Optimising Outcomes in Indolent Systemic Mastocytosis: A Comprehensive Review of Best Practices Across the Patient Journey
PeerVoice
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Sep 19, 2024
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About This Presentation
Frank Siebenhaar, PD, and Vito Sabato, MD, PhD, discuss indolent systemic mastocytosis in this CE activity titled "Optimising Outcomes in Indolent Systemic Mastocytosis: A Comprehensive Review of Best Practices Across the Patient Journey." For the full presentation, please visit us at www....
Slide Content
PeerVoice
Optimising Outcomes in Indolent Systemic Mastocytosis:
A Comprehensive Review of Best Practices Across the Patient Journey
Learning Objectives
* Differentiate the symptoms, causes and outcomes associated with childhood-
onset and adult-onset presentations of mastocytosis
Utilise recommended clinical tools for the evaluation of symptoms and quality of
life (QoL) in patients with indolent systemic mastocytosis (ISM)
Implement evidence-based best practices in the diagnosis, monitoring and
management of ISM
PeerVoice is an EBAC® accredited provider since 2022.
Copyright © 2010-2024, PeerVoice
Optimising Outcomes in Indolent Systemic Mastocytosis:
A Comprehensive Review of Best Practices Across the Patient Journey
Learning Objectives
* Differentiate the symptoms, causes and outcomes associated with childhood-
onset and adult-onset presentations of mastocytosis
Utilise recommended clinical tools for the evaluation of symptoms and quality of
life (QoL) in patients with indolent systemic mastocytosis (ISM)
Implement evidence-based best practices in the diagnosis, monitoring and
management of ISM
PeerVoice is an EBAC® accredited provider since 2022.
Copyright © 2010-2024, PeerVoice
PeerVoice
Part 1 of 3: Recognising and Diagnosing Indolent Systemic Mastocytosis:
Common Misconceptions and Best Practices in Initial Testing
Frank Siebenhaar, PD Vito Sabato, MD, PhD
Doctor of Medicine and Head of Outpatient Clinics Professor of Allergy and Clinical Immunology
Charité Institute of Allergology University of Antwerp
Berlin, Germany Senior Staff Member
‘Antwerp University Hospital
Antwerp, Belgium
Copyright © 2010-2024, PeerVoice
Part 1 of 3: Recognising and Diagnosing Indolent Systemic Mastocytosis:
Common Misconceptions and Best Practices in Initial Testing
Frank Siebenhaar, PD Vito Sabato, MD, PhD
Doctor of Medicine and Head of Outpatient Clinics Professor of Allergy and Clinical Immunology
Charité Institute of Allergology University of Antwerp
Berlin, Germany Senior Staff Member
‘Antwerp University Hospital
Antwerp, Belgium
Copyright © 2010-2024, PeerVoice
PeerVoice
Frank Siebenhaar, PD, has a financial interest/relationship or affiliation in the form of.
Consultant for Cogent Biosciences, Inc; Granular Therapeutics; Invea Therapeutics, Inc;
and Vintura.
Other Financial or Material Support from Blueprint Medicines Corporation; Cogent
Biosciences, Inc; Granular Therapeutics; Invea Therapeutics, Inc.; Noucor; Novartis AG;
and Sanofi.
Vito Sabato, MD, PhD, has a financial interest/relationship or affiliation in the form of:
Grant/Research Support from Blueprint Medicines Corporation and Cogent Biosciences,
Inc.
Speakers Bureau participant with Blueprint Medicines Corporation and Cogent
Biosciences, Inc.
Advisory Board for Blueprint Medicines Corporation; Cogent Biosciences, Inc; Novartis
AG; and Telios Pharma, Inc.
Speaker or participant in accredited CME/CPD for Blueprint Medicines Corporation and
Cogent Biosciences, Inc.
www.peervoice.com/NXY870 Copyright © 2010-2024, PeerVoice
Frank Siebenhaar, PD, has a financial interest/relationship or affiliation in the form of.
Consultant for Cogent Biosciences, Inc; Granular Therapeutics; Invea Therapeutics, Inc;
and Vintura.
