Suspect Wiskott-Aldrich syndrome in boys with persistent microthrombocytopenia, eczema, infections, autoimmunity, or lymphoid malignancy; confirm the WAS variant and expedite curative therapy planning, because hematopoietic transplantation outcomes decline with older age and severe pretransplant infection.

Diagnostic Pivot

Recognize the phenotype and confirm a WAS-related disorder

Small platelets in an affected male are the key discriminator.

Evaluate a male patient with persistent thrombocytopenia for platelet size on peripheral smear and complete blood count indices before diagnosing isolated immune thrombocytopenia. The canonical WAS phenotype combines thrombocytopenia with small platelets, eczema, and immune deficiency; recurrent infections, autoimmunity, and lymphoreticular malignancy increase diagnostic concern. fda+3fdaFDA Approves First Gene Therapy Treatment for Wiskott-Aldrich Syndrome | FDA BMJA novel mutation in Wiskott-Aldrich gene manifesting as ...cellEvidence for Long-term Efficacy and Safety of Gene Therapy for ...Wolters KluwerConfirming or Excluding the Diagnosis of Wiskott-Aldrich Syndrome ...

Order molecular testing of the WAS gene to establish the diagnosis. In patients being considered for curative therapy, assess WAS protein in peripheral-blood mononuclear cells by western blotting and flow cytometry; severe disease in a gene-therapy study was defined by clinical severity score 3-5 or absent WAS protein, with molecular confirmation by WAS sequencing. NatureNatureLong-term safety and efficacy of lentiviral hematopoietic stem/progenitor cell gene therapy for Wiskott–Aldrich syndrome | Nature Medicine

Do not exclude a WAS-related disorder solely because eczema or recurrent infections are not prominent. X-linked thrombocytopenia represents a milder phenotype, whereas absent or markedly reduced WAS protein and more severe clinical manifestations favor classic WAS and support early definitive-therapy planning. Nature+2NatureWiskott Aldrich syndrome: healthcare utilizations and disparities in transplant care | Scientific Reports ScienceDirectHow I treat Wiskott-Aldrich syndrome - ScienceDirect WileyPediatric Blood & Cancer - Wiley Online Library

Document bleeding phenotype, eczema burden, serious or recurrent infections, autoimmune manifestations, and prior or current lymphoid malignancy at the first specialty assessment; these features define severity and influence urgency of cellular-therapy evaluation. fda+3fdaFDA Approves First Gene Therapy Treatment for Wiskott-Aldrich Syndrome | FDA JAMAOutcomes of Gene Therapy for Severe Wiskott-Aldrich Syndrome ScienceDirectHow I treat Wiskott-Aldrich syndrome - ScienceDirect WileyPediatric Blood & Cancer - Wiley Online Library
Obtain a detailed family history of thrombocytopenia, bleeding, infections, or early male deaths compatible with X-linked inheritance, but proceed with WAS testing when the clinical phenotype is compelling regardless of family history. BMJ+2BMJA novel mutation in Wiskott-Aldrich gene manifesting as ...cellEvidence for Long-term Efficacy and Safety of Gene Therapy for ...ScienceDirectHow I treat Wiskott-Aldrich syndrome - ScienceDirect

