Unrelated umbilical cord blood transplantation in infancy for mucopolysaccharidosis type IIB (Hunter syndrome) complicated by autoimmune hemolytic anemia

Mullen, CA; Thompson, J. A.; Richard, LA; Chan, KW

doi:10.1038/sj.bmt.1702397

Cited by 58 publications

(30 citation statements)

References 18 publications

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“…32 The current treatment of MPS II has focused on managing symptoms, since no therapy is available. Therapies targeted to provide sufficient enzyme activity to slow or stop the progression of the disease, [33][34][35] such as hematopoietic cell transplantation, have been proposed, [33][34][35] but no systematic studies have been performed and the limited reports have been disappointing. 36 The results of the present study suggest that idursulfase may be the first treatment that can benefit patients with MPS II by addressing the underlying enzymatic defect.…”

Section: Discussionmentioning

confidence: 99%

A phase II/III clinical study of enzyme replacement therapy with idursulfase in mucopolysaccharidosis II (Hunter syndrome)

Muenzer

Wraith

Beck³

et al. 2006

Genetics in Medicine

512

509

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Purpose: To evaluate the safety and efficacy of recombinant human iduronate-2-sulfatase (idursulfase) in the treatment of mucopolysaccharidosis II. Methods: Ninety-six mucopolysaccharidosis II patients between 5 and 31 years of age were enrolled in a double-blind, placebo-controlled trial. Patients were randomized to placebo infusions, weekly idursulfase (0.5 mg/kg) infusions or every-other-week infusions of idursulfase (0.5 mg/kg).Efficacy was evaluated using a composite endpoint consisting of distance walked in 6 minutes and the percentage of predicted forced vital capacity based on the sum of the ranks of change from baseline. Results: Patients in the weekly and every-other-week idursulfase groups exhibited significant improvement in the composite endpoint compared to placebo (P ϭ 0.0049 for weekly and P ϭ 0.0416 for every-other-week) after one year. The weekly dosing group experienced a 37-m increase in the 6-minute-walk distance (P ϭ 0.013), a 2.7% increase in percentage of predicted forced vital capacity (P ϭ 0.065), and a 160 mL increase in absolute forced vital capacity (P ϭ 0.001) compared to placebo group at 53 weeks. Idursulfase was generally well tolerated, but infusion reactions did occur.

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Section: Discussionmentioning

confidence: 99%

A phase II/III clinical study of enzyme replacement therapy with idursulfase in mucopolysaccharidosis II (Hunter syndrome)

Muenzer

Wraith

Beck³

et al. 2006

Genetics in Medicine

512

509

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“…5 However, the incidence and risk factors for the development of AIHA, as well as its prognosis and response to treatment are still not well defined. In fact, our current knowledge of AIHA is based on single case reports [6][7][8][9] and small series in adults 4,10 and children. 5 The aim of the study was to analyze the characteristics of patients and transplants, as well as outcome, in a series of patients who developed AIHA after allogeneic HSCT at a single institution.…”

Section: Introductionmentioning

confidence: 99%

Autoimmune hemolytic anemia following allogeneic hematopoietic stem cell transplantation in adult patients

Sanz

Arriaga

Montesinos

et al. 2007

Bone Marrow Transplant

138

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Autoimmune hemolytic anemia (AIHA) after allogeneic hematopoietic stem cell transplantation (HSCT) is still not well characterized. The aim of this study was to analyze the incidence and risk factors for the development of AIHA, as well as its prognosis and response to treatment in a series of patients undergoing allogeneic HSCT at a single institution. Between 1996 and 2004, 272 adult patients with a variety of malignant hematopoietic disorders underwent allogeneic HSCT. Direct antiglobulin testing was performed in routine pretransfusion compatibility testing or after clinical suspicion of AIHA. Twelve patients developed AIHA after HSCT at a median time of 147 days (range, 41-170). The 3-year cumulative incidence of AIHA was 4.44%. Eight cold antibodies and four warm antibodies were detected. Multivariate analysis shows that HSCT from unrelated donors (P ¼ 0.02) and the development of chronic extensive graft-versus-host disease (GVHD) (P ¼ 0.0004) were the only independent factors associated with AIHA. Two patients are still alive. AIHA was never the primary cause of death but added morbidity in patients with other concomitant complications. Patients undergoing HSCT from unrelated donors and those who develop chronic extensive GVHD are especially predisposed for this complication.

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“…5,6 Several reports have considered a dysregulation between B and T cells to be the source of immune problems after transplant. 1,3,4 The absence of certain T cell clones or a differential reconstitution of various T cell subsets, for instance a recently described CD25…”

Section: Discussionmentioning

confidence: 99%

“…[1][2][3][4] A few cases have also been reported after cord blood grafts. 5,6 Descriptions of immune reconstitution in patients with autoimmune complications after transplantation where progenitors have derived from different sources may help in the understanding of such immune complications, including cGHVD. We report a severe immune-mediated hemolytic anemia and the associated lymphocyte subset findings, manifesting as the first cGVHD, in a patient diagnosed with osteopetrosis, 12 months after an unrelated cord blood stem cell transplant.…”

mentioning

confidence: 99%

Acute autoimmune hemolytic anemia following unrelated cord blood transplantation as an early manifestation of chronic graft-versus-host disease

Sevilla

González‐Vicent

Madero

et al. 2001

Bone Marrow Transplant

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Summary:A 16-month-old girl diagnosed with osteopetrosis underwent an unrelated, partially matched (with major mismatch at A locus) cord blood stem cell transplant. Twelve months later she developed severe acute autoimmune hemolytic anemia (AIHA). Immunophenotype analysis of lymphocyte subsets 8 months post transplant showed a low number of T lymphocytes, with normal subsets, and with NK cells and B lymphocytes within normal ranges. When the hemolytic anemia developed, the lymphocytes subsets changed and analysis showed higher numbers of B lymphocytes than previously, lower CD3+ T lymphocytes with inversion of the CD4/CD8 ratio and an abnormal proportion of T lymphocyte subsets. She was being treated with cyclosporine, and steroids and immunoglobulins were added. Initially the AIHA improved, but repeated infectious episodes led us to tail off the immunosuppressive treatment. The AIHA relapsed and cyclosporine was restarted. Currently, she is on cyclosporine and lowdose steroid treatment with no hemolytic features. During the 3 months when the AIHA was being treated, she developed extensive skin cGVHD and recurrent pneumothoraces. AIHA may be the first manifestation of abnormal reconstitution of immunity developing after a hematopoietic transplant. This abnormal reconstitution is also the basis of cGVHD. We suggest that aggressive immunosuppressive treatment with intensive measures against infection could give a better prognosis to such patients. Bone Marrow Transplantation (2001) 28, 89-92.

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Unrelated umbilical cord blood transplantation in infancy for mucopolysaccharidosis type IIB (Hunter syndrome) complicated by autoimmune hemolytic anemia

Cited by 58 publications

References 18 publications

A phase II/III clinical study of enzyme replacement therapy with idursulfase in mucopolysaccharidosis II (Hunter syndrome)

A phase II/III clinical study of enzyme replacement therapy with idursulfase in mucopolysaccharidosis II (Hunter syndrome)

Autoimmune hemolytic anemia following allogeneic hematopoietic stem cell transplantation in adult patients

Acute autoimmune hemolytic anemia following unrelated cord blood transplantation as an early manifestation of chronic graft-versus-host disease

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