Other Financial or Material Support from Blueprint Medicines Corporation; Cogent
Biosciences, Inc; Granular Therapeutics; Invea Therapeutics, Inc.; Noucor; Novartis AG;
and Sanofi.
Vito Sabato, MD, PhD, has a financial interest/relationship or affiliation in the form of:
Grant/Research Support from Blueprint Medicines Corporation and Cogent Biosciences,
Inc.
Speakers Bureau participant with Blueprint Medicines Corporation and Cogent
Biosciences, Inc.
Advisory Board for Blueprint Medicines Corporation; Cogent Biosciences, Inc; Novartis
AG; and Telios Pharma, Inc.
Speaker or participant in accredited CME/CPD for Blueprint Medicines Corporation and
Cogent Biosciences, Inc.
www.peervoice.com/NXY870 Copyright © 2010-2024, PeerVoice
PeerVoice
ical Case: 61-Year-Old Man Presenting With Skin Lesions
Medical History
Skin lesions for 15 years
* Pruritus: 7/10 (NRS)
+ Darier's sign: Positive
+ Noallergic reactions
+ Vertebral compression
fractures (2 years ago)
>80% of patients with ISM
present with monomorphic,
maculopapular skin lesions
Images used with patient permission; Courtesy of Frank Siebenhaar, PD.
Copyright © 2010-2024, PeerVoice
www.peervoice.com/NXY870
ical Case: 61-Year-Old Man Presenting With Skin Lesions
Medical History
Skin lesions for 15 years
* Pruritus: 7/10 (NRS)
+ Darier's sign: Positive
+ Noallergic reactions
+ Vertebral compression
fractures (2 years ago)
>80% of patients with ISM
present with monomorphic,
maculopapular skin lesions
Images used with patient permission; Courtesy of Frank Siebenhaar, PD.
Copyright © 2010-2024, PeerVoice
www.peervoice.com/NXY870
PeerVoice
Indolent Systemic Mastocytosis: Skin Lesions
Variantand — Mostly foundin Estimated prevalence Estimated median
subvariant _(children/adults) inmastocytosiss survival, y
cm
MPCM (UP) Children 30-60% >50'
DCM Children 10% »50°
oma Chicren «x »60°
sm
Nonadvanced forms of SM
BMM Adults 10% 20”
ISM Adults 30-50% 20
ssM Adults 5% >10
Advanced forms of SM
‘SM with AHN Adults 5% 1-5
ASM Adults 5% 2-5
MCL Adults % ea“
Localised aggressive mast cell tumour
MCs Adults core 2
*Mastocytosisis a rare disease with an estimated incidence of approximately 100.000 per year
in industriised countries. Pin these groups of patients, Ihe ite expectancy is normal or near
‘rormal.‘In chronic MCL, defined by the absence of SM-induced organ damage, the survival time is Images used with patient permission;
more than 2 years unless the patient progresses to acute MCL within a short time. Courtesy of Frank Siebenhaar, PD.
www.peervoice.com/NXY870 Copyright © 2010-2024, PeerVoice
Indolent Systemic Mastocytosis: Skin Lesions
Variantand — Mostly foundin Estimated prevalence Estimated median
subvariant _(children/adults) inmastocytosiss survival, y
cm
MPCM (UP) Children 30-60% >50'
DCM Children 10% »50°
oma Chicren «x »60°
sm
Nonadvanced forms of SM
BMM Adults 10% 20”
ISM Adults 30-50% 20
ssM Adults 5% >10
Advanced forms of SM
‘SM with AHN Adults 5% 1-5
ASM Adults 5% 2-5
MCL Adults % ea“
Localised aggressive mast cell tumour
MCs Adults core 2
*Mastocytosisis a rare disease with an estimated incidence of approximately 100.000 per year
in industriised countries. Pin these groups of patients, Ihe ite expectancy is normal or near
‘rormal.‘In chronic MCL, defined by the absence of SM-induced organ damage, the survival time is Images used with patient permission;
more than 2 years unless the patient progresses to acute MCL within a short time. Courtesy of Frank Siebenhaar, PD.
www.peervoice.com/NXY870 Copyright © 2010-2024, PeerVoice
PeerVoice
Who Amongst the Following Is a Candidate for BM Analysis?
