Assessment of Bone Marrow Failure Syndrome Management Outcome in Pediatrics – Saudi Perspective

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Sarwar Naseer Siddiqui
Sergey Pamarenko

Abstract

Bone Marrow Failure Syndrome (BMFS) is a rare yet severe condition affecting pediatric populations, characterized by a reduced production of hematopoietic lineages leading to pancytopenia. This article explores the multifaceted nature of BMFS in children, its diverse etiologies, treatment modalities, and outcomes, with a particular focus on the Saudi Arabian context. BMFS encompasses both inherited and acquired forms, often presenting diagnostic challenges due to its heterogeneity. Inherited BMFS accounts for 30% of cases and includes rare conditions like Fanconi Anemia and Schwachman-Diamond Syndrome, where treatment approaches vary depending on severity. Acquired BMFS, constituting 70% of cases, may exhibit complete recovery or require prolonged treatment with immunosuppressants. Hematopoietic Stem Cell Transplantation (HSCT) remains a primary treatment option, with outcomes influenced by factors such as donor type and graft success. Saudi Arabia, despite its high-income status, has faced limited data availability on BMFS outcomes, but the number of HSCT procedures performed in the country is steadily increasing. Survival rates in HSCT patients vary based on factors such as donor match and the underlying cause of BMFS. Additionally, the risk of secondary malignancies is relatively high in BMFS patients, adding complexity to long-term management. While Saudi studies indicate survival rates comparable to international standards, challenges in the assessment of BMFS outcomes persist, given the condition's rarity and diversity. This article underscores the importance of continued research and data collection to enhance our understanding and management of BMFS in the pediatric population, both in Saudi Arabia and globally.

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How to Cite
Siddiqui, S. N., & Pamarenko, S. (2023). Assessment of Bone Marrow Failure Syndrome Management Outcome in Pediatrics – Saudi Perspective. Annals of Innovation in Medicine, 1(2). https://doi.org/10.59652/aim.v1i2.52
Section
Literature review

References

H. El Solh, A. Al-Nasser, and P. Kurre, “Bone Marrow Failure Disorders,” in Textbook of Clinical Pediatrics, A. Y. Elzouki, H. A. Harfi, H. M. Nazer, F. B. Stapleton, W. Oh, and R. J. Whitley, Eds. Berlin, Heidelberg: Springer, 2012, pp. 3091–3100. doi: 10.1007/978-3-642-02202-9_333.

H. Fassel and S. Sheth, “Bone Marrow Failure in Children: Approach to Diagnosis and Treatment,” Indian J Pediatr, vol. 87, no. 2, pp. 141–149, Feb. 2020, doi: 10.1007/s12098-019-03066-4.

C. A. Moore and K. Krishnan, “Bone Marrow Failure,” in StatPearls, Treasure Island (FL): StatPearls Publishing, 2022. Accessed: Dec. 09, 2022. [Online]. Available: http://www.ncbi.nlm.nih.gov/books/NBK459249/

N. AlMozain et al., “Spectrum of myelodysplastic syndrome in patients evaluated for cytopenia(s). A Report from a Reference Centre in Saudi Arabia,” Hematology/Oncology and Stem Cell Therapy, Nov. 2020, doi: 10.1016/j.hemonc.2020.11.001.

A. Bawazir, N. Al-Zamel, A. Amen, M. A. Akiel, N. M. Alhawiti, and A. Alshehri, “The burden of leukemia in the Kingdom of Saudi Arabia: 15 years period (1999–2013),” BMC Cancer, vol. 19, no. 1, p. 703, Jul. 2019, doi: 10.1186/s12885-019-5897-5.

A. Locasciulli, “Acquired Aplastic Anemia in Children,” Pediatr-Drugs, vol. 4, no. 11, pp. 761–766, Nov. 2002, doi: 10.2165/00128072-200204110-00008.

K. C. Myers and S. M. Davies, “Hematopoietic Stem Cell Transplantation for Bone Marrow Failure Syndromes in Children,” Biology of Blood and Marrow Transplantation, vol. 15, no. 3, pp. 279–292, Mar. 2009, doi: 10.1016/j.bbmt.2008.11.037.

R. T. Calado and D. V. Clé, “Treatment of inherited bone marrow failure syndromes beyond transplantation,” Hematology Am Soc Hematol Educ Program, vol. 2017, no. 1, pp. 96–101, Dec. 2017.

M. Shaheen et al., “Hematopoietic stem cell transplantation in Saudi Arabia between 1984 and 2016: Experience from four leading tertiary care hematopoietic stem cell transplantation centers,” Hematology/Oncology and Stem Cell Therapy, vol. 14, no. 3, pp. 169–178, Sep. 2021, doi: 10.1016/j.hemonc.2020.07.008.

M. Cancio et al., “Outcomes in Hematopoietic Stem Cell Transplantation for Congenital Amegakaryocytic Thrombocytopenia,” Transplant Cell Ther, vol. 28, no. 2, p. 101.e1-101.e6, Feb. 2022, doi: 10.1016/j.jtct.2021.10.009.

C. Dufour et al., “Outcome of aplastic anaemia in children. A study by the severe aplastic anaemia and paediatric disease working parties of the European group blood and bone marrow transplant,” British Journal of Haematology, vol. 169, no. 4, pp. 565–573, 2015, doi: 10.1111/bjh.13297.

Y. J. Lim et al., “Poor outcome after hematopoietic stem cell transplantation of patients with unclassified inherited bone marrow failure syndromes,” European Journal of Haematology, vol. 108, no. 4, pp. 278–287, 2022, doi: 10.1111/ejh.13733.

L. T. Aljudaibi et al., “Survival outcomes of pediatric hematopoietic stem cell transplant patients admitted to the intensive care unit: A case–control study from a tertiary care center in Saudi Arabia,” Journal of Applied Hematology, vol. 13, no. 4, p. 192, 2022.