51 patients with thalassemia needing blood transfusion. We enlisted thalassemia patients, who received treatment with thalidomide between 01 July 2017 and 29 Feb 2020 in Combined Military Hospital, Dhaka. Thalidomide was used in a reduced dose (2 mg/ kg to 5 mg/kg). Thalidomide had been stopped in those patients who developed complications. The following factors were recorded: age and sex, hemoglobin (Hb) levels, ferritin level, blood transfusion interval before and after treatment with thalidomide. Furthermore, we documented blood transfusion dependence and the complications subsequently. All data were studied using SPSS v 24.0 (SPSS Inc. Chicago, IL, U.S.A.). For patients, a paired sample t-test was used to compare the changes in the mean Hb level, ferritin level and the difference in blood transfusion interval before and after treatment with thalidomide. P < 0.05 was considered statistically significant. Results: The study included 51 patients comprised of 23 females (45.1%) and 28 males (54.9%) with an age ranging from 3 to 24 years (9.75±4.11 years). Amongst them 18% (n=9) were beta-thalassemia and 82% (n=42) were Hb E beta-thalassemia. Data of Hb and ferritin levels before and after treatment were recorded in all 51 patients. Ferritin levels reduced to 20.52% (3258.11±2291.91 ng/ml to 2589.65± 2072.74 ng/ml). Hb level increased by 19.26% (7.04±0.53 g/dL to 8.39±1.44g/dL). We found 18 (35.3%) patients with no requirement of blood transfusion after the treatment. 63% (n=32) of the patients showed no adverse effect of thalidomide after the treatment. 16% (n=8) of the patients showed high ALT (Alaline Aminotransferase) and 6% (n=3) showed excessive sleepiness. Other effects included- high TSH, acute urticaria, restlessness, edema, facial puffiness, palpitation, and vomiting. Conclusion: Treatment with thalidomide considerably improved Hb level, increased blood transfusion interval even resulted to non-dependence on blood transfusion and reduced ferritin levels. More studies are necessary to clarify the possible use of thalidomide in the treatment of thalassemia.},
 year = {2020}
}

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