Clinical patterns that should redirect chronic thrombocytopenia evaluation toward WAS-related disease. BMJ+5BMJA novel mutation in Wiskott-Aldrich gene manifesting as ...NatureWiskott Aldrich syndrome: healthcare utilizations and disparities in transplant care | Scientific Reports cellEvidence for Long-term Efficacy and Safety of Gene Therapy for ...Wolters KluwerConfirming or Excluding the Diagnosis of Wiskott-Aldrich Syndrome ...Wolters KluwerAtypical Presentation of Wiskott–Aldrich Syndrome... - Ovid WileyPediatric Blood & Cancer - Wiley Online Library
Finding	Interpretation	Next action
Male patient with thrombocytopenia and small platelets	Characteristic laboratory pattern of classic WAS or X-linked thrombocytopenia. BMJ+2BMJA novel mutation in Wiskott-Aldrich gene manifesting as ...Wolters KluwerConfirming or Excluding the Diagnosis of Wiskott-Aldrich Syndrome ...Wolters KluwerMisdiagnosis of Chronic Thrombocytopenia in Childhood - Ovid	Order WAS gene sequencing and refer to clinical immunology and a cellular-therapy center. Nature+1NatureLong-term safety and efficacy of lentiviral hematopoietic stem/progenitor cell gene therapy for Wiskott–Aldrich syndrome \| Nature Medicine WileyPediatric Blood & Cancer - Wiley Online Library
Thrombocytopenia plus eczema, recurrent infection, autoimmunity, or lymphoreticular malignancy	Favors classic WAS with combined immune dysregulation rather than isolated thrombocytopenia. fda+2fdaFDA Approves First Gene Therapy Treatment for Wiskott-Aldrich Syndrome \| FDA cellEvidence for Long-term Efficacy and Safety of Gene Therapy for ...ScienceDirectHow I treat Wiskott-Aldrich syndrome - ScienceDirect	Assess disease severity and WAS protein expression while beginning transplant or gene-therapy planning. Nature+1NatureLong-term safety and efficacy of lentiviral hematopoietic stem/progenitor cell gene therapy for Wiskott–Aldrich syndrome \| Nature Medicine WileyPediatric Blood & Cancer - Wiley Online Library
Persistent thrombocytopenia with limited infections or eczema	Does not exclude a WAS-related disorder; milder X-linked thrombocytopenia can occur. Nature+2NatureWiskott Aldrich syndrome: healthcare utilizations and disparities in transplant care \| Scientific Reports ScienceDirectHow I treat Wiskott-Aldrich syndrome - ScienceDirect WileyPediatric Blood & Cancer - Wiley Online Library	Confirm the genotype and longitudinally assess for escalation to autoimmunity or malignancy. ScienceDirect+1ScienceDirectHow I treat Wiskott-Aldrich syndrome - ScienceDirect WileyPediatric Blood & Cancer - Wiley Online Library
Macrothrombocytopenia	Atypical for the characteristic microthrombocytopenia of WAS and should broaden the inherited-thrombocytopenia differential. Wolters Kluwer+1Wolters KluwerConfirming or Excluding the Diagnosis of Wiskott-Aldrich Syndrome ...Wolters KluwerAtypical Presentation of Wiskott–Aldrich Syndrome... - Ovid	Reassess smear interpretation and pursue an alternative inherited-thrombocytopenia workup while considering WAS testing if other phenotype features remain persuasive. Wolters Kluwer+1Wolters KluwerConfirming or Excluding the Diagnosis of Wiskott-Aldrich Syndrome ...Wolters KluwerAtypical Presentation of Wiskott–Aldrich Syndrome... - Ovid

Immediate Priorities

Control bleeding and infection risk while definitive therapy is arranged

Supportive management does not replace definitive correction of hematopoietic disease.

Treat active bleeding and clinically significant infection as immediate complications of a combined immunodeficiency with thrombocytopenia, while simultaneously expediting consultation with pediatric hematology, clinical immunology, and a transplant or gene-therapy program. WAS is life-threatening and carries susceptibility to bleeding, recurrent infection, autoimmunity, and lymphoreticular malignancy. fda+1fdaFDA Approves First Gene Therapy Treatment for Wiskott-Aldrich Syndrome | FDA cellEvidence for Long-term Efficacy and Safety of Gene Therapy for ...

Use the initial specialty assessment to identify conditions that worsen cellular-therapy risk: serious infection before hematopoietic cell transplantation was associated with worse overall and event-free survival in the PIDTC cohort. Infection control and stabilization should therefore proceed before conditioning when feasible, without allowing a prolonged workup to defer donor assessment or definitive-therapy referral. ScienceDirectScienceDirectHematopoietic Cell Transplantation for Wiskott-Aldrich syndrome: A PIDTC Report - ScienceDirect

Avoid treating an apparent platelet-count response or dermatologic improvement as evidence that the underlying disorder has resolved. Gene-therapy studies measured improvement in eczema, infections, bleeding tendency, and autoimmunity, reflecting the multisystem disease burden that requires ongoing assessment beyond platelet count alone. JAMA+1JAMAOutcomes of Gene Therapy for Severe Wiskott-Aldrich Syndrome ScienceDirectOutcomes of hematopoietic stem cell gene therapy for Wiskott-Aldrich syndrome - ScienceDirect

