Treatment for Hemophagocytic Syndromes

Phase-Based Estimates
1
Effectiveness
1
Safety
Masonic Cancer Center, University of Minnesota, Minneapolis, MN
Hemophagocytic Syndromes+24 More
Eligibility
< 65
All Sexes
Eligible conditions
Hemophagocytic Syndromes

Study Summary

This study is evaluating whether a guideline for the treatment of patients with primary immune deficiencies is effective.

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Eligible Conditions

  • Hemophagocytic Syndromes
  • Lymphohistiocytosis, Hemophagocytic
  • Chediak-Higashi Syndrome
  • Pulmonary Histiocytosis X
  • Wiskott-Aldrich Syndrome
  • Hyper-IgM Immunodeficiency Syndrome, Type 1
  • Primary Antibody Deficiencies
  • Omenn Syndrome
  • Antibody Deficiency Syndrome
  • Hypogammaglobulinemia, Acquired
  • Histiocytosis
  • Syndrome
  • Lymphoproliferative Disorders
  • Hyper-IgM Immunodeficiency Syndrome
  • Granulomatous Disease, Chronic
  • Histiocytosis, Langerhans-Cell
  • Langerhans Cell Histiocytosis (LCH)
  • Primary Immunodeficiency Diseases
  • Severe Combined Immunodeficiency
  • Immunologic Deficiency Syndromes
  • Common Variable Immunodeficiency
  • Reticular Dysgenesis
  • Hemophagocytic Lymphohistiocytosis
  • SCID
  • Omenn's Syndrome
  • X-linked Lymphoproliferative Disease
  • Bare Lymphocyte Syndrome
  • CD40 Ligand Deficiency
  • Chronic Granulomatous Disease (CGD)
  • Hyper-IgM Syndrome
  • Griscelli Syndrome

Treatment Effectiveness

Effectiveness Estimate

1 of 3

Study Objectives

This trial is evaluating whether Treatment will improve 1 primary outcome and 7 secondary outcomes in patients with Hemophagocytic Syndromes. Measurement will happen over the course of Day 42.

6 Months
Disease-Free Survival
Incidence of Transplant-Related Mortality
Overall Survival
Year 1
Incidence of Chronic Graft-Versus-Host Disease
Day 100
Incidence of Acute Graft-Versus-Host Disease
Incidence of Graft Failure
Year 1
Incidence of Chimerism
Day 42
Neutrophil Engraftment

Trial Safety

Trial Design

1 Treatment Groups

Control

This trial requires 30 total participants across 1 different treatment groups

This trial involves a single treatment. Treatment is the primary treatment being studied. Participants will all receive the same treatment. There is no placebo group. The treatments being tested are not being studied for commercial purposes.

ControlNo treatment in the control group

Trial Logistics

Trial Timeline

Approximate Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: 6 months and 1 year
This trial has the following approximate timeline: 3 weeks for initial screening, variable treatment timelines, and roughly 6 months and 1 year for reporting.

Closest Location

Masonic Cancer Center, University of Minnesota - Minneapolis, MN

Eligibility Criteria

This trial is for patients born any sex aged 65 and younger. There are 10 eligibility criteria to participate in this trial as listed below.

Mark “yes” if the following statements are true for you:
is an autosomal recessive immunodeficiency disorder caused by mutations in the genes encoding MHC class II proteins show original
A patient who had a second bone marrow transplant for SCID (severe combined immunodeficiency) after graft rejection has been reported to have good long-term outcome show original
Reticular dysgenesis
Wiskott-Aldrich syndrome
Hyper IgM Syndrome is a rare disorder that occurs when the body does not produce enough of a protein called CD40 ligand show original
Common variable immunodeficiency (CVID) with severe phenotype
Chronic Granulomatous Disease (CGD)
is a rare and potentially fatal disorder, caused by a genetic defect that affects the immune system show original
Other severe Combined Immune Deficiencies (CID)
Omenn's Syndrome

Patient Q&A Section

Please Note: These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

What are common treatments for hemophagocytic syndromes?

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Some patients may be cured with corticosteroid therapy, but immunosuppression is needed to control other complications. In those who develop hematopoietic toxicity from aHDS treatment, autologous stem cell transplantation may be effective. Most patients with aHDS requiring treatment do not respond adequately to the first-line therapy, and subsequent treatment options may be immunosuppression, splenectomy, or autologous or allogeneic stem cell transplantation alone or in combination.

Unverified Answer

How many people get hemophagocytic syndromes a year in the United States?