Male: 2 years old Female: 53 years old
Tryptase 35 pg/L Tryptase 5 pg/L
Male: 3 years old
Tryptase 15 pg/L
Female: 33 years old
Tryptase 25 pg/L
Images used with patient permission; Courtesy of Vito Sabato, MD, PhD.
www.peervoice.com/NXY870 Copyright © 2010-2024, PeeVoice
Who Amongst the Following Is a Candidate for BM Analysis?
Male: 2 years old Female: 53 years old
Tryptase 35 pg/L Tryptase 5 pg/L
Male: 3 years old
Tryptase 15 pg/L
Female: 33 years old
Tryptase 25 pg/L
Images used with patient permission; Courtesy of Vito Sabato, MD, PhD.
www.peervoice.com/NXY870 Copyright © 2010-2024, PeeVoice
PeerVoice
Clinical Case: 6 Id Man Presenting Without Skin Lesions
Medical History
+ Presents with hypertension and syncope after wasp sting
» Patient has no skin lesions
+ Serum tryptase 19 pg/L
www.peervoice.com/NXY870 Copyright © 2010-2024, PoerVoice
Clinical Case: 6 Id Man Presenting Without Skin Lesions
Medical History
+ Presents with hypertension and syncope after wasp sting
» Patient has no skin lesions
+ Serum tryptase 19 pg/L
www.peervoice.com/NXY870 Copyright © 2010-2024, PoerVoice
PeerVoice
KIT D816V Mutation: A Main Driver for SM
Serum tryptase testing and screening for KIT
D816V mutation in peripheral blood are key
elements of SM diagnosis
The KIT receptor
major mutation
(KIT D816V) found in
SM
KIT mutation
- In ~95% of mastocytosis cases the KIT mutation is detectable in the
peripheral blood
» Recommended screening tests for SM are highly sensitive technologies
(PCR-based) for the detection of KIT D816V in peripheral blood leukocytes
» Analysis of KIT mutational status should include BM evaluation
www.peervoice.com/NXY870 Copyright © 2010-2024, PeerVoice
KIT D816V Mutation: A Main Driver for SM
Serum tryptase testing and screening for KIT
D816V mutation in peripheral blood are key
elements of SM diagnosis
The KIT receptor
major mutation
(KIT D816V) found in
SM
KIT mutation
- In ~95% of mastocytosis cases the KIT mutation is detectable in the
peripheral blood
» Recommended screening tests for SM are highly sensitive technologies
(PCR-based) for the detection of KIT D816V in peripheral blood leukocytes
» Analysis of KIT mutational status should include BM evaluation
www.peervoice.com/NXY870 Copyright © 2010-2024, PeerVoice
PeerVoice
Clinical Case: 61-Year-Old Man Presenting With Skin Lesions
(Cont'd)
Medical History
Skin lesions for 15 years
Pruritus: 7/10 (NRS)
Darier's sign: Positive
No allergic reactions
Vertebral compression fractures (2 years ago)
Lab Parameters
KIT D816V VAF: 4.1%
ALP: 110 IU/L (N: 20-140)
CBC: Normal
BM Aspirate and Biopsy
+ Sonography: Hepatosplenomegaly + Multifocal dense aggregates of CD25+
* Bone density scan: Osteoporosis » Spindle-shaped BM mast cells
Images used with patient permission; Courtesy of Frank Siebenhaar, PD.
www.peervoice.com/NXY870 Copyright © 2010-2024, PeerVoice
Clinical Case: 61-Year-Old Man Presenting With Skin Lesions
(Cont'd)
Medical History
Skin lesions for 15 years
Pruritus: 7/10 (NRS)
Darier's sign: Positive
No allergic reactions
Vertebral compression fractures (2 years ago)
Lab Parameters
KIT D816V VAF: 4.1%
ALP: 110 IU/L (N: 20-140)
CBC: Normal
BM Aspirate and Biopsy
+ Sonography: Hepatosplenomegaly + Multifocal dense aggregates of CD25+
* Bone density scan: Osteoporosis » Spindle-shaped BM mast cells
Images used with patient permission; Courtesy of Frank Siebenhaar, PD.
