Trial Outcomes & Findings for Evaluation of IFNγ and Inflammatory Mediators in Patients With Malignancy-Associated Hemophagocytic Lymphohistiocytosis (NCT NCT03259230)

NCT ID: NCT03259230

Last Updated: 2024-09-19

Results Overview

Serum samples was collected at various times after HLH diagnosis to determine the concentration of CXCL10.

• Recruitment status: COMPLETED
• Target enrollment: 40 participants
• Primary outcome timeframe: From Day 1 up to 39.5 months
• Results posted on: 2024-09-19

Participant Flow

This Phase 4 observational study was conducted in patients diagnosed with malignancy-associated hemophagocytic lymphohistiocytosis (M-HLH) at a single center in the United States of America.

A total of 40 patients were enrolled in the study. Of which 39 patients had a confirmed malignancy and were included in the malignancy set for analysis.

Participant milestones

Measure	Hemophagocytic Lymphohistiocytosis (HLH) Patients All patients diagnosed with HLH in the context of a malignancy.	Non-HLH Patients All patients with a diagnosis of hematological malignancy and never diagnosed with HLH or no suspicion of HLH (at any time, including time after sample collection in case of use of retrospective sample).
Overall Study STARTED	20	20
Overall Study Malignancy Analysis Set	19	20
Overall Study COMPLETED	19	20
Overall Study NOT COMPLETED	1	0

Reasons for withdrawal

Measure	Hemophagocytic Lymphohistiocytosis (HLH) Patients All patients diagnosed with HLH in the context of a malignancy.	Non-HLH Patients All patients with a diagnosis of hematological malignancy and never diagnosed with HLH or no suspicion of HLH (at any time, including time after sample collection in case of use of retrospective sample).
Overall Study Excluded from the Malignancy Analysis Set	1	0

Baseline Characteristics

Race and Ethnicity were not collected from any participant.

Baseline characteristics by cohort

Measure	HLH Patients n=19 Participants All patients diagnosed with HLH in the context of a malignancy.	Non-HLH Patients n=20 Participants All patients with a diagnosis of hematological malignancy and never diagnosed with HLH or no suspicion of HLH (at any time, including time after sample collection in case of use of retrospective sample).	Total n=39 Participants Total of all reporting groups
Age, Continuous	56.7 years STANDARD_DEVIATION 18.6 • n=19 Participants	63.2 years STANDARD_DEVIATION 15.3 • n=20 Participants	60.0 years STANDARD_DEVIATION 17.1 • n=39 Participants
Sex: Female, Male Female	4 Participants n=19 Participants	8 Participants n=20 Participants	12 Participants n=39 Participants
Sex: Female, Male Male	15 Participants n=19 Participants	12 Participants n=20 Participants	27 Participants n=39 Participants
Race and Ethnicity Not Collected	—	—	0 Participants Race and Ethnicity were not collected from any participant.

PRIMARY outcome

Timeframe: From Day 1 up to 39.5 months

Population: The malignancy set included all patients who were diagnosed with malignancy. Only patients with data available are reported.

Serum samples was collected at various times after HLH diagnosis to determine the concentration of CXCL10.

Outcome measures

Measure	HLH Patients n=19 Participants All patients diagnosed with HLH in the context of a malignancy.	Non-HLH Patients n=5 Participants All patients with a diagnosis of hematological malignancy and never diagnosed with HLH or no suspicion of HLH (at any time, including time after sample collection in case of use of retrospective sample).
Mean Serum Concentration of Inflammatory Biomarker - C-X-C Chemokine Ligand 10 (CXCL10)	5040.638 picogram/milliliter (pg/mL) Standard Deviation 9622.745	454.392 picogram/milliliter (pg/mL) Standard Deviation 246.307

PRIMARY outcome

Timeframe: From Day 1 up to 39.5 months

Population: The malignancy set included all patients who were diagnosed with malignancy.

Serum samples was collected at various times after HLH diagnosis to determine the concentration of CXCL9.

