ABSTRACT
MHC class II deficiency is a rare and fatal form of primary combined immunodeficiency caused by a lack of T-cell-dependent humoral and cellular immune response to foreign antigens, which can only be cured by allogenic stem cell transplantation. In the literature search, we identified 68 cases of HSCT in MHC class II deficiency in the last 14 yr. Pre- and post-transplant MHC class II deficiency is complicated by overwhelming viral infections, a high incidence of GvHD, and graft failure with a poor overall survival rate below 50%. We report an eight-month-old boy presenting with severe respiratory infections and chronic diarrhea, whose sister died at the age of four yr from septicemia. MHC II deficiency was caused by an RFXANK-mutation and treated successfully by 4/6 mismatched unrelated CBT after a myeloablative conditioning regimen based on anti-thymocyte globulin, busulfane, fludarabine, and cyclophosphamide. At present, our patient is well with full immune reconstitution 3(4/12) yr after CBT. CB may represent an alternative source of stem cells for children with MHC class II deficiency without a suitable donor.
Subject(s)
Fetal Blood/transplantation , Hematopoietic Stem Cell Transplantation/methods , Histocompatibility Antigens Class II/immunology , Immunologic Deficiency Syndromes/surgery , Follow-Up Studies , Graft Survival , Histocompatibility Antigens Class II/genetics , Histocompatibility Testing , Humans , Immunologic Deficiency Syndromes/diagnosis , Infant , Male , Risk Assessment , Severity of Illness Index , Transplantation Conditioning/methods , Transplantation, Homologous , Treatment OutcomeABSTRACT
On the basis of experimental set-ups in vitro and in vivo and by making use of specific autoradiographical techniques, the following data on the proliferation of astrocytes from newborn rats in vitro and unpretreated rats and mice in vivo could be obtained: (i) The commonly employed immunohistochemical staining techniques in vitro are not applicable in tissue sections. (ii) In vivo, astrocytes show increasing durations of cell cycle (tc) as well as S phase (ts) prenatally until about birth. A similar trend can be observed in vitro. However, the absolute values for ts and tc can be substantially modified depending on the culture conditions. (iii) As regards the mode of proliferation, astrocytes in vitro grow exponentially and without transition of quiescent cells from the non-growth fraction into the growth fraction (GF). In contrast, astrocytes in vivo exhibit steady-state growth and continuous recruitment of proliferating cells from the non-GF. These differences show that there is a need for further in vivo-experiments when studying new strategies in the treatment of gliomas.
