Omalizumab prevents anaphylaxis and improves symptoms in systemic mastocytosis: Efficacy and safety observations

S Broesby-Olsen, H Vestergaard, C G Mortz, B Jensen, T Havelund, A P Hermann, F Siebenhaar, M B Møller, T K Kristensen, C Bindslev-Jensen, Mastocytosis Centre Odense University Hospital (MastOUH)

Research output: Contribution to journalJournal articleResearchpeer-review

Abstract

BACKGROUND: Patients with systemic mastocytosis (SM) may suffer from mast cell (MC) mediator-related symptoms insufficiently controlled by conventional therapy. Omalizumab is an established treatment in other MC-driven diseases, but experiences in SM are limited.

OBJECTIVE: To assess the efficacy and safety of omalizumab in SM.

METHODS: In our patient cohort, we evaluated all SM patients treated with omalizumab. A physician global assessment of type and severity of symptoms was performed at baseline, at 3 and 6 months and at latest follow-up. Quality of life was assessed by visual analogue scale. S-tryptase and KIT D816V allele burden were monitored.

RESULTS: A total of 14 adult SM patients (10 ISM, 2 BMM, 1 SSM, and 1 ASM-AHN) received omalizumab with a median duration of 17 months (range: 1-73 months). One patient was excluded due to concomitant cytoreductive therapy. In the remaining 13 patients, we observed a significant reduction in symptoms, with complete symptom control in five (38.5%), major response in three (23.1%), and a partial response in three (23.1%) patients, whereas two patients (15.4%) withdrew due to subjective side-effects at first dose. The treatment was most effective for recurrent anaphylaxis and skin symptoms, less for gastrointestinal, musculoskeletal, and neuropsychiatric symptoms. Patient-reported quality of life showed significant improvement. No significant changes in s-tryptase/KIT D816V allele burden were observed. No severe adverse events were recorded.

CONCLUSIONS: Omalizumab appears to be a promising treatment option in SM, effectively preventing anaphylaxis and improving chronic MC mediator-related symptoms, insufficiently controlled by conventional therapy. Controlled studies are needed to substantiate findings.

Original languageEnglish
JournalAllergy: European Journal of Allergy and Clinical Immunology
Volume73
Issue number1
Pages (from-to)230–238
ISSN0105-4538
DOIs
Publication statusPublished - Jan 2018

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Systemic Mastocytosis
Safety
Tryptases
Mast Cells
Alleles
Quality of Life
Mastocytosis
Visual Analog Scale
Physicians

Keywords

  • Journal Article
  • KIT D816V
  • mastocytosis
  • anaphylaxis
  • omalizumab
  • mast cell disorders
  • systemic mastocytosis
  • tryptase
  • Anaphylaxis/etiology
  • Humans
  • Middle Aged
  • Symptom Assessment
  • Skin/pathology
  • Male
  • Treatment Outcome
  • Anti-Allergic Agents/administration & dosage
  • Omalizumab/administration & dosage
  • Young Adult
  • Biomarkers
  • Quality of Life
  • Adult
  • Female
  • Mastocytosis, Systemic/diagnosis