www.peervoice.com/NXY870 Copyright © 2010-2024, PeerVoice
PeerVoice
B- and C-Findings in Patients With SM
BM + Hypercellular BM with signs of myeloproliferation, BM cell
dysplasia
Live
Spleen, | + Organomegaly (lymphadenopathy or splenomegaly)
ai
Skeletal _
lesions
Borderline Benign (involvement of the neoplastic
process)
+ Involvement of additional haematopoietic cell
lineages, not only mast cells
+ Not associated with organ damage by
definition
+ BM MC (30% by histology and immunohistochemistry) + a
high serum tryptase level (5200 ng/mL)
C-Findings
Consider Cytoreduction or Chemotherapy (or
targeted drugs/SCT)
+ An advanced form of SM
+ Clinically relevant organ damage (may be
irreversible) caused by a local neoplastic MC
infiltration
+ Decreased production and number of blood cells:
- anaemia
= leukopenia
- and/or thrombocytopenia
+ Decreased production and/or loss of albumin
(hypoalbuminaemia)
+ Liver damage with ascites and increased liver enzymes
(especially ALPs)
+ Malabsorption (clinically relevant damage of the GI tract and
its ability to digest and take up nutrients)
+ Huge bone lesions resembling osteolyses (caused by MC
infiltrate) with pathologic fractures (fractures occurring after
minimal injuries or spontaneously)
Symptoms that are caused by MC mediators must not be regarded as B- or C-Findings
www.peervoice.com/NXY870
Copyright © 2010-2024, PeerVoice
B- and C-Findings in Patients With SM
BM + Hypercellular BM with signs of myeloproliferation, BM cell
dysplasia
Live
Spleen, | + Organomegaly (lymphadenopathy or splenomegaly)
ai
Skeletal _
lesions
Borderline Benign (involvement of the neoplastic
process)
+ Involvement of additional haematopoietic cell
lineages, not only mast cells
+ Not associated with organ damage by
definition
+ BM MC (30% by histology and immunohistochemistry) + a
high serum tryptase level (5200 ng/mL)
C-Findings
Consider Cytoreduction or Chemotherapy (or
targeted drugs/SCT)
+ An advanced form of SM
+ Clinically relevant organ damage (may be
irreversible) caused by a local neoplastic MC
infiltration
+ Decreased production and number of blood cells:
- anaemia
= leukopenia
- and/or thrombocytopenia
+ Decreased production and/or loss of albumin
(hypoalbuminaemia)
+ Liver damage with ascites and increased liver enzymes
(especially ALPs)
+ Malabsorption (clinically relevant damage of the GI tract and
its ability to digest and take up nutrients)
+ Huge bone lesions resembling osteolyses (caused by MC
infiltrate) with pathologic fractures (fractures occurring after
minimal injuries or spontaneously)
Symptoms that are caused by MC mediators must not be regarded as B- or C-Findings
www.peervoice.com/NXY870
Copyright © 2010-2024, PeerVoice
PeerVoice
Abbreviations and References
Clinical Case: 61-Year-Old Man Presenting With Skin Lesions
Abbreviation(s): ISM: indolent systemic mastocytosis; NRS: numeric rating scale.
Indolent Systemic Mastocytosis: Skin Lesions
Abbreviation(s): AHN: associated haematologic neoplasm; ASM: aggressive SM; BMM: bone marrow mastocytosi
CM: cutaneous mastocytosis; DCM: diffuse CM; MCL: mast cell leukaemia; MCS: mast cell sarcoma;
MPCM: maculopapular CM; SM: systemic mastocytosis; SSM: smoldering SM; UP: urticaria pigmentosa.
Reference(s): Hartmann K et al. J Allergy Clin Immunol. 2016;137:35-45.
Who Amongst the Following Is a Candidate for BM Analysis?
Abbreviation(s): BM: bone marrow.
www.peervoice.com/NXY870 Copyright © 2010-2024, PeerVoice
Abbreviations and References
Clinical Case: 61-Year-Old Man Presenting With Skin Lesions
Abbreviation(s): ISM: indolent systemic mastocytosis; NRS: numeric rating scale.