Outcome measures

Measure	HLH Patients n=19 Participants All patients diagnosed with HLH in the context of a malignancy.	Non-HLH Patients n=19 Participants All patients with a diagnosis of hematological malignancy and never diagnosed with HLH or no suspicion of HLH (at any time, including time after sample collection in case of use of retrospective sample).
Mean Serum Concentration of Inflammatory Biomarker - C-X-C Chemokine Ligand 9 (CXCL9)	8878.698 pg/mL Standard Deviation 17582.571	1864.255 pg/mL Standard Deviation 4986.931

PRIMARY outcome

Timeframe: From Day 1 up to 39.5 months

Population: The malignancy set included all patients who were diagnosed with malignancy.

Serum samples was collected at various times after HLH diagnosis to determine the concentration of IFNg.

Outcome measures

Measure	HLH Patients n=19 Participants All patients diagnosed with HLH in the context of a malignancy.	Non-HLH Patients n=19 Participants All patients with a diagnosis of hematological malignancy and never diagnosed with HLH or no suspicion of HLH (at any time, including time after sample collection in case of use of retrospective sample).
Mean Serum Concentration of Inflammatory Biomarker - Interferon Gamma (IFNg)	279.951 pg/mL Standard Deviation 898.740	8.014 pg/mL Standard Deviation 2.370

PRIMARY outcome

Timeframe: From Day 1 up to 39.5 months

Population: The malignancy set included all patients who were diagnosed with malignancy.

Serum samples was collected at various times after HLH diagnosis to determine the concentration of IL-10.

Outcome measures

Measure	HLH Patients n=19 Participants All patients diagnosed with HLH in the context of a malignancy.	Non-HLH Patients n=19 Participants All patients with a diagnosis of hematological malignancy and never diagnosed with HLH or no suspicion of HLH (at any time, including time after sample collection in case of use of retrospective sample).
Mean Serum Concentration of Inflammatory Biomarker - Interleukin 10 (IL-10)	46.562 pg/mL Standard Deviation 124.537	2.185 pg/mL Standard Deviation 2.953

PRIMARY outcome

Timeframe: From Day 1 up to 39.5 months

Population: The malignancy set included all patients who were diagnosed with malignancy. Only patients with data available are reported.

Serum samples was collected at various times after HLH diagnosis to determine the concentration of IL-1B.

Outcome measures

Measure	HLH Patients n=19 Participants All patients diagnosed with HLH in the context of a malignancy.	Non-HLH Patients n=5 Participants All patients with a diagnosis of hematological malignancy and never diagnosed with HLH or no suspicion of HLH (at any time, including time after sample collection in case of use of retrospective sample).
Mean Serum Concentration of Inflammatory Biomarker - Interleukin 1 Beta (IL-1B)	2.140 pg/mL Standard Deviation 0.000	2.140 pg/mL Standard Deviation 0.000

PRIMARY outcome

Timeframe: From Day 1 up to 39.5 months

Population: The malignancy set included all patients who were diagnosed with malignancy.

Serum samples was collected at various times after HLH diagnosis to determine the concentration of IL-6.

Outcome measures

Measure	HLH Patients n=19 Participants All patients diagnosed with HLH in the context of a malignancy.	Non-HLH Patients n=19 Participants All patients with a diagnosis of hematological malignancy and never diagnosed with HLH or no suspicion of HLH (at any time, including time after sample collection in case of use of retrospective sample).
Mean Serum Concentration of Inflammatory Biomarker - Interleukin 6 (IL-6)	597.385 pg/mL Standard Deviation 1776.853	10.059 pg/mL Standard Deviation 28.771

PRIMARY outcome

Timeframe: From Day 1 up to 39.5 months

Population: The malignancy set included all patients who were diagnosed with malignancy. Only patients with data available are reported.

Serum samples was collected at various times after HLH diagnosis to determine the concentration of neopterin.

Outcome measures

Measure	HLH Patients n=14 Participants All patients diagnosed with HLH in the context of a malignancy.	Non-HLH Patients n=19 Participants All patients with a diagnosis of hematological malignancy and never diagnosed with HLH or no suspicion of HLH (at any time, including time after sample collection in case of use of retrospective sample).
Mean Serum Concentration of Inflammatory Biomarker - Neopterin	257.120 nanomoles Standard Deviation 223.475	51.909 nanomoles Standard Deviation 47.869

PRIMARY outcome

Timeframe: From Day 1 up to 39.5 months

Population: The malignancy set included all patients who were diagnosed with malignancy.

