CONCLUSIONS & FUTURE SCOPE:
In conclusion, our study identifies that HCT is the largest contributor to healthcare utilization in WAS patients. It also identifies the healthcare disparities based on race and socioeconomic status. There is a disparity in use of splenectomy in our database compared to other studies which suggest a change in practice in this immunodeficient population. We also found that only a few centers across the nation contribute to maximum inpatient admissions for WAS, suggesting need for wider resources for this very rare disease. There is a need for a bigger national database and longitudinal studies to follow these patients and assess for long term complications.
Ethics approval : Not applicable
Consent for publication : Not applicable
Availability of data and material : The data that support the findings of this study are available from KIDS database at www.hcup-us.ahrq.gov/kidoverview.jsp. Restrictions apply to the availability of these data, which were used under license for this study. Data are available
available on request and with permission from HCUP.
Competing interests : None of the authors have any financial or non-financial competing interests to declare.
Funding : The authors have no funding to declare.
Authors’ contributions : JD and SA were responsible for conceptualizing the project with NA and DS. SM and NP were involved in statistical analysis of the dataset. DS and NA were responsible for drafting the manuscript and review of literature. All authors read and approved the final manuscript.
Acknowledgements : To Anjali Shekar, MD for providing consent and access to the KIDS dataset.
REFERENCES :
  1. Ochs HD, Filipovich AH, Veys P, Cowan MJ, Kapoor N. Wiskott­ Aldrich syndrome: diagnosis, clinical and laboratory manifestations, and treatment. Biol Blood Marrow Transplant 2009;15 Suppl 1:84–90.
  2. Massaad MJ, Ramesh N, Geha RS. Wiskott-Aldrich syndrome: a comprehensive review. Ann N Y Acad Sci 2013;1285:26–43.
  3. Buchbinder D, Nugent DJ, Fillipovich AH. Wiskott-Aldrich syndrome: Diagnosis, current management, and emerging treatments. Appl Clin Genet 2014;7:55-66.
  4. Parkman R et al. Complete correction of the Wiskott-Aldrich Syndrome by allogeneic bone-marrow transplantation. N Engl J Med 1978;298:921-927.
  5. Filipovich AH et al. Impact of donor type on outcome of bone marrow transplantation for Wiskott-Aldrich syndrome: collaborative study of the International Bone Marrow Transplant Registry and the National Marrow Donor Program. Blood 2001;97:1598-1603.
  6. Shin CR et al. Outcomes following hematopoietic cell transplantation for Wiskott–Aldrich syndrome. Bone Marrow Transplantation 2012;47:1428-1435.
  7. HCUP Kids’ Inpatient Database (KID). Healthcare Cost and Utilization Project (HCUP). 2006 and 2009. Agency for Healthcare Research and Quality, Rockville, MD. www.hcup-us.ahrq.gov/kidoverview.jsp.
  8. Houchens R. Inferences with HCUP State Databases Final Report. HCUP Methods Series Report. U.S. Agency for Healthcare Research and Quality, 2010. https://www.hcup-us.ahrq.gov/reports/methods/2010_05.pdf
  9. HCUP Quality Control Procedures. Rockville, MD: Agency for Healthcare Research and Quality. https://www.hcup-us.ahrq.gov/db/quality.jsp
  10. Steiner C, Elixhauser A, Schnaier J. The healthcare cost and utilization project: an overview. Eff Clin Pract 2002;5:143-151.
  11. Heckbert SR et al. Comparison of self-report, hospital discharge codes, and adjudication of cardiovascular events in the Women’s Health Initiative. Am J Epidemiol 2004;160:1152-1158.
  12. Prchal JT et al. Wiskott-Aldrich syndrome : cellular impairments and their implication for cellular detection. Blood 1980;56:1048-1054.
  13. Fearson ER, Kohn DB, Winkelstein JA, Vogelstein B, Blaese RM. Carrier detection in the Wiskott Aldrich syndrome. Blood 1988;72:1735-1739.
  14. Greer WL, Kwong PC, Peacocke M, Ip R, Rubin LA, Siminovitch KA. X-chromosome inactivation in Wiskott-Aldrich syndrome: a marker for detection of the carrier state and identification of cell lineages expressing gene defect. Genomics 1989;4:60-67.
