Pediatric Hematology/Oncology and ImmunopathologyPediatric Hematology/Oncology and ImmunopathologyВопросы гематологии/онкологии и иммунопатологии в педиатрии1726-17082414-9314Fund Doctors, Innovations, Science for Children6810.24287/1726-1708-2017-16-3-55-58СтатьиThe use of plerixafor and G-CSF during conditioning for hematopoietic stem cell transplantation in a patient with Wiscott–Aldrich syndromeПрименение плериксафора и гранулоцитарного колониестимулирующего фактора в кондиционировании перед трансплантацией гемопоэтических стволовых клеток у пациентов с синдромом Вискотта-Олдричаhttps://orcid.org/0000-0003-2689-0569BalashovD. N.БалашовД. Н.
Russian Federation

Head of Hematopoietic Stem Cell Transplantation Department #2

Russia 117997, Moscow, Samory Mashela st., 1

д-р мед. наук, заведующий отделением трансплантации  гемопоэтических стволовых клеток № 2

117997, Москва, ГСП-7, ул. Саморы Машела, 1 Тел.: 8 (495) 287-6570, доб. 5527

bala8@yandex.ruhttps://orcid.org/0000-0002-3800-8927GutovskayaE. I.ГутовскаяЕ. И.
Russian Federation
https://orcid.org/0000-0002-1754-1220KozlovskayaS. N.КозловскаяС. Н.
Russian Federation
https://orcid.org/0000-0002-7696-1153RadyginaS. A.РадыгинаС. А.
Russian Federation
https://orcid.org/0000-0002-2354-2588LaberkoA. L.ЛаберкоА. Л.
Russian Federation
https://orcid.org/0000-0002-0016-6698MaschanA. A.МасчанА. А.
Russian Federation
Dmitriy Rogachev National Medical Research Center of Pediatric Hematology, Oncology, Immunology Ministry of Healthcare of Russian FederationФГБУ «Национальный медицинский исследовательский центр детской гематологии, онкологии и иммунологии им. Дмитрия Рогачева» Минздрава России, Москва10082017163555810082018Copyright ©; 2017, «D. Rogachev NMRCPHOI»Copyright ©; 2017, ФГБУ «НМИЦ ДГОИ им. Дмитрия Рогачева» Минздрава России2017«D. Rogachev NMRCPHOI»ФГБУ «НМИЦ ДГОИ им. Дмитрия Рогачева» Минздрава Россииhttps://creativecommons.org/licenses/by/4.0https://hemoncim.com/jour/article/view/68

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.

Тяжелые дисфункции и отторжение трансплантата после трансплантации гемопэтических стволовых клеток (ТГСК) - одна из серьезных проблем у пациентов с синдромом Вискотта-Олдрича. В данной публикации представлено описание клинического случая отторжения трансплантата и повторной ТГСК с применением альтернативного подхода к подготовительной терапии. Мы включили гранулоцитарный колониестимулирующий фактор и плериксафор в режим кондиционирования для мобилизации гемопоэтических стволовых клеток в сосудистое русло и образования свободного пространства в строме костного мозга для более эффективного приживления донорских клеток.

Wiskott–Aldrich syndromehematopoietic stem cell transplantationplerixaforсиндром Вискотта-Олдричатрансплантация гемопоэтических стволовых клетокплериксафор1. Wiskott A. Familiarer, angeborener Morbus Werlhofii? Monatsschr Kinderheilkd 1937; 68: 212-6.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.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.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.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.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.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.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.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.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.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.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.