At baseline and before cellular therapy, record bleeding events, infection frequency and severity, eczema activity, autoimmune disease, and malignancy history; these outcomes are clinically relevant measures of disease control. fda+2fdaFDA Approves First Gene Therapy Treatment for Wiskott-Aldrich Syndrome | FDA JAMAOutcomes of Gene Therapy for Severe Wiskott-Aldrich Syndrome ScienceDirectOutcomes of hematopoietic stem cell gene therapy for Wiskott-Aldrich syndrome - ScienceDirect
If lymphadenopathy, organ enlargement, unexplained constitutional symptoms, or other concern for lymphoreticular malignancy is present, coordinate urgent hematology-oncology evaluation because malignancy is a recognized WAS complication. fda+2fdaFDA Approves First Gene Therapy Treatment for Wiskott-Aldrich Syndrome | FDA cellEvidence for Long-term Efficacy and Safety of Gene Therapy for ...ScienceDirectHow I treat Wiskott-Aldrich syndrome - ScienceDirect

Management priorities before definitive cellular therapy. fda+3fdaFDA Approves First Gene Therapy Treatment for Wiskott-Aldrich Syndrome | FDA JAMAOutcomes of Gene Therapy for Severe Wiskott-Aldrich Syndrome ScienceDirectHematopoietic Cell Transplantation for Wiskott-Aldrich syndrome: A PIDTC Report - ScienceDirect ScienceDirectOutcomes of hematopoietic stem cell gene therapy for Wiskott-Aldrich syndrome - ScienceDirect
Clinical problem	Decision consequence	Action
Active or prior serious infection	Pre-HCT severe infection is associated with poorer overall and event-free survival. ScienceDirectScienceDirectHematopoietic Cell Transplantation for Wiskott-Aldrich syndrome: A PIDTC Report - ScienceDirect	Treat and stabilize infection promptly while advancing donor search and cellular-therapy consultation. ScienceDirectScienceDirectHematopoietic Cell Transplantation for Wiskott-Aldrich syndrome: A PIDTC Report - ScienceDirect
Bleeding phenotype	Bleeding is a core disease manifestation and a treatment-response outcome. fda+1fdaFDA Approves First Gene Therapy Treatment for Wiskott-Aldrich Syndrome \| FDA JAMAOutcomes of Gene Therapy for Severe Wiskott-Aldrich Syndrome	Coordinate hematology-directed bleeding management and track bleeding tendency longitudinally. fda+1fdaFDA Approves First Gene Therapy Treatment for Wiskott-Aldrich Syndrome \| FDA JAMAOutcomes of Gene Therapy for Severe Wiskott-Aldrich Syndrome
Autoimmunity	Autoimmune disease may persist or flare despite cellular therapy; two patients with prior autoimmunity had post-gene-therapy flares. ScienceDirectScienceDirectOutcomes of hematopoietic stem cell gene therapy for Wiskott-Aldrich syndrome - ScienceDirect	Document active and historical autoimmune disease before therapy and monitor after immune reconstitution. ScienceDirectScienceDirectOutcomes of hematopoietic stem cell gene therapy for Wiskott-Aldrich syndrome - ScienceDirect
Eczema	Eczema is a core manifestation and a measurable response domain after gene therapy. fda+1fdaFDA Approves First Gene Therapy Treatment for Wiskott-Aldrich Syndrome \| FDA JAMAOutcomes of Gene Therapy for Severe Wiskott-Aldrich Syndrome	Treat the skin disease while using eczema severity as one marker of systemic disease control. JAMAJAMAOutcomes of Gene Therapy for Severe Wiskott-Aldrich Syndrome

Curative Therapy

Choose allogeneic transplantation or autologous gene therapy

Donor availability, age, disease severity, and transplant risk drive selection.

Refer patients with severe WAS, particularly those with absent WAS protein, for definitive cellular therapy without delay. Allogeneic hematopoietic stem cell transplantation is regarded as first-line treatment when an HLA-compatible donor is available; outcomes are substantially better when treatment occurs before age 5 years in one reported cohort, with overall survival of 94% before age 5 years versus 66% at older ages. Nature+1NatureLong-term safety and efficacy of lentiviral hematopoietic stem/progenitor cell gene therapy for Wiskott–Aldrich syndrome | Nature Medicine WileyPediatric Blood & Cancer - Wiley Online Library

For patients aged 6 months or older with a WAS mutation in whom HSCT is appropriate but no suitable HLA-matched related donor is available, FDA-approved etuvetidigene autotemcel (Waskyra) provides an autologous genetically corrected hematopoietic stem-cell option. This strategy avoids the requirement for a matched related donor and avoids graft-versus-host disease associated with allogeneic transplantation. fda+1fdaFDA Approves First Gene Therapy Treatment for Wiskott-Aldrich Syndrome | FDA ScienceDirectOutcomes of hematopoietic stem cell gene therapy for Wiskott-Aldrich syndrome - ScienceDirect

