Nonmyeloablative hematopoietic stem cell transplantation corrects the disease phenotype in the canine model of leukocyte adhesion deficiency

Reduced-intensity conditioning hematopoietic SCT for pediatric patients with LAD-1: clinical efficacy and importance of chimerism

“…On the other hand, as LAD-I patients have hyperactive myeloid macrophage lineage, RIC regimen results in a high incidence of mixed chimerism and rejection in such affected patients. [8][9][10][11] In 2007, the first pediatric transplant unit was established in our center. As all of the patients referred to our center were found to be infected at the time of admission or transplantation, RIC regimen was used as the best treatment option for affected patients.…”

“…[6][7][8][9] Recent data have shown that reduced-intensity conditioning (RIC) regimen, may lead to establishment of mixed chimerism in humans and canines following HSCT. [8][9][10][11] Despite this known fact, treatment based on RIC regimen because of its low transplant-related morbidity and mortality, has been increasingly utilized in recent years. [11][12][13][14][15][16][17] This study provides evaluation of RIC regimen, utilized in 10 LAD-1 patients who underwent HSCT at Hematology-Oncology and Stem Cell Transplantation research center (HORCSCT).…”

“…[2][3][4][5][6] Hematopoietic SCT (HSCT) is the only known curative approach to LAD-I treatment. [6][7][8][9][10][11] Reduced intensity and myeloablative conditioning regimens are currently being used in the treatment of patients affected by LAD-I. Although full donor chimerism can be achieved with the use of myeloablative conditioning regimen, this regimen can lead to a higher risk of infections in patients with LAD-I who suffer from comorbid complications.…”

Matched unrelated bone marrow transplantation with reduced-intensity conditioning for leukocyte adhesion deficiency

“…Pre-clinical data suggest that donor chimerism of only 5% may be sufficient to protect patients with LAD from recurrent pyogenic infections post transplant. [5][6][7][8] As such, strategies that maximize the probability of engraftment and donor tolerance while minimizing toxicity may be particularly efficacious in definitive LAD treatment.…”

Leukocyte integrin activation mediates transient neutropenia after G-CSF administration

“…14 Lack of CD18 results in the inability of neutrophils to migrate to sites of infection, leading to severe life-threatening bacterial infections. Canine LAD-1 (CLAD) in Irish setter dogs has been corrected by both allogeneic bone marrow transplantation 15,17 and by gene transfer. 18 Low numbers of CD18 ϩ neutrophils have been sufficient to correct the phenotype.…”