Serum samples was collected at various times after HLH diagnosis to determine the concentration of TNFa.

Outcome measures

Measure	HLH Patients n=19 Participants All patients diagnosed with HLH in the context of a malignancy.	Non-HLH Patients n=19 Participants All patients with a diagnosis of hematological malignancy and never diagnosed with HLH or no suspicion of HLH (at any time, including time after sample collection in case of use of retrospective sample).
Mean Serum Concentration of Inflammatory Biomarker - Tumor Necrosis Factor Alpha (TNFa)	12.412 pg/mL Standard Deviation 14.899	4.751 pg/mL Standard Deviation 4.054

PRIMARY outcome

Timeframe: From Day 1 up to 39.5 months

Population: The malignancy set included all patients who were diagnosed with malignancy.

Serum samples was collected at various times after HLH diagnosis to determine the concentration of hIFNg.

Outcome measures

Measure	HLH Patients n=19 Participants All patients diagnosed with HLH in the context of a malignancy.	Non-HLH Patients n=19 Participants All patients with a diagnosis of hematological malignancy and never diagnosed with HLH or no suspicion of HLH (at any time, including time after sample collection in case of use of retrospective sample).
Mean Serum Concentration of Inflammatory Biomarker - Total Human Interferon Gamma (hIFNg)	82.227 pg/mL Standard Deviation 196.240	417.603 pg/mL Standard Deviation 1573.368

PRIMARY outcome

Timeframe: From Day 1 up to 39.5 months

Population: The malignancy set included all patients who were diagnosed with malignancy.

Serum samples was collected at various times after HLH diagnosis to determine the concentration of sCD163.

Outcome measures

Measure	HLH Patients n=19 Participants All patients diagnosed with HLH in the context of a malignancy.	Non-HLH Patients n=19 Participants All patients with a diagnosis of hematological malignancy and never diagnosed with HLH or no suspicion of HLH (at any time, including time after sample collection in case of use of retrospective sample).
Mean Serum Concentration of Inflammatory Biomarker - Soluble CD163 (sCD163)	3796.175 nanogram/mL Standard Deviation 3154.561	1812.384 nanogram/mL Standard Deviation 2852.523

PRIMARY outcome

Timeframe: From Day 1 up to 39.5 months

Population: The malignancy set included all patients who were diagnosed with malignancy.

Serum samples was collected at various times after HLH diagnosis to determine the concentration of sIL2Ra.

Outcome measures

Measure	HLH Patients n=19 Participants All patients diagnosed with HLH in the context of a malignancy.	Non-HLH Patients n=19 Participants All patients with a diagnosis of hematological malignancy and never diagnosed with HLH or no suspicion of HLH (at any time, including time after sample collection in case of use of retrospective sample).
Mean Serum Concentration of Inflammatory Biomarker - Soluble IL-2 Receptor Alpha (sIL2Ra)	10183.509 pg/mL Standard Deviation 12244.564	2575.569 pg/mL Standard Deviation 3395.536

PRIMARY outcome

Timeframe: From Day 1 up to 39.5 months

Population: The malignancy set included all patients who were diagnosed with malignancy. Only patients with data available are reported.

Blood samples was collected at various times after HLH diagnosis to determine the concentration of hemoglobin.

Outcome measures

Measure	HLH Patients n=18 Participants All patients diagnosed with HLH in the context of a malignancy.	Non-HLH Patients n=5 Participants All patients with a diagnosis of hematological malignancy and never diagnosed with HLH or no suspicion of HLH (at any time, including time after sample collection in case of use of retrospective sample).
Mean Serum Concentration of Hemoglobin	9.31 gram/deciliter (g/dL) Standard Deviation 0.98	8.52 gram/deciliter (g/dL) Standard Deviation 1.95

PRIMARY outcome

Timeframe: From Day 1 up to 39.5 months

Population: The malignancy set included all patients who were diagnosed with malignancy. Only patients with data available are reported.

