scholarly journals Posttransplant Autoimmune Hemolytic Anemia and Other Autoimmune Cytopenias are Increased in Very Young Infants Undergoing Unrelated Donor Umbilical Cord Blood Transplantation

2008 ◽  
Vol 14 (10) ◽  
pp. 1108-1117 ◽  
Author(s):  
Kristin M. Page ◽  
Adam M. Mendizabal ◽  
Vinod K. Prasad ◽  
Paul L. Martin ◽  
Suhag Parikh ◽  
...  
Keyword(s):  
Cord Blood ◽  
Hemolytic Anemia ◽  
Umbilical Cord Blood ◽  
Autoimmune Hemolytic Anemia ◽  
Cord Blood Transplantation ◽  
Unrelated Donor ◽  
Umbilical Cord Blood Transplantation ◽  
Blood Transplantation ◽  
Young Infants ◽  
Autoimmune Cytopenias

Unique Reversible but Serious Adverse Events in Very Young Infants Undergoing Unrelated Donor Umbilical Cord Blood Transplantation for Non-Malignant Conditions: Increased Autoimmune Cytopenias and Hypertrophic Cardiomyopathy.

Blood ◽  
2005 ◽  
Vol 106 (11) ◽  
pp. 5456-5456
Author(s):  
Sonali Lakshminarayanan ◽  
Paul Szabolcs ◽  
Suhag Parikh ◽  
Susan J. Wood ◽  
June Allison ◽  
...  
Keyword(s):  
Cord Blood ◽  
Umbilical Cord ◽  
Umbilical Cord Blood ◽  
Cord Blood Transplantation ◽  
Unrelated Donor ◽  
Umbilical Cord Blood Transplantation ◽  
Mps Ii ◽  
Blood Transplantation ◽  
Young Infants ◽  
Autoimmune Cytopenias

Abstract Between September 1998 and June 2004, 15 young infants, <3 months of age, 60% males, 14 Caucasian, 1 African American, underwent unrelated donor umbilical cord blood transplantation for treatment of non-malignant conditions [early infantile Krabbe disease (n = 9), MLD (n = 1), MPS I (n = 1), MPS II (n = 2), Tay Sachs Disease (N = 1) or Beta Thalassemia major (n = 1)]. All infants were prepared for transplant with oral busulfan 20mg/m2/dose (targeting a steady state of 600–900ng/ml), cyclophosphamide 50mg/kg/dose daily x 4, and equine ATG 30mg/kg/dose daily x 3. Cyclosporine and solumedrol were used for prophylaxis against GvHD. All infants engrafted with donor cells, although 1 patient with MPS II never achieved >50% donor chimerism. Two patients died 3 and 5 years post transplant from respiratory failure (1 patient with MPS II and incomplete chimerism) and progressive Tay Sachs Disease, respectively. Grafts were mismatched at 1 (n = 8) or 2 (n = 7) HLA loci and 8/15 donor/recipient pairs were ABO mismatched. Nine patients developed mild to moderate acute GvHD. Within 6–10 months of transplant, six of the 15 patients developed autoimmune cytopenias (autoimmune hemolytic anemia +/− ITP), diagnosed clinically by Coombs positivity and antiplatelet antibodies respectively. They required therapy with steroids, imuran, dacluzimab, rituximab, and in one patient, splenectomy. There was no correlation with ABO or HLA matching or GvHD and the incidence of autoimmune disease. All patients recovered within 3–24 months. Eight of the 15 newborns developed concentric cardiomyopathy while on solumedrol and either cyclosporine or FK506 for GvHD treatment and/or prophylaxis. One patient required intubation and prolonged mechanical ventilation. The others remained relatively asymptomatic. Over time and with weaning of steroid therapy, their left ventricular hypertrophy resolved and heart function returned to normal. Both autoimmune cytopenias and hypertrophic cardiomyopathy occur in higher frequency in very young infants undergoing unrelated donor umbilical cord blood transplantation. The etiology of the autoimmune disease is not currently known while the cardiac disease is a result of therapies used to treat and prophylax against GvHD.

Post Transplant Autoimmune Hemolytic Anemia and Other Cytopenias Are Increased in Very Young Babies after Unrelated Donor Umbilical Cord Blood Transplantation.

Blood ◽  
2007 ◽  
Vol 110 (11) ◽  
pp. 1054-1054 ◽  
Author(s):  
Kristin M. Page ◽  
Adam Mendizabal ◽  
Paul Szabolcs ◽  
Susan Wood ◽  
Vinod Prasad ◽  
...  
Keyword(s):  
Cord Blood ◽  
Umbilical Cord Blood ◽  
Autoimmune Hemolytic Anemia ◽  
Cumulative Incidence ◽  
Acute Gvhd ◽  
Cord Blood Transplantation ◽  
Unrelated Donor ◽  
Umbilical Cord Blood Transplantation ◽  
Post Transplant ◽  
Blood Transplantation

