Вопросы гематологии/онкологии и иммунопатологии в педиатрии. 2017; 16: 55-58
Применение плериксафора и гранулоцитарного колониестимулирующего фактора в кондиционировании перед трансплантацией гемопоэтических стволовых клеток у пациентов с синдромом Вискотта-Олдрича
https://doi.org/10.24287/1726-1708-2017-16-3-55-58Аннотация
Список литературы
1. Wiskott A. Familiarer, angeborener Morbus Werlhofii? Monatsschr Kinderheilkd 1937; 68: 212-6.
2. Villa A., Notarangelo L., Kraus N.D., et al. X-linked thrombocytopenia and Wiskott-Aldrich syndrome are allelic diseases with mutation in WASp gene. Nat Genet 1995; 9: 414-7.
3. Devriendt K., Kim A.S., Mathius G., et al. Constitutively activating mutation in WASp causes X-linked severe congenital neutropenia. Nat Genet 2001; 27: 313-7.
4. Pai S.Y., Notarangelo L.D. Hematopoietic Cell Transplantation for Wiskott-Aldrich Syndrome: Advances in Biology and Future Directions for Treatment. Immunol Allergy Clin North Am 2010; 30 (2): 179-94.
5. Dupuis-Girod S., Medioni J., Haddad E., et al. Autoimmunityin Wiskott-Aldrich syndrome: risk factors, clinical features, and outcome in a single center cohort of 55 patients. Pediatr 2003; 111 (5): e622-e627.
6. Imai K., Morio T., Zhu Y., et al. Clinical course of patients with WASP gene mutations. Blood 2004; 103 (2): 456-64.
7. Schurman S.H., Candotti F. Autoimmunity in Wiskott-Aldrich syndrome. Curr Opin Rheumatol 2003; 15 (4): 446-53.
8. Ochs H.D., Filipovich A.H., Veys P., Cowan M.J., Kapoor N. Wiskott-Aldrich syndrome: diagnosis, clinical and laboratory manifestations, and treatment. Biol Blood Marrow Transplant 2009; 15 (1): 84-90.
9. Konopleva M., Benton C.B., Thall P.F., et al. Leukemia cell mobilization with G-CSF plus plerixafor during busulfan-fludarabine conditioning for allogeneic stem cell transplantation. Bone Marrow Transplant 2015; 50: 939-46.
10. Dvorak C.C., Horn B.N., Puck J.M., et al. A trial of plerixafor adjunctive therapy in allogeneic hematopoietic cell transplantation with minimal conditioning for severe combined immunodeficiency. Pediatr Transplant 2014; 18: 602-8.
11. Maschan A.A., Balashov D.N., Kurnikova E.E., et al. Efficacy of plerixafor in children with malignant tumors failing to mobilize a sufficient number of hematopoietic progenitors with G-CSF. Bone Marrow Transplant 2015; 50 (8): 1089-91.
Pediatric Hematology/Oncology and Immunopathology. 2017; 16: 55-58
The use of plerixafor and G-CSF during conditioning for hematopoietic stem cell transplantation in a patient with Wiscott–Aldrich syndrome
https://doi.org/10.24287/1726-1708-2017-16-3-55-58Abstract
High incidence of impaired graft function remains a significant issue in patients with Wiskott–Aldrich Syndrome after hematopoietic stem cell transplantation (HSCT). We report a clinical case of graft rejection and second HSCT with novel alternative approach to the conditioning regimen. We used of G-CSF and plerixafor as additional agents in conditioning regimen for stem cell release and opening of bone marrow niches to improve stem cell engraftment.
References
1. Wiskott A. Familiarer, angeborener Morbus Werlhofii? Monatsschr Kinderheilkd 1937; 68: 212-6.
2. Villa A., Notarangelo L., Kraus N.D., et al. X-linked thrombocytopenia and Wiskott-Aldrich syndrome are allelic diseases with mutation in WASp gene. Nat Genet 1995; 9: 414-7.
3. Devriendt K., Kim A.S., Mathius G., et al. Constitutively activating mutation in WASp causes X-linked severe congenital neutropenia. Nat Genet 2001; 27: 313-7.
4. Pai S.Y., Notarangelo L.D. Hematopoietic Cell Transplantation for Wiskott-Aldrich Syndrome: Advances in Biology and Future Directions for Treatment. Immunol Allergy Clin North Am 2010; 30 (2): 179-94.
5. Dupuis-Girod S., Medioni J., Haddad E., et al. Autoimmunityin Wiskott-Aldrich syndrome: risk factors, clinical features, and outcome in a single center cohort of 55 patients. Pediatr 2003; 111 (5): e622-e627.
6. Imai K., Morio T., Zhu Y., et al. Clinical course of patients with WASP gene mutations. Blood 2004; 103 (2): 456-64.
7. Schurman S.H., Candotti F. Autoimmunity in Wiskott-Aldrich syndrome. Curr Opin Rheumatol 2003; 15 (4): 446-53.
8. Ochs H.D., Filipovich A.H., Veys P., Cowan M.J., Kapoor N. Wiskott-Aldrich syndrome: diagnosis, clinical and laboratory manifestations, and treatment. Biol Blood Marrow Transplant 2009; 15 (1): 84-90.
9. Konopleva M., Benton C.B., Thall P.F., et al. Leukemia cell mobilization with G-CSF plus plerixafor during busulfan-fludarabine conditioning for allogeneic stem cell transplantation. Bone Marrow Transplant 2015; 50: 939-46.
10. Dvorak C.C., Horn B.N., Puck J.M., et al. A trial of plerixafor adjunctive therapy in allogeneic hematopoietic cell transplantation with minimal conditioning for severe combined immunodeficiency. Pediatr Transplant 2014; 18: 602-8.
11. Maschan A.A., Balashov D.N., Kurnikova E.E., et al. Efficacy of plerixafor in children with malignant tumors failing to mobilize a sufficient number of hematopoietic progenitors with G-CSF. Bone Marrow Transplant 2015; 50 (8): 1089-91.