Blood samples was collected at various times after HLH diagnosis to determine the concentration of hematocrit.

Outcome measures

Measure	HLH Patients n=18 Participants All patients diagnosed with HLH in the context of a malignancy.	Non-HLH Patients n=5 Participants All patients with a diagnosis of hematological malignancy and never diagnosed with HLH or no suspicion of HLH (at any time, including time after sample collection in case of use of retrospective sample).
Mean Serum Concentration of Hematocrit	27.28 liter (L) of cells/L of blood Standard Deviation 3.41	25.94 liter (L) of cells/L of blood Standard Deviation 4.99

PRIMARY outcome

Timeframe: From Day 1 up to 39.5 months

Population: The malignancy set included all patients who were diagnosed with malignancy. Only patients with data available are reported.

Blood samples was collected at various times after HLH diagnosis to determine the concentration of erythrocytes.

Outcome measures

Measure	HLH Patients n=18 Participants All patients diagnosed with HLH in the context of a malignancy.	Non-HLH Patients n=5 Participants All patients with a diagnosis of hematological malignancy and never diagnosed with HLH or no suspicion of HLH (at any time, including time after sample collection in case of use of retrospective sample).
Mean Serum Concentration of Erythrocytes	3.208 cells*10^12/L Standard Deviation 0.371	2.896 cells*10^12/L Standard Deviation 0.543

PRIMARY outcome

Timeframe: From Day 1 up to 39.5 months

Population: The malignancy set included all patients who were diagnosed with malignancy. Only patients with data available are reported.

Blood samples was collected at various times after HLH diagnosis to determine the concentration of leukocytes.

Outcome measures

Measure	HLH Patients n=18 Participants All patients diagnosed with HLH in the context of a malignancy.	Non-HLH Patients n=5 Participants All patients with a diagnosis of hematological malignancy and never diagnosed with HLH or no suspicion of HLH (at any time, including time after sample collection in case of use of retrospective sample).
Mean Serum Concentration of Leukocytes	2.11 cells*10^9/L Standard Deviation 2.85	8.42 cells*10^9/L Standard Deviation 8.94

PRIMARY outcome

Timeframe: From Day 1 up to 39.5 months

Population: The malignancy set included all patients who were diagnosed with malignancy. Only patients with data available are reported.

Blood samples was collected at various times after HLH diagnosis to determine the concentration of neutrophils.

Outcome measures

Measure	HLH Patients n=18 Participants All patients diagnosed with HLH in the context of a malignancy.	Non-HLH Patients n=5 Participants All patients with a diagnosis of hematological malignancy and never diagnosed with HLH or no suspicion of HLH (at any time, including time after sample collection in case of use of retrospective sample).
Mean Serum Concentration of Neutrophils	1.743 cells*10^3/microliter (mcL) Standard Deviation 2.550	1.984 cells*10^3/microliter (mcL) Standard Deviation 1.776

PRIMARY outcome

Timeframe: From Day 1 up to 39.5 months

Population: The malignancy set included all patients who were diagnosed with malignancy. Only patients with data available are reported.

Blood samples was collected at various times after HLH diagnosis to determine the concentration of lymphocytes.

Outcome measures

Measure	HLH Patients n=18 Participants All patients diagnosed with HLH in the context of a malignancy.	Non-HLH Patients n=5 Participants All patients with a diagnosis of hematological malignancy and never diagnosed with HLH or no suspicion of HLH (at any time, including time after sample collection in case of use of retrospective sample).
Mean Serum Concentration of Lymphocytes	0.468 cells*10^3/mcL Standard Deviation 0.647	0.968 cells*10^3/mcL Standard Deviation 1.084

PRIMARY outcome

Timeframe: From Day 1 up to 39.5 months

Population: The malignancy set included all patients who were diagnosed with malignancy. Only patients with data available are reported.

Blood samples was collected at various times after HLH diagnosis to determine the concentration of monocytes.