Cite this

@article{584092a25d614e8a98ca5fe7cf237110,
title = "Omalizumab prevents anaphylaxis and improves symptoms in systemic mastocytosis: Efficacy and safety observations",
abstract = "BACKGROUND: Patients with systemic mastocytosis (SM) may suffer from mast cell (MC) mediator-related symptoms insufficiently controlled by conventional therapy. Omalizumab is an established treatment in other MC-driven diseases, but experiences in SM are limited.OBJECTIVE: To assess the efficacy and safety of omalizumab in SM.METHODS: In our patient cohort, we evaluated all SM patients treated with omalizumab. A physician global assessment of type and severity of symptoms was performed at baseline, at 3 and 6 months and at latest follow-up. Quality of life was assessed by visual analogue scale. S-tryptase and KIT D816V allele burden were monitored.RESULTS: A total of 14 adult SM patients (10 ISM, 2 BMM, 1 SSM, and 1 ASM-AHN) received omalizumab with a median duration of 17 months (range: 1-73 months). One patient was excluded due to concomitant cytoreductive therapy. In the remaining 13 patients, we observed a significant reduction in symptoms, with complete symptom control in five (38.5{\%}), major response in three (23.1{\%}), and a partial response in three (23.1{\%}) patients, whereas two patients (15.4{\%}) withdrew due to subjective side-effects at first dose. The treatment was most effective for recurrent anaphylaxis and skin symptoms, less for gastrointestinal, musculoskeletal, and neuropsychiatric symptoms. Patient-reported quality of life showed significant improvement. No significant changes in s-tryptase/KIT D816V allele burden were observed. No severe adverse events were recorded.CONCLUSIONS: Omalizumab appears to be a promising treatment option in SM, effectively preventing anaphylaxis and improving chronic MC mediator-related symptoms, insufficiently controlled by conventional therapy. Controlled studies are needed to substantiate findings.",
keywords = "Journal Article, KIT D816V, mastocytosis, anaphylaxis, omalizumab, mast cell disorders, systemic mastocytosis, tryptase, Anaphylaxis/etiology, Humans, Middle Aged, Symptom Assessment, Skin/pathology, Male, Treatment Outcome, Anti-Allergic Agents/administration & dosage, Omalizumab/administration & dosage, Young Adult, Biomarkers, Quality of Life, Adult, Female, Mastocytosis, Systemic/diagnosis",
author = "S Broesby-Olsen and H Vestergaard and Mortz, {C G} and B Jensen and T Havelund and Hermann, {A P} and F Siebenhaar and M{\o}ller, {M B} and Kristensen, {T K} and C Bindslev-Jensen and {Mastocytosis Centre Odense University Hospital (MastOUH)}",
note = "{\circledC} 2017 EAACI and John Wiley and Sons A/S. Published by John Wiley and Sons Ltd.",
year = "2018",
month = "1",
doi = "10.1111/all.13237",
language = "English",
volume = "73",
pages = "230–238",
journal = "Allergy: European Journal of Allergy and Clinical Immunology",
issn = "0105-4538",
publisher = "Wiley Online",
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Omalizumab prevents anaphylaxis and improves symptoms in systemic mastocytosis : Efficacy and safety observations. / Broesby-Olsen, S; Vestergaard, H; Mortz, C G; Jensen, B; Havelund, T; Hermann, A P; Siebenhaar, F; Møller, M B; Kristensen, T K; Bindslev-Jensen, C; Mastocytosis Centre Odense University Hospital (MastOUH).

In: Allergy: European Journal of Allergy and Clinical Immunology, Vol. 73, No. 1, 01.2018, p. 230–238.

Research output: Contribution to journalJournal articleResearchpeer-review

TY - JOUR

T1 - Omalizumab prevents anaphylaxis and improves symptoms in systemic mastocytosis

T2 - Efficacy and safety observations

AU - Broesby-Olsen, S

AU - Vestergaard, H

AU - Mortz, C G

AU - Jensen, B

AU - Havelund, T

AU - Hermann, A P

AU - Siebenhaar, F

AU - Møller, M B

AU - Kristensen, T K

AU - Bindslev-Jensen, C

AU - Mastocytosis Centre Odense University Hospital (MastOUH)

N1 - © 2017 EAACI and John Wiley and Sons A/S. Published by John Wiley and Sons Ltd.