Indolent Systemic Mastocytosis: Skin Lesions
Abbreviation(s): AHN: associated haematologic neoplasm; ASM: aggressive SM; BMM: bone marrow mastocytosi
CM: cutaneous mastocytosis; DCM: diffuse CM; MCL: mast cell leukaemia; MCS: mast cell sarcoma;
MPCM: maculopapular CM; SM: systemic mastocytosis; SSM: smoldering SM; UP: urticaria pigmentosa.
Reference(s): Hartmann K et al. J Allergy Clin Immunol. 2016;137:35-45.
Who Amongst the Following Is a Candidate for BM Analysis?
Abbreviation(s): BM: bone marrow.
www.peervoice.com/NXY870 Copyright © 2010-2024, PeerVoice
PeerVoice
Abbreviations and References (Cont'd)
KIT D816V Mutation: A Main Driver for SM
Abbreviation(s): PCR: polymerase chain reaction.
Reference(s): Ustun € et al. Haematologica. 2016:101:
Bose P, Verstovesek S. Expert Rev Hematol. 2021;14:687-696.
Valent P et al. J Allergy Clin Immunol Pract. 2022;10:1999-2012.
Arock M et al. Leukemia, 2015;29:1223-1232.
Kristensen T et al. Eur J Haematol. 2016;96:381-388.
Hoermann G et al. J Allergy Clin Immunol Pract. 2022 10:1953-1963.
Hoermann G et al. Allergy. 2014;69:810-813.
Clinical Case: 61-Year-Old Man Presenting With Skin Lesions (Cont'd)
Abbreviation(s): ALP: alkaline phosphatase; CBC: complete blood count; NRS: numerical rating scale; VAF: variant allele
frequency.
B- and C-Findings in Patients With SM
Abbreviation(s): MC: mast cell; SCT: stem cell transplant.
Reference(s): Universitatsklinik für Innere Medizin |. Information on Mastocytosis. https://innere-med-
Imeduniwien.ac.at/unsere-klinischen-abteilungen/haematologie-und-haemostaseologie/projekte/ecnm-registry-
european-competence-network-mastocytosis/information-on-mastocytosis/english/. Accessed 29 August, 2024.
www.peervoice.com/NXY870 Copyright © 2010-2024, PeerVoice
Abbreviations and References (Cont'd)
KIT D816V Mutation: A Main Driver for SM
Abbreviation(s): PCR: polymerase chain reaction.
Reference(s): Ustun € et al. Haematologica. 2016:101:
Bose P, Verstovesek S. Expert Rev Hematol. 2021;14:687-696.
Valent P et al. J Allergy Clin Immunol Pract. 2022;10:1999-2012.
Arock M et al. Leukemia, 2015;29:1223-1232.
Kristensen T et al. Eur J Haematol. 2016;96:381-388.
Hoermann G et al. J Allergy Clin Immunol Pract. 2022 10:1953-1963.
Hoermann G et al. Allergy. 2014;69:810-813.
Clinical Case: 61-Year-Old Man Presenting With Skin Lesions (Cont'd)
Abbreviation(s): ALP: alkaline phosphatase; CBC: complete blood count; NRS: numerical rating scale; VAF: variant allele
frequency.
B- and C-Findings in Patients With SM
Abbreviation(s): MC: mast cell; SCT: stem cell transplant.
Reference(s): Universitatsklinik für Innere Medizin |. Information on Mastocytosis. https://innere-med-
Imeduniwien.ac.at/unsere-klinischen-abteilungen/haematologie-und-haemostaseologie/projekte/ecnm-registry-
european-competence-network-mastocytosis/information-on-mastocytosis/english/. Accessed 29 August, 2024.
www.peervoice.com/NXY870 Copyright © 2010-2024, PeerVoice
PeerVoice
Part 2 of 3: Managing Indolent Systemic Mastocytosis:
The Importance of Continuous Assessment
Frank Siebenhaar, PD Vito Sabato, MD, PhD
Doctor of Medicine and Head of Outpatient Clinics Professor of Allergy and Clinical Immunology
Charité Institute of Allergology University of Antwerp
Berlin, Germany Senior Staff Member
‘Antwerp University Hospital
Antwerp, Belgium
Copyright © 2010-2024, PeerVoice
Part 2 of 3: Managing Indolent Systemic Mastocytosis:
The Importance of Continuous Assessment
Frank Siebenhaar, PD Vito Sabato, MD, PhD
Doctor of Medicine and Head of Outpatient Clinics Professor of Allergy and Clinical Immunology
Charité Institute of Allergology University of Antwerp
Berlin, Germany Senior Staff Member
‘Antwerp University Hospital
Antwerp, Belgium
Copyright © 2010-2024, PeerVoice
PeerVoice
Frank Siebenhaar, PD, has a financial interest/relationship or affiliation in the form of.