Do not restrict allogeneic HCT consideration to children solely on the basis of age. Adult patients may still be candidates when clinically indicated, but age at transplant, donor type, and prior serious infection are important adverse prognostic variables. ScienceDirect+1ScienceDirectAllogeneic hematopoietic stem cell transplantation outcome in oldest known surviving patients with Wiskott-Aldrich syndrome - ScienceDirect ScienceDirectHematopoietic Cell Transplantation for Wiskott-Aldrich syndrome: A PIDTC Report - ScienceDirect

Start HLA donor assessment early in every patient with severe WAS being evaluated for allogeneic HCT. HLA-compatible donor availability is the principal branch point between standard allogeneic transplantation and the FDA-labeled gene-therapy population. fda+1fdaFDA Approves First Gene Therapy Treatment for Wiskott-Aldrich Syndrome | FDA NatureLong-term safety and efficacy of lentiviral hematopoietic stem/progenitor cell gene therapy for Wiskott–Aldrich syndrome | Nature Medicine
Discuss transplantation conditioning as a chimerism and toxicity tradeoff. Reduced-intensity regimens were associated with lower T-cell and myeloid donor chimerism in the PIDTC cohort, particularly with non-busulfan regimens. ScienceDirectScienceDirectHematopoietic Cell Transplantation for Wiskott-Aldrich syndrome: A PIDTC Report - ScienceDirect
Discuss gene therapy as a specialized cellular intervention rather than a nonmyeloablative alternative: reported lentiviral gene-therapy protocols used busulfan and fludarabine conditioning. ScienceDirectScienceDirectOutcomes of hematopoietic stem cell gene therapy for Wiskott-Aldrich syndrome - ScienceDirect

Definitive-therapy selection and post-treatment surveillance considerations. fda+4fdaFDA Approves First Gene Therapy Treatment for Wiskott-Aldrich Syndrome | FDA NatureLong-term safety and efficacy of lentiviral hematopoietic stem/progenitor cell gene therapy for Wiskott–Aldrich syndrome | Nature Medicine ScienceDirectHematopoietic Cell Transplantation for Wiskott-Aldrich syndrome: A PIDTC Report - ScienceDirect ScienceDirectOutcomes of hematopoietic stem cell gene therapy for Wiskott-Aldrich syndrome - ScienceDirect WileyPediatric Blood & Cancer - Wiley Online Library
Option	When to prioritize	Key tradeoff	Monitoring focus
Allogeneic HCT	Severe WAS with an HLA-compatible donor; early treatment is favored because outcomes decline with older age. Nature+2NatureLong-term safety and efficacy of lentiviral hematopoietic stem/progenitor cell gene therapy for Wiskott–Aldrich syndrome \| Nature Medicine ScienceDirectHematopoietic Cell Transplantation for Wiskott-Aldrich syndrome: A PIDTC Report - ScienceDirect WileyPediatric Blood & Cancer - Wiley Online Library	Potential cure with donor-derived hematopoiesis, balanced against graft-versus-host disease, donor factors, conditioning toxicity, and mixed chimerism. Nature+2NatureHematopoietic Cell Transplantation in Wiskott-Aldrich Syndrome \| Transplantation Immunology \| Immunology \| Health sciences \| Topics \| Nature Index NatureLong-term safety and efficacy of lentiviral hematopoietic stem/progenitor cell gene therapy for Wiskott–Aldrich syndrome \| Nature Medicine ScienceDirectOutcomes of hematopoietic stem cell gene therapy for Wiskott-Aldrich syndrome - ScienceDirect	Lineage-specific donor chimerism, platelet count, immune reconstitution, autoimmunity, graft-versus-host disease, and infection. Nature+1NatureLong-term safety and efficacy of lentiviral hematopoietic stem/progenitor cell gene therapy for Wiskott–Aldrich syndrome \| Nature Medicine ScienceDirectHematopoietic Cell Transplantation for Wiskott-Aldrich syndrome: A PIDTC Report - ScienceDirect
Etu vetidigene autotemcel	Patient aged at least 6 months with a WAS mutation, appropriate for HSCT, and without a suitable HLA-matched related donor. fdafdaFDA Approves First Gene Therapy Treatment for Wiskott-Aldrich Syndrome \| FDA	Autologous approach avoids matched-related donor dependence and graft-versus-host disease but requires specialized manufacture, conditioning, and long-term follow-up. fda+1fdaFDA Approves First Gene Therapy Treatment for Wiskott-Aldrich Syndrome \| FDA ScienceDirectOutcomes of hematopoietic stem cell gene therapy for Wiskott-Aldrich syndrome - ScienceDirect	Bleeding, infections, eczema, autoimmunity, platelet count, immune function, and durable multilineage vector marking. JAMA+1JAMAOutcomes of Gene Therapy for Severe Wiskott-Aldrich Syndrome ScienceDirectOutcomes of hematopoietic stem cell gene therapy for Wiskott-Aldrich syndrome - ScienceDirect