Outcome measures

Measure	HLH Patients n=10 Participants All patients diagnosed with HLH in the context of a malignancy.	Non-HLH Patients n=5 Participants All patients with a diagnosis of hematological malignancy and never diagnosed with HLH or no suspicion of HLH (at any time, including time after sample collection in case of use of retrospective sample).
Mean Serum Concentration of Monocytes	0.233 cells*10^3/mcL Standard Deviation 0.272	1.278 cells*10^3/mcL Standard Deviation 1.034

PRIMARY outcome

Timeframe: From Day 1 up to 39.5 months

Population: The malignancy set included all patients who were diagnosed with malignancy. Only patients with data available are reported.

Blood samples was collected at various times after HLH diagnosis to determine the concentration of eosinophils.

Outcome measures

Measure	HLH Patients n=4 Participants All patients diagnosed with HLH in the context of a malignancy.	Non-HLH Patients n=3 Participants All patients with a diagnosis of hematological malignancy and never diagnosed with HLH or no suspicion of HLH (at any time, including time after sample collection in case of use of retrospective sample).
Mean Serum Concentration of Eosinophils	0.032 cells*10^3/mcL Standard Deviation 0.044	1.083 cells*10^3/mcL Standard Deviation 1.441

PRIMARY outcome

Timeframe: From Day 1 up to 39.5 months

Population: The malignancy set included all patients who were diagnosed with malignancy. Only patients with data available are reported.

Blood samples was collected at various times after HLH diagnosis to determine the concentration of basophils.

Outcome measures

Measure	HLH Patients n=2 Participants All patients diagnosed with HLH in the context of a malignancy.	Non-HLH Patients n=2 Participants All patients with a diagnosis of hematological malignancy and never diagnosed with HLH or no suspicion of HLH (at any time, including time after sample collection in case of use of retrospective sample).
Mean Serum Concentration of Basophils	0.100 cells*10^3/mcL Standard Deviation 0.087	0.820 cells*10^3/mcL Standard Deviation 0.834

PRIMARY outcome

Timeframe: From Day 1 up to 39.5 months

Population: The malignancy set included all patients who were diagnosed with malignancy. Only patients with data available are reported.

Blood samples was collected at various times after HLH diagnosis to determine the concentration of platelets.

Outcome measures

Measure	HLH Patients n=18 Participants All patients diagnosed with HLH in the context of a malignancy.	Non-HLH Patients n=5 Participants All patients with a diagnosis of hematological malignancy and never diagnosed with HLH or no suspicion of HLH (at any time, including time after sample collection in case of use of retrospective sample).
Mean Serum Concentration of Platelets	43.9 cells*10^3/mcL Standard Deviation 67.7	30.8 cells*10^3/mcL Standard Deviation 38.5

PRIMARY outcome

Timeframe: From Day 1 up to 39.5 months

Population: The malignancy set included all patients who were diagnosed with malignancy. Only patients with data available are reported.

Blood samples was collected at various times after HLH diagnosis to determine the concentration of fibrinogen.

Outcome measures

Measure	HLH Patients n=18 Participants All patients diagnosed with HLH in the context of a malignancy.	Non-HLH Patients n=2 Participants All patients with a diagnosis of hematological malignancy and never diagnosed with HLH or no suspicion of HLH (at any time, including time after sample collection in case of use of retrospective sample).
Mean Plasma Concentration of Fibrinogen	207.5 milligram (mg)/dL Standard Deviation 143.0	357.5 milligram (mg)/dL Standard Deviation 2.1

PRIMARY outcome

Timeframe: From Day 1 up to 39.5 months

Population: The malignancy set included all patients who were diagnosed with malignancy. Only patients with data available are reported.

Blood samples was collected at various times after HLH diagnosis to determine the concentration of D-dimer.

Outcome measures

Measure	HLH Patients n=15 Participants All patients diagnosed with HLH in the context of a malignancy.	Non-HLH Patients n=2 Participants All patients with a diagnosis of hematological malignancy and never diagnosed with HLH or no suspicion of HLH (at any time, including time after sample collection in case of use of retrospective sample).
Mean Plasma Concentration of D-Dimer	10.165 microgram (mcg)/mL Standard Deviation 7.569	2.220 microgram (mcg)/mL Standard Deviation 2.107

PRIMARY outcome

Timeframe: From Day 1 up to 39.5 months

Population: The malignancy set included all patients who were diagnosed with malignancy. Only patients with data available are reported.

