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Research article
Ruxolitinib Might Be an Effective Hormone Reduction and Replacement Drug in Children With Secondary Hemophagocytic Lymphohistiocytosis
Jianguo Li
Children’s Hospital Affiliated to the Capital Institute of Pediatrics, Beijing 100020, China.
Corresponding Author
ORCiD: https://orcid.org/0000-0001-9431-2950
License:
This work is licensed under a CC BY 4.0 License. Read Full License
Background: To analyze the effects of ruxolitinib on children with secondary hemophagocytic lymphohistiocytosis (HLH).
Methods: Eleven pediatric patients diagnosed with HLH and treated with ruxolitinib (ruxolitinib group: group R) between November 2017 and August 2018 were retrospectively analyzed. Eleven age-matched pediatric patients with HLH undergoing conventional treatment (control group: group C) were also analyzed.
Results: In group R, three patients who did not respond to conventional treatment (dexamethasone and etoposide) were treated with Ruxolitinib and their temperature decreased to normal levels. Four patients had normal temperature after conventional treatment, but they had severe organ involvement, including obvious yellowing of the skin, increased liver enzyme levels and neuropsychiatric symptoms, and they were all ameliorated with ruxolitinib treatment. Four patients were relieved with ruxolitinib therapy alone. In group C, the body temperature of eleven patients all decreased to normal levels after conventional treatment. The body temperature of group R patients decreased to normal levels more rapidly than that of group C patients. The hormone dosage in group R was significantly lower than that in group C. Both groups were followed up for 2–2.5 years. No obvious adverse drug reactions of ruxolitinib were observed during treatment and follow-up.
Conclusion: Ruxolitinib might be an effective drug in controlling body temperature and reducing inflammation indicators. It might be a potential replacement for hormone therapy for HLH treatment in children, thereby reducing or avoiding hormone-related adverse reactions.
Keywords
Ruxolitinib, Hemophagocytic lymphohistiocytosis, Janus kinase inhibitor, Children
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On 13 Nov, 2020
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Received 11 Oct, 2020
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Received 25 Sep, 2020
Reviewer #4 agreed
On 25 Sep, 2020
Reviewer #3 agreed
On 22 Sep, 2020
Reviewer #1 agreed
On 21 Sep, 2020
Reviewer #2 agreed
On 21 Sep, 2020
Reviewers invited
Invitations sent on 19 Sep, 2020
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On 18 Sep, 2020
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This preprint is under consideration at Pediatric Rheumatology. A preprint is a preliminary version of a manuscript that has not completed peer review at a journal. Research Square does not conduct peer review prior to posting preprints. The posting of a preprint on this server should not be interpreted as an endorsement of its validity or suitability for dissemination as established information or for guiding clinical practice.
Learn more about In Review
Research article
Ruxolitinib Might Be an Effective Hormone Reduction and Replacement Drug in Children With Secondary Hemophagocytic Lymphohistiocytosis
Jianguo Li
Children’s Hospital Affiliated to the Capital Institute of Pediatrics, Beijing 100020, China.
Corresponding Author
ORCiD: https://orcid.org/0000-0001-9431-2950
Badges
Prescreen
This manuscript passed our Prescreen assessment
Complete author information
Appropriate declaration statements
Potential risks to human health
Prescreen
Peer Review Timeline
CURRENT STATUS: Under Review
Version 1
Posted 22 Sep, 2020
No community comments so far
Editorial decision: Major revision
On 13 Nov, 2020
Review #4 received
Received 20 Oct, 2020
Review #3 received
Received 11 Oct, 2020
Review #1 received
Received 25 Sep, 2020
Review #2 received
Received 25 Sep, 2020
Reviewer #4 agreed
On 25 Sep, 2020
Reviewer #3 agreed
On 22 Sep, 2020
Reviewer #1 agreed
On 21 Sep, 2020
Reviewer #2 agreed
On 21 Sep, 2020
Reviewers invited
Invitations sent on 19 Sep, 2020
First submitted
On 18 Sep, 2020
Editor assigned
On 18 Sep, 2020
Submission checks complete
On 17 Sep, 2020
Editor invited
On 17 Sep, 2020
Metrics
Comments: 0
PDF Downloads: ...
HTML Views: ...
License:
This work is licensed under a CC BY 4.0 License. Read Full License
Background: To analyze the effects of ruxolitinib on children with secondary hemophagocytic lymphohistiocytosis (HLH).
Methods: Eleven pediatric patients diagnosed with HLH and treated with ruxolitinib (ruxolitinib group: group R) between November 2017 and August 2018 were retrospectively analyzed. Eleven age-matched pediatric patients with HLH undergoing conventional treatment (control group: group C) were also analyzed.
Results: In group R, three patients who did not respond to conventional treatment (dexamethasone and etoposide) were treated with Ruxolitinib and their temperature decreased to normal levels. Four patients had normal temperature after conventional treatment, but they had severe organ involvement, including obvious yellowing of the skin, increased liver enzyme levels and neuropsychiatric symptoms, and they were all ameliorated with ruxolitinib treatment. Four patients were relieved with ruxolitinib therapy alone. In group C, the body temperature of eleven patients all decreased to normal levels after conventional treatment. The body temperature of group R patients decreased to normal levels more rapidly than that of group C patients. The hormone dosage in group R was significantly lower than that in group C. Both groups were followed up for 2–2.5 years. No obvious adverse drug reactions of ruxolitinib were observed during treatment and follow-up.
Conclusion: Ruxolitinib might be an effective drug in controlling body temperature and reducing inflammation indicators. It might be a potential replacement for hormone therapy for HLH treatment in children, thereby reducing or avoiding hormone-related adverse reactions.
Figure 1
Figure 2
Figure 3
Figure 4