Allogeneic hematopoietic cell transplantation

Monitor lineage-specific donor chimerism and platelet recovery after HCT. Low myeloid donor chimerism was associated with lower platelet counts in the PIDTC cohort, and prior work links mixed chimerism to poor immune reconstitution and autoimmunity; myeloid donor engraftment below 50% was associated with persistent thrombocytopenia in a reported cohort. Nature+1NatureLong-term safety and efficacy of lentiviral hematopoietic stem/progenitor cell gene therapy for Wiskott–Aldrich syndrome | Nature Medicine ScienceDirectHematopoietic Cell Transplantation for Wiskott-Aldrich syndrome: A PIDTC Report - ScienceDirect

Counsel families that HCT can be effective but remains vulnerable to donor and conditioning effects. In 308 patients treated from 1990 through 2018, 5-year overall survival was 87.2% and event-free survival was 79.7%; older age, donor type, and pre-HCT severe infection negatively affected both outcomes. ScienceDirectScienceDirectHematopoietic Cell Transplantation for Wiskott-Aldrich syndrome: A PIDTC Report - ScienceDirect

A busulfan-based conditioning regimen was associated with higher post-HCT platelet counts in the PIDTC analysis. ScienceDirectScienceDirectHematopoietic Cell Transplantation for Wiskott-Aldrich syndrome: A PIDTC Report - ScienceDirect
Reduced-intensity conditioning may lower toxicity but carries a chimerism tradeoff that is particularly relevant when durable platelet correction is a treatment objective. ScienceDirectScienceDirectHematopoietic Cell Transplantation for Wiskott-Aldrich syndrome: A PIDTC Report - ScienceDirect

Autologous lentiviral gene therapy

Gene therapy uses autologous CD34-positive hematopoietic cells modified with a lentiviral vector expressing WAS complementary DNA, followed by conditioning and reinfusion. In a phase 1/2 study of five patients with severe WAS treated after busulfan-fludarabine conditioning, all were alive and well with sustained multilineage vector marking at a median 7.6 years of follow-up. ScienceDirectScienceDirectOutcomes of hematopoietic stem cell gene therapy for Wiskott-Aldrich syndrome - ScienceDirect

Set expectations that immune and clinical improvement may exceed platelet correction. In that study, immune function improved despite subphysiologic transgenic WAS protein expression; higher vector copy number in the infused product was associated with more prominent platelet-count and myeloid cytoskeletal improvement. Prior autoimmunity remained clinically relevant because two patients experienced autoimmune flares after gene therapy. ScienceDirectScienceDirectOutcomes of hematopoietic stem cell gene therapy for Wiskott-Aldrich syndrome - ScienceDirect

The FDA indication requires a WAS mutation, age at least 6 months, appropriateness for HSCT, and absence of a suitable HLA-matched related stem-cell donor. fdafdaFDA Approves First Gene Therapy Treatment for Wiskott-Aldrich Syndrome | FDA
Use long-term follow-up to assess bleeding, infections, eczema, autoimmunity, platelet count, and multilineage hematopoietic correction. JAMA+1JAMAOutcomes of Gene Therapy for Severe Wiskott-Aldrich Syndrome ScienceDirectOutcomes of hematopoietic stem cell gene therapy for Wiskott-Aldrich syndrome - ScienceDirect

Follow-up

Monitor disease activity and cellular-therapy durability by lineage and complication

Platelet count alone is insufficient to judge control.