Blood samples was collected at various times after HLH diagnosis to determine the concentration of ferritin.

Outcome measures

Measure	HLH Patients n=18 Participants All patients diagnosed with HLH in the context of a malignancy.	Non-HLH Patients n=2 Participants All patients with a diagnosis of hematological malignancy and never diagnosed with HLH or no suspicion of HLH (at any time, including time after sample collection in case of use of retrospective sample).
Mean Serum Concentration of Ferritin	17240.3470 mcg/L Standard Deviation 30134.8903	3.8940 mcg/L Standard Deviation 4.1748

PRIMARY outcome

Timeframe: From Day 1 up to 39.5 months

Population: The malignancy set included all patients who were diagnosed with malignancy. Only patients with data available are reported.

Blood samples was collected at various times after HLH diagnosis to determine the concentration of fasting triglycerides.

Outcome measures

Measure	HLH Patients n=16 Participants All patients diagnosed with HLH in the context of a malignancy.	Non-HLH Patients All patients with a diagnosis of hematological malignancy and never diagnosed with HLH or no suspicion of HLH (at any time, including time after sample collection in case of use of retrospective sample).
Mean Serum Concentration of Fasting Triglycerides	231.5 mg/dL Standard Deviation 88.6	—

PRIMARY outcome

Timeframe: From Day 1 up to 39.5 months

Population: The malignancy set included all patients who were diagnosed with malignancy. Only patients with data available are reported.

Blood samples was collected at various times after HLH diagnosis to determine the concentration of creatinine.

Outcome measures

Measure	HLH Patients n=18 Participants All patients diagnosed with HLH in the context of a malignancy.	Non-HLH Patients n=4 Participants All patients with a diagnosis of hematological malignancy and never diagnosed with HLH or no suspicion of HLH (at any time, including time after sample collection in case of use of retrospective sample).
Mean Serum Concentration of Creatinine	1.682 mg/dL Standard Deviation 2.726	0.808 mg/dL Standard Deviation 0.287

PRIMARY outcome

Timeframe: From Day 1 up to 39.5 months

Population: The malignancy set included all patients who were diagnosed with malignancy. Only patients with data available are reported.

Blood samples was collected at various times after HLH diagnosis to determine the concentration of blood urea nitrogen.

Outcome measures

Measure	HLH Patients n=13 Participants All patients diagnosed with HLH in the context of a malignancy.	Non-HLH Patients n=4 Participants All patients with a diagnosis of hematological malignancy and never diagnosed with HLH or no suspicion of HLH (at any time, including time after sample collection in case of use of retrospective sample).
Mean Plasma Concentration of Blood Urea Nitrogen	44.68 mg/dL Standard Deviation 30.53	29.00 mg/dL Standard Deviation 3.92

PRIMARY outcome

Timeframe: From Day 1 up to 39.5 months

Population: The malignancy set included all patients who were diagnosed with malignancy. Only patients with data available are reported.

Blood samples was collected at various times after HLH diagnosis to determine the concentration of albumin.

Outcome measures

Measure	HLH Patients n=18 Participants All patients diagnosed with HLH in the context of a malignancy.	Non-HLH Patients n=4 Participants All patients with a diagnosis of hematological malignancy and never diagnosed with HLH or no suspicion of HLH (at any time, including time after sample collection in case of use of retrospective sample).
Mean Serum Concentration of Albumin	2.88 g/dL Standard Deviation 0.60	3.48 g/dL Standard Deviation 0.82

PRIMARY outcome

Timeframe: From Day 1 up to 39.5 months

Population: The malignancy set included all patients who were diagnosed with malignancy. Only patients with data available are reported.

Blood samples was collected at various times after HLH diagnosis to determine the concentration of sodium.

