ABSTRACT
Early results of unrelated cord blood transplantation (UCBT) for severe aplastic anemia (SAA) were poor with a high rate of engraftment failure. This was attributed to the combination of lower graft cell dose and intact host immune system. We performed UCBT in nine children (median age 9 years) with refractory SAA using increasingly immunosuppressive preparative regimens. The time from diagnosis to UCBT was 3.4-20 months (median age 7.2 years), with all children having failed at least one course of immunosuppression. Donor/recipient HLA matching was six of six (n=1), five of six (n=2) and four of six (n=6). The median nucleated cell dose infused was 5.7 x 10(7) cells/kg (range 3.5-20 x 10(7) cells/kg). Six patients were engrafted after the first UCBT. Two of the three patients without hematopoietic reconstitution were engrafted after a second UCBT. All children receiving >or=120 mg/kg of CY in the preparative regimen were engrafted. The median time to myeloid engraftment was 25 (17-59 days) days. Acute GVHD developed in two, and chronic GVHD in five patients. Five patients developed EBV viremia post transplant (lymphoproliferative disorder in three patients). At a median follow-up of 34 months, seven patients are alive and transfusion-independent. UCBT is a feasible treatment strategy for children with refractory SAA lacking a well-matched adult donor.
Subject(s)
Anemia, Aplastic/therapy , Cord Blood Stem Cell Transplantation/methods , Adolescent , Child , Child, Preschool , Cord Blood Stem Cell Transplantation/adverse effects , Disease-Free Survival , Graft Rejection/immunology , Graft Survival/immunology , Graft vs Host Disease/immunology , Humans , Infant , Transplantation Chimera , Treatment Outcome , Young AdultABSTRACT
Delayed hematologic recovery is common after unrelated donor umbilical cord blood transplants (UCBT). Clinically it is important to quickly differentiate slow engraftment from graft failure (GF). We report the engraftment data on 110 pediatric UCBT recipients. By day 28, 71 patients achieved an ANC >0.5 x 10(9) per liter, and 6 others died early without recovery. Of the remaining 33 patients who were still neutropenic, 20 eventually attained donor myeloid recovery, 3 died of transplant-related complications or recurrent leukemia and 10 survived without donor-derived hematopoiesis. These patients received a second UCBT 33-95 days after the first transplant, after additional immunosuppression. One patient died early, the remaining nine patients were engrafted; eight demonstrated complete, and one mixed, donor chimerism (with subsequent graft loss). Acute GVHD developed in three, and chronic GVHD in six of the eight engrafted patients. Two patients developed EBV-lymphoproliferative disorder. Infections, especially viral, were common and protracted. Six of 10 patients are alive, 165-1375 (median 1147) days after second UCBT. Chimerism studies correlated with subsequent engraftment course. Any result showing <5% donor cells was associated with irreversible graft loss. In conclusion, early second UCBT after primary GF is a feasible treatment option. Chronic GVHD and viral reactivation are common post transplant.
Subject(s)
Cord Blood Stem Cell Transplantation/adverse effects , Graft Survival , Hematopoietic Stem Cell Transplantation/adverse effects , Salvage Therapy , Adolescent , Child , Child, Preschool , Cohort Studies , Female , Humans , Infant , Male , Retrospective Studies , Transplantation Chimera , Transplantation Conditioning/adverse effects , Transplantation, Homologous , Treatment FailureSubject(s)
Hematologic Neoplasms/genetics , Leukemia/genetics , Neoplasms, Multiple Primary/genetics , Age of Onset , Child, Preschool , Cytogenetic Analysis , Family Health , Hematologic Neoplasms/ethnology , Hematologic Neoplasms/therapy , Hematopoietic Stem Cell Transplantation , Hispanic or Latino/genetics , Humans , Leukemia/ethnology , Leukemia/therapy , Male , Neoplasms, Multiple Primary/ethnology , Neoplasms, Multiple Primary/therapy , PedigreeABSTRACT
PURPOSE: To compare gadolinium-enhanced inversion-recovery magnetic resonance (MR) imaging with renal cortical scintigraphy in the diagnosis of childhood pyelonephritis. MATERIALS AND METHODS: Thirty-seven patients with fever-producing urinary tract infection underwent gadolinium-enhanced inversion-recovery MR imaging and technetium-99m renal cortical scintigraphy. Each study was read in double-blind fashion by two radiologists. The kidney was divided into three zones, and each was graded as positive, equivocal, or negative for pyelonephritis. RESULTS: Seventy kidneys (210 zones) were imaged. Twenty-six kidneys (54 zones) had evidence of pyelonephritis at both MR imaging and scintigraphy. Twenty-four kidneys (100 zones) were negative on both studies. Twelve kidneys (42 zones) were positive at MR imaging but negative at scintigraphy, and four kidneys (seven zones) were negative at MR imaging but positive at scintigraphy. The results of MR imaging for pyelonephritis were not equivalent to the results of scintigraphy (P = .001 for renal zones). The proportion of positive agreement between readers for the presence of pyelonephritis was 0.85 and 0.57 for MR imaging and scintigraphy, respectively. The proportion of negative agreement was 0.88 and 0.80 for MR imaging and scintigraphy, respectively. CONCLUSION: Gadolinium-enhanced inversion-recovery MR imaging enabled detection of more pyelonephritic lesions than did renal cortical scintigraphy and had superior interobserver agreement.