Hematopoietic stem cell transplantation for hemophagocytic lymphohistiocytosis: a retrospective analysis of data from the Italian Association of Pediatric Hematology Oncology (AIEOP)

Abstract

BackgroundHemophagocytic lymphohistiocytosis is a life-threatening disease. Hematopoietic stem cell transplantation still represents the treatment of choice for most patients with this disease.

Allogeneic hematopoietic cell transplantation for XIAP deficiency: an international survey reveals poor outcomes

“…This is in sharp contrast to the typical survival rates in other forms of HLH, which are generally greater than 50%. [1][2][3][4][5][6][7][11][12][13] There was a preponderance of hepatic VOD and pulmonary hemorrhage in MAC patients. Although VOD has been reported in patients with HLH who undergo MAC regimens, it appears that the 50% incidence of VOD in this series is high compared with previous reports of 20%-30%.…”

“…[4][5][6][7][8][9][10][11][12][13][14][15][16][17][18][19]. Patient characteristics before HCT and XIAP/BIRC4 mutations are listed in Table 1.…”

“…Over the past 10 years, survival has generally approximated 60% with myeloablative conditioning (MAC) regimens. [1][2][3][4][5][6][7] More recently, improvements have been made with reduced-intensity conditioning (RIC) protocols, and current survival rates are as high as 80%. [8][9][10][11] There is less experience with transplantation in patients with XLP because of SLAM-associated protein (SAP) deficiency, but survival is generally accepted to be greater than 70% regardless of the intensity of the conditioning protocol.…”

Hematopoietic stem cell transplantation in Griscelli syndrome type 2: a single-center report on 10 patients

“…12 Few cases of successful HSCT from compatible donors have been reported in children with GS2 thereafter. [13][14][15][16][17] A recent study emphasizes the neurologic sequelae in 5 of 5 GS2 patients who received HSCT after treatment according to the HLH-94/HLH-2004 protocol. 18 The present study shows and discusses the results and complications of HSCT performed in 10 GS2 patients in a single center between 1996 and 2008. neurologic involvement and another additional patient's family refused HSCT.…”

Autoimmune-Associated Hemophagocytic Syndrome/Macrophage Activation Syndrome

“…Since then, information regarding the role of alloSCT in the treatment of HLH has mostly concerned FHL (Janka, 2009;Marsh et al, 2010 (Baker et al, 1997;Cesaro et al, 2008;Dürken et al, 1999;Horne et al, 2005;Imashuku et al, 1999;Jabado et al, 1997;Ouachèe-Chardin et al, 2006), and 75-92% with reduced-intensity conditioning (RIC) (Cooper et al, 2006;Marsh et al, 2010). So far, alloSCT has been performed only occasionally in cases of acquired HLH and its role in the treatment of sHLH is not yet established.…”