Fludarabine‐based reduced‐intensity conditioning regimen for hematopoietic stem cell transplantation in primary hemophagocytic lymphohistiocytosis

Hamidieh, Amir Ali; Pourpak, Zahra; Hashemi, Susan; Yari, Kolsoum; Fazlollahi, Mohammad Reza; Movahedi, Masoud; Behfar, Maryam; Moin, Mostafa; Ghavamzadeh, Ardeshir

doi:10.1111/ejh.12244

Cited by 10 publications

(7 citation statements)

References 21 publications

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“…The establishment of diagnostic criteria and the use of immunochemotherapy with or without appropriate allogeneic hematopoietic stem cell transplantation (HSCT) have significantly improved survival outcomes in patients with HLH . Early recognition and the prompt initiation of treatment are mandatory for an improved prognosis.…”

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confidence: 99%

Clinical features, genetics, and outcome of pediatric patients with hemophagocytic lymphohistiocytosis in Korea: report of a nationwide survey from Korea Histiocytosis Working Party

Koh

Chung

et al. 2014

European J of Haematology

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Our study showed the unique predominance of a UNC13D mutation and vulnerability to Epstein-Barr virus infection in Korean children with HLH and emphasizes the prognostic significance of age, liver dysfunction, and treatment responses in this disease. A multicenter prospective trial that builds on the present results is warranted to identify subgroups of patients with a poor prognosis and identify optimal treatments.

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confidence: 99%

Clinical features, genetics, and outcome of pediatric patients with hemophagocytic lymphohistiocytosis in Korea: report of a nationwide survey from Korea Histiocytosis Working Party

Koh

Chung

et al. 2014

European J of Haematology

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“…PIDs are rare genetic diseases and most of them are lethal in childhood, mainly due to serious infections. Despite allogeneic HSCT being recognized as a curative approach in children suffering from various types of PIDs [27][28][29] and in spite of improved techniques a number of issues remain to be answered including the best conditioning regimen and the level of chimerism that would ensure cure. Introduction of RIC has offered many patients ineligible for MAC a chance for cure.…”

Section: Discussionmentioning

confidence: 99%

Long-term outcomes of fludarabine, melphalan and antithymocyte globulin as reduced-intensity conditioning regimen for allogeneic hematopoietic stem cell transplantation in children with primary immunodeficiency disorders: a prospective single center study

Hamidieh

Behfar

Pourpak

et al. 2015

Bone Marrow Transplant

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Reduced-intensity conditioning (RIC) has offered many primary immunodeficiency disorder (PID) patients who are ineligible for myeloablative regimens a chance of cure. However, the beneficial role of RIC was questioned following reports suggesting higher chance of rejection and lower symptom resolution rate in mixed chimerism settings. Forty-five children affected by PIDs with a median age of 21 months underwent allogeneic hematopoietic stem cell transplantation in our institute from 2007 to 2013. All patients received an identical RIC regimen. Forty-one patients had successful primary engraftment (91%). Of the successful engraftments, 80% (n = 33) had stable full donor chimerism at last contact. Overall, eleven transplant-related mortalities were reported including five patients due to sepsis, three children due to grade IV acute GvHD, two due to chronic GvHD and one patient due to sepsis after primary graft failure. The median post-transplantation follow-up of deceased patients was 55 days. Five-year overall survival and disease-free survival was 75.6% and 68.89%, respectively. All surviving patients with successful engraftment became disease free, regardless of having full or mixed chimerism. Our study suggests that RIC regimen provides satisfactory rates of successful engraftment and full chimerism. Furthermore, patients with mixed chimerism were stable in long-term follow-up and this chimerism status offered the potential to resolve symptoms of immunodeficiency.

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“…This resulted in an 89% OS for RIC HSCT compared with 43% OS with myeloablative conditioning at the same institution [18]. Other centers utilized horse antithymocyte globulin as part of RIC with evidence of engraftment in all patients and 80% survival [19]. Recently, Lehmberg et al reported the use of treosulfan-based RIC regimens in 19 patients with HLH.…”

Section: Abstract: Bmt • Children • Complications • Hemophagocytic Lymentioning

confidence: 99%

Risk for complications in patients with hemophagocytic lymphohistiocytosis who undergo hematopoietic stem cell transplantation: myeloablative versus reduced-intensity conditioning regimens

Schechter

Naqvi

Weitzman

2014

Expert Review of Clinical Immunology

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Allogeneic hematopoietic stem cell transplantation (HSCT) is the only curative option for patients with primary hemophagocytic lymphohistiocytosis (HLH) and for patients with secondary HLH who fail to respond to therapy. Advances in HSCT and supportive care measures have resulted in improved patient outcomes and decreased treatment-related mortality. Despite the overall improvement in outcome, HLH patients who undergo HSCT using myeloablative conditioning regimens are still at significant risk for complications. The HLH-94 study conducted by the Histiocyte Society reported a 30% TRM with increased pulmonary and hepatic complications. Recently, the use of reduced-intensity conditioning (RIC) regimens has shown favorable outcomes when compared to conventional HSCT and lower rate of acute complications. In this review we compare the potential complications of myeloablative and RIC regimens for HSCT in HLH patients.

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Fludarabine‐based reduced‐intensity conditioning regimen for hematopoietic stem cell transplantation in primary hemophagocytic lymphohistiocytosis

Cited by 10 publications

References 21 publications

Clinical features, genetics, and outcome of pediatric patients with hemophagocytic lymphohistiocytosis in Korea: report of a nationwide survey from Korea Histiocytosis Working Party

Clinical features, genetics, and outcome of pediatric patients with hemophagocytic lymphohistiocytosis in Korea: report of a nationwide survey from Korea Histiocytosis Working Party

Long-term outcomes of fludarabine, melphalan and antithymocyte globulin as reduced-intensity conditioning regimen for allogeneic hematopoietic stem cell transplantation in children with primary immunodeficiency disorders: a prospective single center study

Risk for complications in patients with hemophagocytic lymphohistiocytosis who undergo hematopoietic stem cell transplantation: myeloablative versus reduced-intensity conditioning regimens

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