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Respiratory Failure in a 10 month old Pursuing a Diagnosis

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Respiratory failure in a 10-month-old: Pursuing a diagnosis Todd D Green Children’s Hospital of Pittsburgh November 4, 2007 Case Presentation  10 mo male transferred from outside hospital   Admission 8 months for bilat pneumonia, pneumothorax WBC 4.4 k/ul, ALC 1628/mm3, ANC 2288/mm3, AEC 308/mm3  Resp failure, intubation, ECMO      BAL unremarkable (including Pneumocystis, fungal) ET sputum: Klebsiella, Stenotrophomonas, Serratia Urine negative for CMV, adenovirus EBV PCR negative Negative molecular testing for CF Skin biopsy “contact dermatitis”, resolved with steroids  Macular rash with eosinophilia (2500/mm3)  Case Presentation  PMH Chronic clear rhinorrhea since 1-2 months  Recurrent otitis media beginning 4 months   PE tubes placed 7 months, continued infections Rashes with azithromycin and amoxicillin  No chronic thrush, diarrhea, failure to thrive   FH 4 yo brother with JRA, mat first cousin with CF  No consanguinity, PID, early childhood deaths  Case Presentation  Medications on admission Bactrim, Zosyn, Vancomycin  Prednisolone  Methadone, diuretics, sedatives   Exam: Ht 5th %ile, Wt 5th %ile, HC 25th %ile HEENT: intubated (tonsils later visualized)  Nodes: 2 mm node rt axilla, 3 mm node lt groin  Liver 4-5 cm below rcm  No murmur, rash    Differential? Combined immunodeficiency SCID Omenn’s Syndrome MHC I or II Antigen Deficiency p56 Lck deficiency DiGeorge Syndrome Hyper-IgM Syndrome CGD CVID XLP XLA Others…  10 month old male with respiratory failure History chronic rhinorrhea, OM History eosinophilia and rash       Tonsils and lymph nodes present Lymphopenic     Further assessment:    IgG 38 mg/dL, IgA 9 mg/dL, IgM 9 mg/dL, IgE ≤2 U/mL Abs lymph count 2046/mm3 (3400-9000)  CD3+ 1550/mm3 (19005900)  CD4+ 500/mm3 (1400-4300)  33% 4/45RA/62L (58-91)  CD8+ 1053/mm3 (500-1700)  69% 8/45RA/62L (47-87)  CD19+ 308/mm3 (610-2600) Lymphocyte prolif normal to mitogens, absent antigens Clinical course:      Gradual recovery No rash 300-2700/mm3 eosinophils during 4 wk admission Bone marrow biopsy nl trilineage hematopoesis, no malignancy Home on IVIG, presumptive CVID Normals from Shearer et al., J Allergy Clin Immunol 2003 Follow-Up     Continued recurrent OM, mult sets PE tubes Recurrent rhinorrhea, diarrhea, slow wt gain Resolution eosinophilia, no further rash Developed PCP at 15 months  LE thromboses during hospitalization, diagnosed with MTHFR gene mutation (1p36.3)   Generally well since Recurrent nephrolithiasis with ammonium urate stones (unusual in US) at 31 months Follow-up: Immunologic Parameters 19 months ALC CD3+ CD4+ CD8+ CD4:8 CD16/56+ CD19+ CD45RA/62L 4320 (3600-8900) 2458 (2100-6200) 760 (1300-3400) 1659 (620-2000) 0.46 151 (180-920) 1581 (720-2600) 53% of T-cells 31 months 2695 (2300-5400) 1730 (1400-3700 507 (700-2200) 1145 (490-1300) 0.44 137 (130-720) 776 (390-1400) 70% of T-cells Normals from Shearer et al., J Allergy Clin Immunol 2003 Follow-up: Immunologic Studies Mitogen Prolif Antigen Prolif IgA IgM IgE 19 months Normal Absent 0 mg/dL 0 mg/dL 4 IU/mL 31 months Normal Absent 0 mg/dL 0 mg/dL 5 IU/mL Bacteriophage Φx-174 immunization response abnormal: No detectable antibody after primary immunization, but with phage clearance, suggesting presence of neutralizing antibody Very low antibody titer after second (no IgG isotype) Further Immunologic Workup        ADA, PNP activity Common gamma chain sequencing (pt, mom) SH2D1A sequencing BTK sequencing CD40Ligand sequencing CH50 Platelet size, number  All normal Summary      Now 3 3/12 yo, h/o PCP Agammaglobulinemia with normal % B-cells Abnormal response to bacteriophage immunization Low CD4 %, o/w normal lymphocyte populations, including CD45RA/62L T-cell fxn with nl mitogen prolif, absent antigen  Where to next?
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