Patients with early relapse of primary hemophagocytic syndromes or with persistent CNS involvement may benefit from immediate hematopoietic stem cell transplantation

Sparber‐Sauer, Monika; Hönig, Manfred; Schulz, Ansgar; Stadt, Udo zur; Schütz, Christian; Debatin, Klaus‐Michael; Friedrich, Wilhelm

doi:10.1038/bmt.2009.34

Cited by 24 publications

(14 citation statements)

References 23 publications

(28 reference statements)

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“…Some case series suggest that active disease at transplantation might be a risk factor, especially when an HLA-haploidentical donor is used [10,12]; however, other data indicate that initial response to treatment (CR after 2 months of treatment) could be more informative about the prognosis [11,17,24]. Moreover, in published experiences around 30% to 60% of patients have been transplanted with active disease, indicating that CR is difficult to obtain in many patients with HLH [11][12][13]15,17,24]. Our data could shed further light on this issue: Active disease at transplantation was not statistically associated with adverse outcomes, whereas, interestingly, patients had a trend for a worse outcome if the interval between diagnosis and transplantation was longer than 6 months.…”

Section: Discussionmentioning

confidence: 99%

Outcomes of Children with Hemophagocytic Lymphohistiocytosis Given Allogeneic Hematopoietic Stem Cell Transplantation in Italy

Messina

Zecca

Rovelli

et al. 2018

Biology of Blood and Marrow Transplantation

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We report on 109 patients with hemophagocytic lymphohistiocytosis (HLH) undergoing 126 procedures of allogeneic hematopoietic stem cell transplantation (HSCT) between 2000 and 2014 in centers associated with the Italian Pediatric Hematology Oncology Association. Genetic diagnosis was FHL2 (32%), FHL3 (33%), or other defined disorders known to cause HLH (15%); in the remaining patients no genetic abnormality was found. Donor for first transplant was an HLA-matched sibling for 25 patients (23%), an unrelated donor for 73 (67%), and an HLA-partially matched family donor for 11 children (10%). Conditioning regimen was busulfan-based for 61 patients (56%), treosulfan-based for 21 (20%), and fludarabine-based for 26 children (24%). The 5-year probabilities of overall survival (OS) and event-free survival (EFS) were 71% and 60%, respectively. Twenty-six patients (24%) died due to transplant-related causes, whereas 14 (13%) and 10 (9%) patients experienced graft rejection and/or relapse, respectively. Twelve of 14 children given a second HSCT after graft failure/relapse are alive and disease-free. Use of HLA-partially matched family donors was associated with higher risk of graft failure and thus with lower EFS (but not with lower OS) in multivariable analysis. Active disease at transplantation did not significantly affect prognosis. These data confirm that HSCT can cure most HLH patients, active disease not precluding successful transplantation. Because in HLH patients HLA-haploidentical HSCT performed through CD34 cell positive selection was found to be associated with poor sustained engraftment of donor cells, innovative approaches able to guarantee a more robust engraftment are warranted in patients given this type of allograft.

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Section: Discussionmentioning

confidence: 99%

Outcomes of Children with Hemophagocytic Lymphohistiocytosis Given Allogeneic Hematopoietic Stem Cell Transplantation in Italy

Messina

Zecca

Rovelli

et al. 2018

Biology of Blood and Marrow Transplantation

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“…The best treatment option for CHS is not well defined and it is still controversial (18). Parenteral vitamin C administered in the stable phase may normalize neutrophils bactericidal activity (19,20).…”

Section: Discussionmentioning

confidence: 99%

Chediak-Higashi Syndrome and Premature Exfoliation of Primary Teeth

et al. 2013

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The Chediak-Higashi syndrome (CHS) is a rare hereditary fatal disease, if not treated. These changes are associated with various diseases and syndromes that mainly cause periodontal disease and thus the premature loss of teeth. This paper describes the monitoring of premature loss of primary teeth that began when the child was 5 years old. On presentation his teeth were mobile and there was a history of gingival bleeding. Panoramic radiography revealed generalized and severe bone loss, and the teeth showed no bony support enough for their stability. Blood test was ordered to assess the overall health of the child and giant cells with cytoplasmic granules were found, confirming the diagnosis of CHS. The management of periodontal disease focused on the control of infection and bacterial plaque by mechanical and chemical methods.

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“…Treatment-Long-term survival of over 60% has been reported with HCT therapy, even if treated during an active state of hemophagocytic syndrome (Eapen et al, 2007;Sparber-Sauer et al, 2009).…”

Section: Diseases Of Immune Dysregulationmentioning

confidence: 99%

Periodontal and other oral manifestations of immunodeficiency diseases

2016

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The list of immunodeficiency diseases grows each year as novel disorders are discovered, classified, and sometimes reclassified due to our ever-increasing knowledge of immune system function. Although the number of patients with secondary immunodeficiencies (SIDs) greatly exceeds those with primary immunodeficiencies (PIDs), the prevalence of both appears to be on the rise probably because of scientific breakthroughs that facilitate earlier and more accurate diagnosis. Primary immunodeficiencies in adults are not as rare as once thought. Globally, the main causes of secondary immunodeficiency are HIV infection and nutritional insufficiencies. Persons with acquired immune disorders such as AIDS caused by the Human Immunodeficiency Virus (HIV) are now living long and fulfilling lives as a result of highly active antiretroviral therapy (HAART). Irrespective of whether the patient’s immune deficient state is a consequence of a genetic defect or is secondary in nature, dental and medical practitioners must be aware of the constant potential for infections and/or expressions of autoimmunity in these individuals. The purpose of this paper is to review the most common conditions resulting from primary and secondary immunodeficiency states, how they are classified, and the detrimental manifestations of these disorders on the periodontal and oral tissues.

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Patients with early relapse of primary hemophagocytic syndromes or with persistent CNS involvement may benefit from immediate hematopoietic stem cell transplantation

Cited by 24 publications

References 23 publications

Outcomes of Children with Hemophagocytic Lymphohistiocytosis Given Allogeneic Hematopoietic Stem Cell Transplantation in Italy

Outcomes of Children with Hemophagocytic Lymphohistiocytosis Given Allogeneic Hematopoietic Stem Cell Transplantation in Italy

Chediak-Higashi Syndrome and Premature Exfoliation of Primary Teeth

Periodontal and other oral manifestations of immunodeficiency diseases

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