At each longitudinal review, assess the domains that define ongoing WAS morbidity: bleeding tendency, eczema, frequency and severity of infections, autoimmunity, and malignancy. These were the principal clinical outcome domains in gene-therapy evaluation and remain appropriate targets after HCT or autologous therapy. fda+3fdaFDA Approves First Gene Therapy Treatment for Wiskott-Aldrich Syndrome | FDA JAMAOutcomes of Gene Therapy for Severe Wiskott-Aldrich Syndrome cellEvidence for Long-term Efficacy and Safety of Gene Therapy for ...ScienceDirectOutcomes of hematopoietic stem cell gene therapy for Wiskott-Aldrich syndrome - ScienceDirect

After allogeneic HCT, interpret a falling platelet count in the context of donor myeloid chimerism rather than assuming isolated thrombocytopenia. Lower myeloid chimerism was associated with lower platelet counts, while reduced-intensity regimens were associated with lower T-cell and myeloid donor chimerism. ScienceDirectScienceDirectHematopoietic Cell Transplantation for Wiskott-Aldrich syndrome: A PIDTC Report - ScienceDirect

After gene therapy, persistent or recurrent autoimmune disease requires active reassessment even if gene marking and WAS protein-expressing-cell proportions appear comparable to those in patients without autoimmunity. Autoimmune flares occurred in two patients with prior autoimmunity in a long-term phase 1/2 cohort. ScienceDirectScienceDirectOutcomes of hematopoietic stem cell gene therapy for Wiskott-Aldrich syndrome - ScienceDirect

Escalate evaluation for any new lymphadenopathy, systemic symptoms, or organ involvement because WAS confers increased susceptibility to lymphoreticular malignancy. fda+2fdaFDA Approves First Gene Therapy Treatment for Wiskott-Aldrich Syndrome | FDA cellEvidence for Long-term Efficacy and Safety of Gene Therapy for ...ScienceDirectHow I treat Wiskott-Aldrich syndrome - ScienceDirect
Use the patient’s pretherapy phenotype as the comparator: fewer serious infections, reduced bleeding tendency, improved eczema, and controlled autoimmunity are clinically meaningful measures of response. JAMA+1JAMAOutcomes of Gene Therapy for Severe Wiskott-Aldrich Syndrome ScienceDirectOutcomes of hematopoietic stem cell gene therapy for Wiskott-Aldrich syndrome - ScienceDirect

Problem-oriented surveillance after definitive therapy. fda+4fdaFDA Approves First Gene Therapy Treatment for Wiskott-Aldrich Syndrome | FDA JAMAOutcomes of Gene Therapy for Severe Wiskott-Aldrich Syndrome NatureLong-term safety and efficacy of lentiviral hematopoietic stem/progenitor cell gene therapy for Wiskott–Aldrich syndrome | Nature Medicine ScienceDirectHematopoietic Cell Transplantation for Wiskott-Aldrich syndrome: A PIDTC Report - ScienceDirect ScienceDirectOutcomes of hematopoietic stem cell gene therapy for Wiskott-Aldrich syndrome - ScienceDirect
Domain	Concerning finding	Interpretation and next step
Platelets	Suboptimal or declining platelet count after HCT	Assess donor myeloid chimerism; low myeloid chimerism is associated with lower platelet counts. ScienceDirectScienceDirectHematopoietic Cell Transplantation for Wiskott-Aldrich syndrome: A PIDTC Report - ScienceDirect
Immune function	Recurrent or severe infections	Assess immune reconstitution and investigate infection promptly; infection burden is a core WAS outcome domain. JAMA+2JAMAOutcomes of Gene Therapy for Severe Wiskott-Aldrich Syndrome NatureLong-term safety and efficacy of lentiviral hematopoietic stem/progenitor cell gene therapy for Wiskott–Aldrich syndrome \| Nature Medicine ScienceDirectOutcomes of hematopoietic stem cell gene therapy for Wiskott-Aldrich syndrome - ScienceDirect
Autoimmunity	New or recurrent autoimmune manifestations	Reassess disease activity even after gene therapy or HCT; prior autoimmunity may flare after gene therapy. ScienceDirectScienceDirectOutcomes of hematopoietic stem cell gene therapy for Wiskott-Aldrich syndrome - ScienceDirect
Malignancy	Lymphadenopathy, constitutional symptoms, or other concern for lymphoid disease	Initiate hematology-oncology evaluation because lymphoreticular malignancy is a recognized WAS complication. fda+1fdaFDA Approves First Gene Therapy Treatment for Wiskott-Aldrich Syndrome \| FDA cellEvidence for Long-term Efficacy and Safety of Gene Therapy for ...