Unrelated umbilical cord blood transplantation in children with immune deficiency: results of a multicenter study

Abstract

In the absence of a related donor, unrelated cord blood transplant (CBT) may be a potential option for patients with a primary immune deficiency (PID). Most published experience consists of single-center data using multiple preparative regimens and GVHD prophylaxis. We report the results of a multicenter prospective trial of unrelated CBT for PID. A total of 24 children with PID, with a median age of 1 year (range: 0.23-7.81 years) and a median weight of 10.5 kg (range: 4-24.4 kg) received unrelated CBT between 1999 and 2003. All patients received a fully ablative conditioning regimen with identical GVHD prophylaxis and supportive care. Most patients (79%) received a 1 or 2 HLA Ag-mismatched cord unit with a median nucleated cell infused of 9.3 Â 10 7 /kg (range: 1.0-31.2) and a median CD34 of 2.7 Â 10 5 /kg 2.9 (range: 0.6-84.5). The cumulative incidence of neutrophil engraftment by day 42 was 58% (95% CI: 38-79%) at a median of 19 days. Cumulative incidence estimates of grade III-IV acute GVHD at day 100 and chronic GVHD at 1 year were 29% (95% CI: 10-48%) and 24% (95% CI: 3-44%), respectively. The probability of survival at 180 days and 1 year was 66.7% (95% CI: 44.3-81.7%) and 62.5% (95% CI: 40.3-78.4%), respectively. Unrelated CBT should be considered in children with PID.

The Mannheim Cord Blood Bank: Experiences and Perspectives for the Future

“…More than 6,000 unrelated CB transplantations to treat malignant and non-malignant diseases have been performed since the first CB transplant in 1988. In accordance with recent publications our data confirm that main indications for CB transplantations are malignant diseases (acute myeloid leukemia (AML); chronic myelogenous leukemia (CML); non-Hodgkin's lymphoma (NHL);) and non-malignant hematologic and metabolic diseases as well as immune deficiencies [2,3,13,17,18]. Although our CB units were mainly used for children, CB is becoming increasingly relevant for the transplantation of adult patients too [5,[19][20][21].…”

“…It is a curative option for many hematologic malignancies and genetic diseases [1][2][3][4][5][6]. Although there are about 13 million registered stem cell donors worldwide (source: Bone Marrow Donors Worldwide (BMDW), September 2009), it is still not possible to find a suitable donor for approximately one third of patients in need of a transplant [7].…”

Umbilical cord blood banking: an update

“…Frangoul et al [2] reported their multicenter experience in the use of cord blood for non-oncology purposes in patients with primary immune deficiency (PID). They enrolled 364 adults and children in a study from 1999 to 2003 using an approved NIH sponsored protocol involving several sites and reported their results on 24 children with PID.…”

“…Cord blood is considered a treatment option in pediatric and adult patients with hematologic malignancies and disorders (leukemia, thalassemia, sickle cell disease, etc. ), bone marrow failures, inherited metabolic disorders, immunological defects and other genetic diseases [1][2][3][4][5][6][7][8][9][10][11][12]. Double umbilical cord blood grafts, that use cord blood units from two donors, mitigate cell dose limitations for larger children and adults with malignant disorders [12][13][14].…”

Transplantation in patients with SCID: mismatched related stem cells or unrelated cord blood?

“…It has been shown recently that UCBT in PID patients is a feasible alternative. [9][10][11][12] A critical end point after HSCT for PID is successful immune reconstitution and the latter's kinetics. In fact, after transplantation of a T cell-depleted graft, circulating T cells may not be detected for several months and normal counts are generally only achieved between 6 and 12 months after transplantation.…”