Chemoimmunotherapy for hemophagocytic lymphohistiocytosis: long-term results of the HLH-94 treatment protocol.
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Chemoimmunotherapy for hemophagocytic lymphohistiocytosis: long-term results of the HLH-94 treatment protocol. / Trottestam, Helena; Horne, Annacarin; Aricò, Maurizio; Egeler, R Maarten; Filipovich, Alexandra H; Gadner, Helmut; Imashuku, Shinsaku; Ladisch, Stephan; Webb, David; Janka-Schaub, Gritta; Henter, Jan-Inge; Society, Histiocyte.
in: BLOOD, Jahrgang 118, Nr. 17, 17, 2011, S. 4577-4584.Publikationen: SCORING: Beitrag in Fachzeitschrift/Zeitung › SCORING: Zeitschriftenaufsatz › Forschung › Begutachtung
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TY - JOUR
T1 - Chemoimmunotherapy for hemophagocytic lymphohistiocytosis: long-term results of the HLH-94 treatment protocol.
AU - Trottestam, Helena
AU - Horne, Annacarin
AU - Aricò, Maurizio
AU - Egeler, R Maarten
AU - Filipovich, Alexandra H
AU - Gadner, Helmut
AU - Imashuku, Shinsaku
AU - Ladisch, Stephan
AU - Webb, David
AU - Janka-Schaub, Gritta
AU - Henter, Jan-Inge
AU - Society, Histiocyte
PY - 2011
Y1 - 2011
N2 - Hemophagocytic lymphohistiocytosis (HLH) used to have a dismal prognosis. We report the final results of HLH-94, the largest prospective diagnostic/therapeutic HLH study so far. The treatment includes immunosuppressive and cytotoxic therapy aiming at clinical remission, followed by HSCT in patients with familial, persistent, or recurrent disease. Altogether, 249 patients fulfilled inclusion criteria and started HLH-94 therapy (July 1994-December 2003); 227 (91%) were followed-up for ? 5 years. At 6.2 years median follow-up, estimated 5-year probability of survival was 54% ± 6%. Seventy-two patients (29%) died before HSCT, 64 within 1 year, 97% of whom had active disease. In 124 patients who underwent HSCT, 5-year survival was 66 ± 8%; tendency to increased survival (P = .064) in patients with nonactive disease at HSCT. Patients with familial disease had a 5-year survival of 50% ± 13%; none survived without HSCT. Patients deceased during the first 2 months more often had jaundice, edema, and elevated creatinine. Forty-nine patients (20%) were alive without signs of HLH activity and off-therapy > 1-year without HSCT; they presented at older age (P <.001), were more often female (P = .011), and less often had CNS disease (P <.001) or hepatomegaly (P = .007). To conclude, HLH-94 chemoimmunotherapy has considerably improved outcome in HLH. Collaborative efforts are needed to further reduce early mortality, HSCT-related mortality, and neurologic late effects.
AB - Hemophagocytic lymphohistiocytosis (HLH) used to have a dismal prognosis. We report the final results of HLH-94, the largest prospective diagnostic/therapeutic HLH study so far. The treatment includes immunosuppressive and cytotoxic therapy aiming at clinical remission, followed by HSCT in patients with familial, persistent, or recurrent disease. Altogether, 249 patients fulfilled inclusion criteria and started HLH-94 therapy (July 1994-December 2003); 227 (91%) were followed-up for ? 5 years. At 6.2 years median follow-up, estimated 5-year probability of survival was 54% ± 6%. Seventy-two patients (29%) died before HSCT, 64 within 1 year, 97% of whom had active disease. In 124 patients who underwent HSCT, 5-year survival was 66 ± 8%; tendency to increased survival (P = .064) in patients with nonactive disease at HSCT. Patients with familial disease had a 5-year survival of 50% ± 13%; none survived without HSCT. Patients deceased during the first 2 months more often had jaundice, edema, and elevated creatinine. Forty-nine patients (20%) were alive without signs of HLH activity and off-therapy > 1-year without HSCT; they presented at older age (P <.001), were more often female (P = .011), and less often had CNS disease (P <.001) or hepatomegaly (P = .007). To conclude, HLH-94 chemoimmunotherapy has considerably improved outcome in HLH. Collaborative efforts are needed to further reduce early mortality, HSCT-related mortality, and neurologic late effects.
KW - Humans
KW - Male
KW - Female
KW - Adolescent
KW - Child
KW - Survival Analysis
KW - Follow-Up Studies
KW - Time Factors
KW - Combined Modality Therapy
KW - Child, Preschool
KW - Infant
KW - Infant, Newborn
KW - Hematopoietic Stem Cell Transplantation
KW - Drug Combinations
KW - Clinical Protocols
KW - Cytotoxins/administration & dosage/adverse effects
KW - Immunosuppressive Agents/administration & dosage/adverse effects
KW - Immunotherapy/adverse effects/methods
KW - Lymphohistiocytosis, Hemophagocytic/diagnosis/drug therapy/mortality/therapy
KW - Maintenance Chemotherapy
KW - Humans
KW - Male
KW - Female
KW - Adolescent
KW - Child
KW - Survival Analysis
KW - Follow-Up Studies
KW - Time Factors
KW - Combined Modality Therapy
KW - Child, Preschool
KW - Infant
KW - Infant, Newborn
KW - Hematopoietic Stem Cell Transplantation
KW - Drug Combinations
KW - Clinical Protocols
KW - Cytotoxins/administration & dosage/adverse effects
KW - Immunosuppressive Agents/administration & dosage/adverse effects
KW - Immunotherapy/adverse effects/methods
KW - Lymphohistiocytosis, Hemophagocytic/diagnosis/drug therapy/mortality/therapy
KW - Maintenance Chemotherapy
M3 - SCORING: Journal article
VL - 118
SP - 4577
EP - 4584
JO - BLOOD
JF - BLOOD
SN - 0006-4971
IS - 17
M1 - 17
ER -
