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Successful allogeneic SCT in infant with FHL, dilemmas in using „unaffected“ sibling

Successful allogeneic SCT in infant with FHL, dilemmas in using „unaffected“ sibling. P.Sedláček, R. Špíšek, et al 2nd Medical School, Charles University, Prague, CZ. ESID – May 10.-11. 2004. Past Medical History (family). mother HBsAg +, no transf.history father – OK

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Successful allogeneic SCT in infant with FHL, dilemmas in using „unaffected“ sibling

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  1. Successful allogeneic SCT in infant with FHL, dilemmas in using „unaffected“ sibling P.Sedláček, R. Špíšek, et al 2nd Medical School, Charles University, Prague, CZ ESID – May 10.-11. 2004

  2. Past Medical History (family) • mother HBsAg +, no transf.history • father – OK 1st girl – died 32nd gest.week (umb. cord problems) 2nd boy (IVF) – Jakub – alive/well 3rd girl (IVF) - Jana * July 8th 2003

  3. History of Present Illness I – early postnatal pancytopenia (WBC : 4,8, RBC: 3,1, ret.: 0,1%, ANC: <100, Plt 11) allo HLA Ab in mother exchange transfusion (D+9), PRBC, Plt, Fbg HSmegaly, hepatopathy, hypertriglycemia, hyperbilirubinemia hyperferritinemia ( >18 000) DIC, infections, fever and elev. CRP, HBsAg and HBeAg negative (Engerix, Hepatect) Admission to our department at the age of 28 days (Aug.2003)

  4. History of Present Illness II • Day of admission Aug. 5th 2003 • liver +4cm, spleen +3-4cm bcm, pancytopenia • Therapy according to HLH 94 • (VP16, Dexa, CsA) • discontinued for acute sepsis • Transfusion history pre SCT • PRBC 13 x (or more) • Plt more than 13

  5. Pre-transplant condition • HSmegaly, ferritin 3153 • antiHBc and antiHBe pos., HBsAg neg. • Failure to thrive (bw 3,45 kg; 2,8 kg at birth) • normal LFT; Plt 100, WBC and RBC normal • brother fully HLA matched, 19kg bw, 3yo • ABO incompat. : A+ vs. B- (low titers)

  6. Control Control Brother´s NK cells Patient´s NK cells Diagnostics-1 FACS analysis of perforin expression in NK cells Exclusion of FHL type 2- perforin deficiency 87% of NK cells are perforin + 87% of NK cells are perforin +

  7. control brother % cytotoxicity • defective cytotoxic activity of patient´s lymphoblasts • normal findings in her brother donor of SC X patient Diagnostics-2 • Cytotoxic activity of T-lymphoblasts generated from T-cells • Target cells: L1210 cells deficient in FAS expression

  8. Transplantation • 11.9.2003 (* 8.7.2003) • Bu(19mgkg) + Cy(200) + rATG(25mg/kg) • CsA + MP (MTX not used due to early and severe VOD) • BM : NC : 8,9x 108/kg; CD34: 22x106/kg • VOD : D+1 (D-3 through D+15 defibrotide) • Bilirubinemia, thrombocytopenia, ascites, ARDS • Engraftment :ANC (500) D+19; Plt (20/50) D+22/+26 • Chimer. : stable, 95% donor • Discharched to outpatient clinic D+34 • no aGVHD, no cGVHD so far (~D+240) • continues on CsA, steroids D/C ~D+50 • growth catch-up

  9. Acknowledgement • Department of Pediatric Hematology and Oncology • HSCT unit; Division of Hematology • Institute of Immunology • diagnostics of primary HLH • Institute of Hematology and Blood Transfusion • HLA typing

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