Primary Immunodeficiencies
13 cases on X-linked SCID gene therapy versus haploidentical transplant, treating active infection in a SCID infant awaiting transplant, immunosuppressing refractory ITP in CVID, GLILD therapy with no agreed standard, X-linked agammaglobulinemia trough targets, specific antibody deficiency prophylaxis thresholds, chronic granulomatous disease transplant timing, Wiskott-Aldrich transplant urgency, off-label dupilumab in hyper-IgE syndrome, DOCK8 deficiency transplant referral, rituximab-induced secondary hypogammaglobulinemia, Fontan-associated protein-losing enteropathy dosing, and IVIG-to-SCIG conversion — choose a case below to open its full multi-voice debate.
A newborn-screened X-linked SCID case weighing autologous lentiviral gene therapy against haploidentical transplant, gated on genotype, donor availability, and real-world enrollment timelines.
A SCID infant awaiting hematopoietic transplant with active vaccine-strain rotavirus infection, weighing immunoglobulin bridging and antiviral therapy against the timing of definitive conditioning.
A CVID patient with refractory immune thrombocytopenia weighing rituximab against continued steroids, and whether immunoglobulin replacement can be optimized enough to make immunosuppression safer.
A CVID patient newly diagnosed with granulomatous-lymphocytic interstitial lung disease, weighing rituximab and azathioprine against monitoring given the absence of an established treatment standard.
An adolescent with X-linked agammaglobulinemia and breakthrough sinopulmonary infections despite standard immunoglobulin dosing, weighing a higher trough target against bronchiectasis-directed airway therapy.
A child with specific antibody deficiency and recurrent sinopulmonary infections despite normal immunoglobulin levels, weighing continued antibiotic prophylaxis against a defined trial of immunoglobulin replacement.
A newly diagnosed chronic granulomatous disease case weighing antimicrobial prophylaxis with interferon-gamma against early hematopoietic stem cell transplantation, given a matched sibling donor and modern reduced-toxicity conditioning outcomes.
A Wiskott-Aldrich syndrome case weighing hematopoietic transplant timing against active bleeding risk, gated on the WAS clinical severity score and platelet stabilization before conditioning.
A STAT3 hyper-IgE syndrome case weighing off-label dupilumab for debilitating eczema against theoretical concern for further blunting an already-impaired Th17 and infection-control immune axis.
A DOCK8 deficiency case weighing early referral for hematopoietic stem cell transplantation against continued supportive management while the child remains clinically stable.
Two patients with rituximab-induced secondary hypogammaglobulinemia — one with relapsing EGPA still needing the biologic, one with IgG4-related disease in remission — weighing immunoglobulin replacement against holding future biologic dosing.
A Fontan-associated protein-losing enteropathy case with secondary hypogammaglobulinemia, weighing immunoglobulin dosing intensity and interval against addressing the underlying lymphatic physiology directly.
A CVID patient stable on hospital-based IVIG weighing a switch to home subcutaneous immunoglobulin, comparing trough stability, quality of life, and monitoring tradeoffs between the two modalities.