Outcome measures

Measure	HLH Patients n=18 Participants All patients diagnosed with HLH in the context of a malignancy.	Non-HLH Patients n=4 Participants All patients with a diagnosis of hematological malignancy and never diagnosed with HLH or no suspicion of HLH (at any time, including time after sample collection in case of use of retrospective sample).
Mean Serum Concentration of Sodium	137.3 millimoles/L Standard Deviation 7.4	139.5 millimoles/L Standard Deviation 4.1

PRIMARY outcome

Timeframe: From Day 1 up to 39.5 months

Population: The malignancy set included all patients who were diagnosed with malignancy. Only patients with data available are reported.

Blood samples was collected at various times after HLH diagnosis to determine the concentration of beta2-microglobulin.

Outcome measures

Measure	HLH Patients n=10 Participants All patients diagnosed with HLH in the context of a malignancy.	Non-HLH Patients n=1 Participants All patients with a diagnosis of hematological malignancy and never diagnosed with HLH or no suspicion of HLH (at any time, including time after sample collection in case of use of retrospective sample).
Mean Serum Concentration of Beta2-Microglobulin	9.48 mg/L Standard Deviation 15.19	3.10 mg/L Standard Deviation NA Standard deviation is not applicable for N=1.

PRIMARY outcome

Timeframe: From Day 1 up to 39.5 months

Population: The malignancy set included all patients who were diagnosed with malignancy. Only patients with data available are reported.

Blood samples was collected at various times after HLH diagnosis to determine the concentration of IgG.

Outcome measures

Measure	HLH Patients n=15 Participants All patients diagnosed with HLH in the context of a malignancy.	Non-HLH Patients n=2 Participants All patients with a diagnosis of hematological malignancy and never diagnosed with HLH or no suspicion of HLH (at any time, including time after sample collection in case of use of retrospective sample).
Mean Serum Concentration of Immunoglobulin G (IgG)	602.2 mg/dL Standard Deviation 250.6	722.0 mg/dL Standard Deviation 353.6

PRIMARY outcome

Timeframe: From Day 1 up to 39.5 months

Population: The malignancy set included all patients who were diagnosed with malignancy. Only patients with data available are reported.

Blood samples was collected at various times after HLH diagnosis to determine the concentration of c-reactive protein.

Outcome measures

Measure	HLH Patients n=13 Participants All patients diagnosed with HLH in the context of a malignancy.	Non-HLH Patients All patients with a diagnosis of hematological malignancy and never diagnosed with HLH or no suspicion of HLH (at any time, including time after sample collection in case of use of retrospective sample).
Mean Serum Concentration of C-Reactive Protein	102.508 mg/dL Standard Deviation 174.824	—

PRIMARY outcome

Timeframe: From Day 1 up to 39.5 months

Population: The malignancy set included all patients who were diagnosed with malignancy. Only patients with data available are reported.

Blood samples was collected at various times after HLH diagnosis to determine the concentration of aspartate aminotransferase.

Outcome measures

Measure	HLH Patients n=17 Participants All patients diagnosed with HLH in the context of a malignancy.	Non-HLH Patients n=4 Participants All patients with a diagnosis of hematological malignancy and never diagnosed with HLH or no suspicion of HLH (at any time, including time after sample collection in case of use of retrospective sample).
Mean Serum Concentration of Aspartate Aminotransferase	177.8 international units/L (IU/L) Standard Deviation 279.9	44.8 international units/L (IU/L) Standard Deviation 18.4

PRIMARY outcome

Timeframe: From Day 1 up to 39.5 months

Population: The malignancy set included all patients who were diagnosed with malignancy. Only patients with data available are reported.

Blood samples was collected at various times after HLH diagnosis to determine the concentration of alanine aminotransferase.

Outcome measures

Measure	HLH Patients n=18 Participants All patients diagnosed with HLH in the context of a malignancy.	Non-HLH Patients n=5 Participants All patients with a diagnosis of hematological malignancy and never diagnosed with HLH or no suspicion of HLH (at any time, including time after sample collection in case of use of retrospective sample).
Mean Serum Concentration of Alanine Aminotransferase	127.9 IU/L Standard Deviation 179.0	34.0 IU/L Standard Deviation 25.4

PRIMARY outcome

Timeframe: From Day 1 up to 39.5 months

Population: The malignancy set included all patients who were diagnosed with malignancy. Only patients with data available are reported.

