Síndrome de Wiskott-Aldrich (SWA) atenuada
Acredita-se que a perspectiva em longo prazo seja boa. As complicações em longo prazo incluem risco de sangramento grave e desenvolvimento de autoimunidade.[14]Imai K, Morio T, Zhu Y, et al. Clinical course of patients with WASP gene mutations. Blood. 2004;103:456-464.
http://bloodjournal.hematologylibrary.org/content/103/2/456.long
http://www.ncbi.nlm.nih.gov/pubmed/12969986?tool=bestpractice.com
SWA grave
A perspectiva em longo prazo na ausência de transplante de medula óssea curativo tem melhorado com a introdução de terapia profilática. Os dados são limitados, mas sugerem que a sobrevida na terceira década seja improvável.[14]Imai K, Morio T, Zhu Y, et al. Clinical course of patients with WASP gene mutations. Blood. 2004;103:456-464.
http://bloodjournal.hematologylibrary.org/content/103/2/456.long
http://www.ncbi.nlm.nih.gov/pubmed/12969986?tool=bestpractice.com
Sangramento grave, especialmente sangramento intracraniano, infecções graves e autoimunidade são as principais causas de morbidade e mortalidade.[1]O'Sullivan E, Kinnon C, Brickell P. Wiskott-Aldrich syndrome protein, WASP. Int J Biochem Cell Biol. 1999;31:383-387.
http://www.ncbi.nlm.nih.gov/pubmed/10224664?tool=bestpractice.com
[14]Imai K, Morio T, Zhu Y, et al. Clinical course of patients with WASP gene mutations. Blood. 2004;103:456-464.
http://bloodjournal.hematologylibrary.org/content/103/2/456.long
http://www.ncbi.nlm.nih.gov/pubmed/12969986?tool=bestpractice.com
[7]Litzman J, Jones A, Hann I, et al. Intravenous immunoglobulin, splenectomy, and antibiotic prophylaxis in Wiskott-Aldrich syndrome. Arch Dis Child. 1996;75:436-439.
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC1511781/?tool=pubmed
http://www.ncbi.nlm.nih.gov/pubmed/8957959?tool=bestpractice.com
[28]Dupuis-Girod S, Medioni J, Haddad E, et al. Autoimmunity in Wiskott-Aldrich syndrome: risk factors, clinical features, and outcome in a single-center cohort of 55 patients. Pediatrics. 2003;111:e622-27.
http://www.ncbi.nlm.nih.gov/pubmed/12728121?tool=bestpractice.com
Além disso, neoplasias hematológicas (linfoma e síndromes mielodisplásicas) complicam entre 10% e 20% dos casos.[14]Imai K, Morio T, Zhu Y, et al. Clinical course of patients with WASP gene mutations. Blood. 2004;103:456-464.
http://bloodjournal.hematologylibrary.org/content/103/2/456.long
http://www.ncbi.nlm.nih.gov/pubmed/12969986?tool=bestpractice.com
[7]Litzman J, Jones A, Hann I, et al. Intravenous immunoglobulin, splenectomy, and antibiotic prophylaxis in Wiskott-Aldrich syndrome. Arch Dis Child. 1996;75:436-439.
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC1511781/?tool=pubmed
http://www.ncbi.nlm.nih.gov/pubmed/8957959?tool=bestpractice.com
Transplante de medula óssea pode ser curativo. Taxas de sobrevida de cinco anos melhoraram significativamente durante a última década e agora estão próximas de 90% tanto para irmãos doadores compatíveis quanto para doadores totalmente compatíveis sem relação de parentesco. Transplantes com doadores familiares não compatíveis geralmente são evitados em virtude do histórico de taxas de sobrevida baixas (50% a 55%), embora os desfechos nesse grupo também estejam melhorando.[29]Filipovich AH, Stone JV, Tomany SC, 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.
http://bloodjournal.hematologylibrary.org/content/97/6/1598.long
http://www.ncbi.nlm.nih.gov/pubmed/11238097?tool=bestpractice.com
[30]Ozsahin H, Cavazzana-Calvo M, Notarangelo LD, 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;111:439-445.
http://bloodjournal.hematologylibrary.org/content/111/1/439.long
http://www.ncbi.nlm.nih.gov/pubmed/17901250?tool=bestpractice.com
[31]Moratto D, Giliani S, Bonfim C, et al. Long-term outcome and lineage-specific chimerism in 194 Wiskott-Aldrich syndrome patients treated by hematopoietic cell transplantation between 1980-2009: an international collaborative study. Blood. 2011;118:1675-1684.
http://bloodjournal.hematologylibrary.org/content/118/6/1675.long
http://www.ncbi.nlm.nih.gov/pubmed/21659547?tool=bestpractice.com
[32]Elfeky RA, Furtado-Silva JM, Chiesa R, et al. One hundred percent survival after transplantation of 34 patients with Wiskott-Aldrich syndrome over 20 years. J Allergy Clin Immunol. 2018 Jul 25. pii: S0091-6749(18)31059-5.
http://www.ncbi.nlm.nih.gov/pubmed/30055182?tool=bestpractice.com