Stem cell transplantation for primary immunodeficiencies

Porta, Fulvio; Forino, Concetta; Martiis, D De; Soncini, Elena; Notarangelo, LD; Tettoni, K.; D’Ippolito, Carmelita; Soresina, Roberta; Shiha, K; Berta, S.; Baffelli, R; Bolda, Federica; Bosi, Alberto; Schumacher, Fabian; Lanfranchi, Arnalda; Mazzolari, Evelina

doi:10.1038/bmt.2008.61

Cited by 11 publications

(8 citation statements)

References 7 publications

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“…This study represents one of the largest single‐center studies of outcomes from the United States, representing diverse transplant strategies and demonstrating a favorable outcome in most cases. Survival has been the most consistently reported outcome for HSCT for SCID because data can be obtained from the SCETIDE and CIBMTR registries (European Stem Cell Transplantation for Immunodeficiencies, Center for International Blood and Marrow Transplant Research) (13, 34). Compared to the most recent reporting from these registries where overall survival was approximately 75%, our patient cohort had a higher survival rate of 86–88%.…”

Section: Discussionmentioning

confidence: 99%

A single‐center study of hematopoietic stem cell transplantation for primary immune deficiencies (PIDD)

DiNardo¹,

Brown²,

Perez

et al. 2011

Pediatric Transplantation

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PIDD are rare inherited disorders that can result in life-threatening infections. Allogeneic HSCT is the only cure for many primary immune deficiencies; however, the specific diseases and optimal type(s) of transplants are not clear. This study compares transplant outcomes in a large cohort with a relatively uniform pre- and post-transplant management strategies. We conducted a retrospective analysis of 39 pediatric patients who underwent HSCT for SCID (n = 25) or other immune deficiencies (n = 14) from 1986 to 2010. A structured case report form was used to collect clinical information. The outcomes of survival, immune reconstitution, engraftment, incidence of GvHD and IVIG dependency were tabulated. Overall survival rates were 88% for SCID and 86% for other primary immune deficiencies, which are high compared to other historical series. No single variable was associated with mortality. Immunoglobulin dependence occurred only in patients who had X-linked SCID and a parental donor haploidentical transplant. Because of improved supportive care and use of alternative donors and conditioning regimens, HSCT has become an acceptable option for an increasing number of PIDD subtypes not previously transplanted with high frequency. This study encourages greater use of transplantation.

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Section: Discussionmentioning

confidence: 99%

A single‐center study of hematopoietic stem cell transplantation for primary immune deficiencies (PIDD)

DiNardo¹,

Brown²,

Perez

et al. 2011

Pediatric Transplantation

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“…The database demonstrated that CGD was more common than perceived, and showed that significant morbidity and mortality were pervasive in CGD [17]. These facts served to accelerate bone marrow transplantation in CGD in the United States and in Europe [19,20]. In Europe, the seminal data collection on children with hyper-IgM syndrome revealed that severe liver diseases were more common than appreciated previously [21][22][23].…”

Section: Pid Patient Registriesmentioning

confidence: 98%

Immunodeficiencies

Ballow

Notarangelo

Grimbacher

et al. 2009

Clinical and Experimental Immunology

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SummaryPrimary immunodeficiencies (PIDs) are uncommon, chronic and severe disorders of the immune system in which patients cannot mount a sufficiently protective immune response, leading to an increased susceptibility to infections. The treatment of choice for PID patients with predominant antibody deficiency is intravenous immunoglobulin (Ig) replacement therapy. Despite major advances over the last 20 years in the molecular characterization of PIDs, many patients remain undiagnosed or are diagnosed too late, with severe consequences. Various strategies to ensure timely diagnosis of PIDs are in place, and novel approaches are being developed. In recent years, several patient registries have been established. Such registries shed light on the pathology and natural history of these varied disorders. Analyses of the registry data may also reveal which patients are likely to respond well to higher Ig infusion rates and may help to determine the optimal dosing of Ig products. Faster infusion rates may lead to improved convenience for patients and thus increase patient compliance, and may reduce nursing time and the need for hospital resources. Data from two recent studies suggest that Gamunex® and Privigen® are well tolerated at high infusion rates. Nevertheless, careful selection of patients for high infusion rates, based on co-morbid conditions and tolerance of the current infusion rate, is advisable. Based on the available data, intravenous Ig offers broad protection against encapsulated organisms. As vaccine trends change, careful monitoring of specific antibody levels in the general population, such as those against pneumococcal and meningococcal bacteria, should be implemented.

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“…Hematopoietic stem cell transplantation (HSCT) is a therapeutic option for a number of non-malignant disorders, such as autoimmune disorders [1], inborn metabolic disorders [2], hemoglobinopathies [3], and immunodeficiency disorders (IDD) [4]. Autoimmune complications (AICs) after HSCT are still not well characterized but have multifactorial occurrence, such as autoimmune cytopenias (AICPs), autoimmune hepatitis (AIH), and autoimmune thyroid disease (AITD).…”

Section: Introductionmentioning

confidence: 99%

Autoimmune Complications after Hematopoietic Stem Cell Transplantation in Children with Nonmalignant Disorders

Khalil

Zaidman

Bergman

et al. 2014

The Scientific World Journal

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Background. Hematopoietic stem cell transplantation (HSCT) remains the only curative treatment for many nonmalignant disorders, such as autoimmune disorders, inborn metabolic disorders, hemoglobinopathies, and immunodeficiency disorders. Autoimmune complications (AICs) after HSCT, such as autoimmune cytopenias, autoimmune hepatitis, primary biliary cirrhosis, and autoimmune cutaneous manifestations, are still neither well defined nor characterized. Patients. Between 2000 and 2012, 92 patients (47 males, 45 females) were treated with HSCT in our hospital, 51 with congenital hemoglobinopathies, 19 with primary immunodeficiency disease, 10 with metabolic disorders, five with Fanconi anemia, three with aplastic anemia, and four with familial hemophagocytic lymphohistiocytosis. Results. Mean age at HSCT was 6.4 years (range, 0.2–32 years) and mean duration of followup after HSCT was 6.81 years (range, 1–11 years). Sixteen (17.4%) patients developed chronic GVHD and five (5.4%) showed sclerodermatous features. Five (5.4%) patients were diagnosed with scleroderma manifestations, six (6.5%) with vitiligo, six (6.5%) with autoimmune hemolytic anemia (AIHA), six (6.5%) with idiopathic thrombocytopenia, three (3.3%) with mild leucopenia, two (2.2%) with aplastic anemia, two (2.2%) (one boy, one girl) with autoimmune thyroid disease, and one (1.1%) with autoimmune hepatitis. Conclusions. It was concluded that AICs are clinically significant complications after HSCT that contribute to morbidity but not to mortality. AICs are more frequent after HSCT for metabolic disorders, and sclerodermatous GVHD is more significant in children who underwent allogeneic HSCT for hemoglobinopathies. The potential to identify risk factors for AICs could lead to less morbidity and mortality and to maintain the patient's quality of life.

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Stem cell transplantation for primary immunodeficiencies

Cited by 11 publications

References 7 publications

A single‐center study of hematopoietic stem cell transplantation for primary immune deficiencies (PIDD)

A single‐center study of hematopoietic stem cell transplantation for primary immune deficiencies (PIDD)

Immunodeficiencies

Autoimmune Complications after Hematopoietic Stem Cell Transplantation in Children with Nonmalignant Disorders

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