Blood samples was collected at various times after HLH diagnosis to determine the concentration of alkaline phosphatase.

Outcome measures

Measure	HLH Patients n=18 Participants All patients diagnosed with HLH in the context of a malignancy.	Non-HLH Patients n=5 Participants All patients with a diagnosis of hematological malignancy and never diagnosed with HLH or no suspicion of HLH (at any time, including time after sample collection in case of use of retrospective sample).
Mean Serum Concentration of Alkaline Phosphatase	216.0 IU/L Standard Deviation 170.5	95.0 IU/L Standard Deviation 74.5

PRIMARY outcome

Timeframe: From Day 1 up to 39.5 months

Population: The malignancy set included all patients who were diagnosed with malignancy. Only patients with data available are reported.

Blood samples was collected at various times after HLH diagnosis to determine the concentration of lactate dehydrogenase.

Outcome measures

Measure	HLH Patients n=18 Participants All patients diagnosed with HLH in the context of a malignancy.	Non-HLH Patients n=5 Participants All patients with a diagnosis of hematological malignancy and never diagnosed with HLH or no suspicion of HLH (at any time, including time after sample collection in case of use of retrospective sample).
Mean Serum Concentration of Lactate Dehydrogenase	7469.5 IU/L Standard Deviation 10456.7	1100.8 IU/L Standard Deviation 718.5

PRIMARY outcome

Timeframe: From Day 1 up to 39.5 months

Population: The malignancy set included all patients who were diagnosed with malignancy. Only patients with data available are reported.

Blood samples was collected at various times after HLH diagnosis to determine the concentration of total bilirubin.

Outcome measures

Measure	HLH Patients n=18 Participants All patients diagnosed with HLH in the context of a malignancy.	Non-HLH Patients n=5 Participants All patients with a diagnosis of hematological malignancy and never diagnosed with HLH or no suspicion of HLH (at any time, including time after sample collection in case of use of retrospective sample).
Mean Serum Concentration of Total Bilirubin	2.64 mg/dL Standard Deviation 3.85	1.68 mg/dL Standard Deviation 1.40

PRIMARY outcome

Timeframe: From Day 1 up to 39.5 months

Population: The malignancy set included all patients who were diagnosed with malignancy. Only patients with data available are reported.

Blood samples was collected at various times after HLH diagnosis to determine the concentration of conjugated bilirubin.

Outcome measures

Measure	HLH Patients n=15 Participants All patients diagnosed with HLH in the context of a malignancy.	Non-HLH Patients n=1 Participants All patients with a diagnosis of hematological malignancy and never diagnosed with HLH or no suspicion of HLH (at any time, including time after sample collection in case of use of retrospective sample).
Mean Serum Concentration of Conjugated Bilirubin	1.96 mg/dL Standard Deviation 3.71	0.00 mg/dL Standard Deviation NA Standard deviation is not applicable for N=1.

PRIMARY outcome

Timeframe: From Day 1 up to 39.5 months

Population: The malignancy set included all patients who were diagnosed with malignancy. Only patients with data available are reported.

Blood samples was collected at various times after HLH diagnosis to determine the concentration of NK cell activity. A lytic unit 30 (LU30) was defined as the number of effector cells necessary to cause lysis of 30% of its target cells.

Outcome measures

Measure	HLH Patients n=2 Participants All patients diagnosed with HLH in the context of a malignancy.	Non-HLH Patients All patients with a diagnosis of hematological malignancy and never diagnosed with HLH or no suspicion of HLH (at any time, including time after sample collection in case of use of retrospective sample).
Mean Serum Concentration of Natural Killer (NK) Cell Activity	3.5 LU30 Standard Deviation 0.7	—

Adverse Events

HLH Patients

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Non-HLH Patients

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Adverse event data not reported

Additional Information

Medical Director

Swedish Orphan Biovitrum

Phone: +4686972000

Email: clinical@sobi.com

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place