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About 15,000 people per year are diagnosed with one or more forms of HLH. This accounts for less than 1% of all hospitalized adult patients in the United States. There are three distinct subtypes of familial hemophagocytic syndromes. Only about 1,000 to 3,000 people per year are diagnosed with HLH of the familial chimaeric form. No cases of HLH attributed to EBV-HLH are seen annually in the United States. The annual number of people with HLH associated with HIV remains uncertain. HLH is uncommon in patients with HIV and, when noticed, rarely causes serious symptoms.

Unverified Answer

What is hemophagocytic syndromes?

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Hemophagocytic syndromes occur mainly in adults but mainly affects children, sometimes resulting in death of the child in the absence of anticoagulants. Hemophagocytic syndromes can occur as a secondary manifestation of autoimmune diseases, such as rheumatic diseases, but can also be the main pathology of some diseases, such as juvenile idiopathic arthritis, and leukemia. It is important to recognize and manage hemophagocytic syndromes in order to prevent severe and even lethal complications.

Unverified Answer

What are the signs of hemophagocytic syndromes?

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In this patient population, the major neurologic manifestations were nonspecific. Seizures were found in approximately 10% of patients. The only radiologically evident abnormality was the usual increased leukocyte number on routine chest x-rays, although this finding is not specific to hemophagocytic syndromes.

Unverified Answer

Can hemophagocytic syndromes be cured?

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Hemophagocytic syndromes can not be cured. However, with the advancement of immunosuppressive, antiviral, and antibiotic drug therapies, patients with hemophagocytic syndromes can live happily and maintain their quality of life.

Unverified Answer

What causes hemophagocytic syndromes?

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Hemophagocytic syndromes occur after one of the following events: a viral infection (either prior to or during the acute episodes), or an immune-mediated disease (either prior to or during the acute episodes), or a malignancy. This condition is frequently associated with lymphoproliferative disturbances (including immunoblastic lymphoma).

Unverified Answer

What is the average age someone gets hemophagocytic syndromes?

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The incidence of Haemophagocytic syndrome is lower than previously thought but more often involves children. It has been found that the average age at the time of diagnosis is higher. This may highlight the importance of early diagnosis and diagnosis of more people with this condition. There is ongoing research to improve diagnosis and treatment of the condition, to decrease the complications. There is potential for more treatments that involve hemophagocytic syndromes as well as the use of the prothrombin complex concentrate, (a-Factor XIII concentrate), intravenous immunoglobulin, and other immunomodulatory therapies when necessary.

Unverified Answer

Is treatment typically used in combination with any other treatments?

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The current literature suggests that immunosuppression, chemotherapy, and radiotherapy is associated with a worsening of symptoms for patients with hemo-related disorders. It appears that therapies typically used as single therapies are less likely to be used in combination, though this may be the case for other patients and disorders.

Unverified Answer

Have there been other clinical trials involving treatment?

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There have been several clinical trials involving treatment for patients with this disease and several patients with hemophagocytic syndrome (a disease similar to hemophagocytic lymphohistiocytosis/HLH) have died within a short time after being treated with various treatments, but it has not been determined if treatment is the cause of the deaths. Patients with hemophagocytic syndromes seem to benefit most from treatment with the agents used in these clinical trials. [Ampheresis, etoposide, and other chemotherapy agents have been used in a few clinical trials for hemophagocytic syndromes when conventional or biologic immunosuppression was inadequate.] [Power](http://www.

Unverified Answer

Has treatment proven to be more effective than a placebo?

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It was evident that both treatment and control subjects experienced benefits. Further, these benefits of control and treatment were not significantly different. Results from a recent clinical trial is the first to show that a patient-controlled, medication-overutilization, regimen-based regimen is as effective as a placebo in the treatment of the disorder. We propose that further studies of treatment of this disease need to be a patient-controlled, medication-overutilization, regimen-based regimen.

Unverified Answer

How does treatment work?

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This first randomized study to assess the efficacy of a leukoreduction filter system in a randomized, controlled trial of patients with aPLDs indicates that rituximab and eculizumab both improve outcome measures. However, while these new drugs treat patients more effectively than the traditional therapies used in the past, patients still experience side effects. More research is needed to assess which medications are most effective for treating thrombotic complications.

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Is treatment safe for people?

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In the United Kingdom, the number of deaths have been decreasing since 1989. Although we found no evidence in our meta-analysis that the use of an anticoagulation for the prevention of VTE was associated with an impact on mortality, there is still concern about the risk of bleeding. On this basis, it is considered dangerous to use an anticoagulation in these patients.

Unverified Answer
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