Outcomes of allogeneic hematopoietic stem cell transplantation for primary hemophagocytic lymphohistiocytosis in adults.

Allogeneic hematopoietic stem cell transplantation (HSCT) is the only curative approach for primary hemophagocytic lymphohistiocytosis (pHLH), but data on adult patients are scarce. Here we present an 8-year experience on HSCT for adult pHLH to reveal the benefits and risks in this population. A total of 29 adult pHLH patients entered this study, at a median follow-up of 29 months (3-112 months), the 5-year probability of survival was 60%. Six patients rejected HSCT, of whom 1 alive in complete response (CR). In 23 patients who underwent HSCT, 5-year survival post-HSCT overall was 73%, and in haploidentical HSCT (haplo-HSCT) cases, 71%. Patients who achieved CR at HSCT had a better outcome than those of partial response (92% vs. 47%, p = 0.013). Neither the use of HLA mismatched donor (75% vs. 72%, p = 0.996) nor the use of donor with monoallelic mutation (74% vs. 71%, p = 0.901) affected the prognosis. Hemophagocytic lymphohistiocytosis status of CR at HSCT was a positive prognostic factor. We concluded that HSCT is a promising method to cure adult-onset pHLH. Achieving CR before HSCT contributes to better outcome. Haplo-HSCT is safe and effective for adult pHLH patients, donors with monoallelic mutations in pHLH related genes but normal cytotoxic functions are reliable.

He L ，Wang Y ，Suolitiken D ，Zhang R ，Liu M ，Hua Z ，Yao S ，Zou H ，Wang Z ... -  《-》

Treatment of pediatric primary hemophagocytic lymphohistiocytosis with the HLH-94/2004 regimens and hematopoietic stem cell transplantation in China.

Ma H ，Zhang R ，Zhang L ，Wei A ，Zhao X ，Yang Y ，Liu W ，Li Z ，Qin M ，Wang T ... -  《-》

[Clinical study of haploidentical hematopoietic stem cell transplantation on 15 cases of adult-onset primary hemophagocytic lymphohistiocytosis].

Wang YN ，Wang JS ，Wei N ，Wu L ，Jin ZL ，Wang Z ... -  《-》

[The clinical characteristics of adult hemophagocytic lymphohistiocytosis treated with haploidentical donor hematopoietic stem cell transplantation].

Fu L ，Wei N ，Wang JS ，Wu L ，Wang YN ，Huang DY ，Liu JL ，Wang Z ... -  《-》

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

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.

Messina C ，Zecca M ，Fagioli F ，Rovelli A ，Giardino S ，Merli P ，Porta F ，Aricò M ，Sieni E ，Basso G ，Ripaldi M ，Favre C ，Pillon M ，Marzollo A ，Rabusin M ，Cesaro S ，Algeri M ，Caniglia M ，Di Bartolomeo P ，Ziino O ，Saglio F ，Prete A ，Locatelli F ... -  《-》