PY - 2018/1

Y1 - 2018/1

N2 - BACKGROUND: Patients with systemic mastocytosis (SM) may suffer from mast cell (MC) mediator-related symptoms insufficiently controlled by conventional therapy. Omalizumab is an established treatment in other MC-driven diseases, but experiences in SM are limited.OBJECTIVE: To assess the efficacy and safety of omalizumab in SM.METHODS: In our patient cohort, we evaluated all SM patients treated with omalizumab. A physician global assessment of type and severity of symptoms was performed at baseline, at 3 and 6 months and at latest follow-up. Quality of life was assessed by visual analogue scale. S-tryptase and KIT D816V allele burden were monitored.RESULTS: A total of 14 adult SM patients (10 ISM, 2 BMM, 1 SSM, and 1 ASM-AHN) received omalizumab with a median duration of 17 months (range: 1-73 months). One patient was excluded due to concomitant cytoreductive therapy. In the remaining 13 patients, we observed a significant reduction in symptoms, with complete symptom control in five (38.5%), major response in three (23.1%), and a partial response in three (23.1%) patients, whereas two patients (15.4%) withdrew due to subjective side-effects at first dose. The treatment was most effective for recurrent anaphylaxis and skin symptoms, less for gastrointestinal, musculoskeletal, and neuropsychiatric symptoms. Patient-reported quality of life showed significant improvement. No significant changes in s-tryptase/KIT D816V allele burden were observed. No severe adverse events were recorded.CONCLUSIONS: Omalizumab appears to be a promising treatment option in SM, effectively preventing anaphylaxis and improving chronic MC mediator-related symptoms, insufficiently controlled by conventional therapy. Controlled studies are needed to substantiate findings.

AB - BACKGROUND: Patients with systemic mastocytosis (SM) may suffer from mast cell (MC) mediator-related symptoms insufficiently controlled by conventional therapy. Omalizumab is an established treatment in other MC-driven diseases, but experiences in SM are limited.OBJECTIVE: To assess the efficacy and safety of omalizumab in SM.METHODS: In our patient cohort, we evaluated all SM patients treated with omalizumab. A physician global assessment of type and severity of symptoms was performed at baseline, at 3 and 6 months and at latest follow-up. Quality of life was assessed by visual analogue scale. S-tryptase and KIT D816V allele burden were monitored.RESULTS: A total of 14 adult SM patients (10 ISM, 2 BMM, 1 SSM, and 1 ASM-AHN) received omalizumab with a median duration of 17 months (range: 1-73 months). One patient was excluded due to concomitant cytoreductive therapy. In the remaining 13 patients, we observed a significant reduction in symptoms, with complete symptom control in five (38.5%), major response in three (23.1%), and a partial response in three (23.1%) patients, whereas two patients (15.4%) withdrew due to subjective side-effects at first dose. The treatment was most effective for recurrent anaphylaxis and skin symptoms, less for gastrointestinal, musculoskeletal, and neuropsychiatric symptoms. Patient-reported quality of life showed significant improvement. No significant changes in s-tryptase/KIT D816V allele burden were observed. No severe adverse events were recorded.CONCLUSIONS: Omalizumab appears to be a promising treatment option in SM, effectively preventing anaphylaxis and improving chronic MC mediator-related symptoms, insufficiently controlled by conventional therapy. Controlled studies are needed to substantiate findings.

KW - Journal Article

KW - KIT D816V

KW - mastocytosis

KW - anaphylaxis

KW - omalizumab

KW - mast cell disorders

KW - systemic mastocytosis

KW - tryptase

KW - Anaphylaxis/etiology

KW - Humans

KW - Middle Aged

KW - Symptom Assessment

KW - Skin/pathology

KW - Male

KW - Treatment Outcome

KW - Anti-Allergic Agents/administration & dosage

KW - Omalizumab/administration & dosage

KW - Young Adult

KW - Biomarkers

KW - Quality of Life

KW - Adult

KW - Female

KW - Mastocytosis, Systemic/diagnosis

U2 - 10.1111/all.13237

DO - 10.1111/all.13237

M3 - Journal article

C2 - 28662309

VL - 73

SP - 230

EP - 238

JO - Allergy: European Journal of Allergy and Clinical Immunology

JF - Allergy: European Journal of Allergy and Clinical Immunology

SN - 0105-4538

IS - 1

ER -