Abstract Background: Unrelated donor umbilical cord blood transplantation (UCBT) is beneficial in the treatment of very young babies with early infantile, lysosomal storage diseases and hemoglobinopathies, diagnosed in utero or in the neonatal period. Methods: Over the past 9 years, we have treated 18 neonates (≤3 months of age at transplant) with Krabbe disease (n=11) metachromatic leukodystrophy (n=1), Tay Sachs disease (n=1), Hurler syndrome (n=2), Hunter syndrome (n=2), and Beta-Thalassemia Major (n=1) with UCBT after myeloablative conditioning therapy with Busulfan, Cyclophosphamide and ATG. All babies received cyclosporine + methylprednisolone (n=16) or cyclosporine + cellcept (n=2) for 9 months post transplant for prophylaxis against GvHD. Engraftment, acute and chronic GvHD, survival, treatment related mortality, and deaths were scored. Results: Eighteen babies were transplanted and 17 were evaluable for engraftment, GvHD, and survival. One baby died before engraftment of pulmonary hypertension day 15 post transplant. The cumulative incidence of overall survival was 94.4% (95% CI 83.9%–100.0%), 88.9 (95% CI 74.4%–100.0%) and 77.8% (95% CI 53.8%–100.0%) at 1, 2, and 5 years, respectively. In these infants receiving very high cell doses from their UCB graft (median total nucleated cell dose of 18.71x107/kg), neutrophil engraftment with an ANC >500/uL occurred at a median of 19 days with a cumulative incidence of engraftment of 94.1% (95%CI 77.9%–100.0%) by 42 days. Platelet engraftment (platelet count of 50K/uL untransfused) occurred in a median of 56 days with a cumulative incidence of engraftment of 94.1% (95%CI 77.8%–100.0%) at 6 months post transplant. Grade I-II acute GvHD occurred in 15/18 infants while one infant developed grade III acute GvHD of skin and gut. The cumulative incidence of grade II-III acute GvHD by day +100 was 29.4% (95%CI 6.9%–51.9%). Nine of seventeen evaluable patients developed cGvHD manifesting as autoimmune cytopenias with a cumulative incidence of 41.2% (95%CI 16.9%–65.5%) and 52.9% (95%CI 28.0–77.8%) at 1 and 2 years, respectively. In six patients, cGvHD presented as autoimmune cytopenia de novo. No graft factors were identified as being significant to development of cGvHD. All patients responded to treatment with methylprednisolone, azithroprine +/− rituximab. One patient required splenectomy. In contrast, the incidence of cGvHD in a group of otherwise similar older patients was 14.7%. Conclusion: We report an unexpected and high incidence of cGvHD manifesting primarily as autoimmune hemolytic anemia with other cytopenias, post UCBT in a population of very young babies. We hypothesize that post transplant immunosuppression interferes with the normal development of the immune system in the first year of life creating immune dysregulation and graft directed cell destruction. After lytic agents to stabilize disease, removal of chronic immunosuppressive therapy appears to facilitate recovery. Alternative strategies to prevent GvHD should be considered for this unique patient population.

Refractory Autoimmune Hemolytic Anemia Following Unrelated Cord Blood Transplantation for Acute Lymphoblastic Leukaemia: A Case Report and Review of the Literature.

Blood ◽  
2007 ◽  
Vol 110 (11) ◽  
pp. 5031-5031
Author(s):  
Huilan Liu ◽  
Liangquan Geng ◽  
Juan Tong ◽  
Zuyi Wang ◽  
Zimin Sun ◽  
...  
Keyword(s):  
Case Report ◽  
Cord Blood ◽  
Hemolytic Anemia ◽  
Umbilical Cord ◽  
Umbilical Cord Blood ◽  
Autoimmune Hemolytic Anemia ◽  
Cord Blood Transplantation ◽  
Unrelated Donor ◽  
Hematopoietic Stem ◽  
Blood Transplantation

Abstract Autoimmune haemolytic anaemia (AIHA) after hematopoietic stem cell transplantation(HSCT) is a rare severe complication particularly in the paediatric population who underwent umbilical cord blood transplantation(UCBT).The pathogenesis of the post-HSCT AIHA is poorly understood, which frequently difficult to treat and overall prognosis is often poor. We present a case of a 13-year-old boy who developed AIHA 6 months after UCBT with a minor ABO blood group incompatiblity, 5/6 HLA matching umbilical cord blood unit from an unrelated donor was used for high risk acute lymphocyte leukemia in third remmision. Following conditioning with busulfan, cyclophosphamide, and antithymocyte globulin (ATG), the patient underwent transplantation and attained sustained hemopoietic reconstitution without severe graft-versus-host disease. At 6 months following UCBT,he developed severe acute autoimmune hemolytic anemia associated with a “warm” IgG antibody. He was being treated with packed RBC transfusions, cyclosporine, steroids and immunoglobulins, His AIHA was partly resolved. Hemolysis recurred after steroid tapering and partially responded to rituximab (375 mg/m(2) once weekly ×4 and plasma exchange.Despite a range of complex treatment regimen, the unstable hemolytic anemia was present and repeated severe infectious episodes occurred.We reduced suppression,strengthened antibiotic therapy,Pulmonary infection and hemolysis could not be controlled, the patient died of respiratory failure and circulatory collapse finally. This case report highlights the difficulty in managing posttransplant AIHA,Further studies about the pathogenesis of AIHA associated in HSCT recipients are needed.

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