  15. Mantuano E et al. Analysis of X-chromosome inactivation in bone marrow precursors from carriers of Wiskott-Aldrich syndrome and X-linked severe combined immunodeficiency : evidence the Wiskott-Aldrich gene is expressed prior to granulocyte-macrophage colony-forming unit. Immunodeficiency 1993;4:271-276.
  16. Wengler G, Gorlin JB, Williamson JM, Rosen FS, Bing DH. Nonrandom inactivation of the X chromosome in early lineage hematopoietic cells in carriers of Wiskott Aldrich syndrome. Blood 1995;85:2471-2477.
  17. Lutskiy MI, Sasahara Y, Kenney DM, Rosen FS, Remold-O’Donnell E. Wiskott-Aldrich syndrome in a female. Blood 2002;100(8):2763-2768.
  18. Tornai I, Kiss A, Laczko J. Wiskott-Aldrich syndrome in a heterozygous carrier woman. Eur J Haematol 1989;42:501-502.
  19. Lin CY, Hsu HC. Acute immune complex mediated glomerulonephritis in a Chinese girl with Wiskott Aldrich syndrome variant. Ann Allergy 1984;53:74-78.
  20. Russell SJ, Nisen PD. Random X chromosome inactivation in a female with a variant of Wiskott Aldrich syndrome. Br J Haematol 1995;90:210-212.
  21. Conley ME, Wang WC, Parolini O, Shapiro DN, Campana D, Siminovitch KA. Atypical Wiskott-Aldrich syndome in a girl. Blood 1992;80:1264-1269.
  22. Parolini O et al. X-linked Wiskott-Aldrich syndrome in a girl. N Engl J Med 1998;338:291-295.
  23. Puck JM, Willard HF. X inactivation in females with X linked disease. N Engl J Med. 1998;338:325-328.
  24. Majhail NS, Mau LW, Denzen EM, Arneson TJ. Costs of autologous and allogeneic hematopoietic cell transplantation in the United States: a study using a large national private claims database. Bone Marrow Transplant 2013;48(2):294-300.
  25. Ballen KK et al. Hospital length of stay in the first 100 days after allogeneic hematopoietic cell transplantation for acute leukemia in remission: comparison among alternative graft sources. Biol Blood Marrow Transplant 2014;20(11):1819-1827.
  26. Cancer Facts & Figures 2019. Atlanta, American Cancer Society (2019). https://www.cancer.org/research/cancer-facts-statistics/all-cancer-facts-figures/cancer-facts-figures-2019.html
  27. National Marrow Donor Program Registry Be A Match Statistics (2016). https://bethematch.org/about-us/financial-information/annual-report/
  28. Barker JN et al. Racial disparities in access to HLA-matched unrelated donor transplants: a prospective 1312-patient analysis. Blood Adv 2019;3(7):939-944.
  29. Sullivan KE, Mullen CA, Blease RM, Winkelstein JA. A multinational survey of the Wiskott-Aldrich syndrome. J Pediatrics 1994;125(6):876-885.
  30. Imai K. et al. Clinical course of patients with WASP  gene mutations. Blood 2004;103(2):456-464.
  31. Dupuis-Girod S et al. Autoimmunity in Wiskott-Aldrich Syndrome: Risk factors, Clinical features and outcome in a single center cohort of 55 patients. Pediatrics 2003;111(5):e622-e627.
  32. Schurman S, Candotti F. Autoimmunity in Wiskott-Aldrich syndrome. Current Opinion in Rheumatology 2003;15(4):446-453.
  33. Mullen CA, Anderson KD, Blease RM. Splenectomy and/or bone marrow transplantation in management of the Wiskott-Aldrich syndrome: long term follow-up of 62 cases. Blood 1993;82(10):2961-2966.
  34. Ozsahin H. et al. Long-term outcome following hematopoietic stem-cell transplantation in Wiskott-Aldrich syndrome: collaborative study of the European Society for Immunodeficiencies and European Group for Blood and Marrow Transplantation. Blood 2008;111(1):439-445.
  35. Moratto D et al. Long-term outcome and lineage-specific chimerism in 194 patients with Wiskott-Aldrich syndrome treated by hematopoietic cell transplantation in the period 1980-2009: an international collaborative study. Blood 2011;118(6):1675-168