Consultant for Cogent Biosciences, Inc; Granular Therapeutics; Invea Therapeutics, Inc;
and Vintura.
Other Financial or Material Support from Blueprint Medicines Corporation; Cogent
Biosciences, Inc; Granular Therapeutics; Invea Therapeutics, Inc.; Noucor; Novartis AG;
and Sanofi.
Vito Sabato, MD, PhD, has a financial interest/relationship or affiliation in the form of:
Grant/Research Support from Blueprint Medicines Corporation and Cogent Biosciences,
Inc.
Speakers Bureau participant with Blueprint Medicines Corporation and Cogent
Biosciences, Inc.
Advisory Board for Blueprint Medicines Corporation; Cogent Biosciences, Inc; Novartis
AG; and Telios Pharma, Inc.
Speaker or participant in accredited CME/CPD for Blueprint Medicines Corporation and
Cogent Biosciences, Inc.
www.peervoice.com/NXY870 Copyright © 2010-2024, PeerVoice
Frank Siebenhaar, PD, has a financial interest/relationship or affiliation in the form of.
Consultant for Cogent Biosciences, Inc; Granular Therapeutics; Invea Therapeutics, Inc;
and Vintura.
Other Financial or Material Support from Blueprint Medicines Corporation; Cogent
Biosciences, Inc; Granular Therapeutics; Invea Therapeutics, Inc.; Noucor; Novartis AG;
and Sanofi.
Vito Sabato, MD, PhD, has a financial interest/relationship or affiliation in the form of:
Grant/Research Support from Blueprint Medicines Corporation and Cogent Biosciences,
Inc.
Speakers Bureau participant with Blueprint Medicines Corporation and Cogent
Biosciences, Inc.
Advisory Board for Blueprint Medicines Corporation; Cogent Biosciences, Inc; Novartis
AG; and Telios Pharma, Inc.
Speaker or participant in accredited CME/CPD for Blueprint Medicines Corporation and
Cogent Biosciences, Inc.
www.peervoice.com/NXY870 Copyright © 2010-2024, PeerVoice
PeerVoice
Indolent Systemic Mastocytosis:
Symptoms
Osteoporosis
Osteopenia
Fractures
Fatigue .
Headaches +
Palpitations +
Pain
Brain fog
Weakness
Dizziness
Spectrum of Signs and
+ Rash
+ Flushing
* Itching
Nausea
Vomiting
* Diarrhoea .
+ Abdominal pain +
Copyright © 2010-2024, PeerVoice
Indolent Systemic Mastocytosis:
Symptoms
Osteoporosis
Osteopenia
Fractures
Fatigue .
Headaches +
Palpitations +
Pain
Brain fog
Weakness
Dizziness
Spectrum of Signs and
+ Rash
+ Flushing
* Itching
Nausea
Vomiting
* Diarrhoea .
+ Abdominal pain +
Copyright © 2010-2024, PeerVoice
PeerVoice
Initial Approaches to Managing Symptoms of Confirmed ISM
Drug Class or Preventive Measure Indication
Avoidance of any triggers Prophylaxis against anaphylaxis
Basis x Prophylaxis against anaphylaxis, mediator-related
eect a Basic therapy with HRI blocker + HR2 blocker ES
[Addition of a proton pump inhibitor to the HR2
blocker
Addition of MC stabiliser Refractory skin or Gl symptoms
Gl symptoms not controlled by HR2 blocker
RMS Addition of MC stabiliser, aspirin, or anti-leukotriene Ha flushing, tachycardia, hypotension, or bone
measures. ‘Addition of ultraviolet light therapy* Refractory skin symptoms.
Addition of corticosteroids Refractory allergic symptoms, anaphylaxis, MCAS.
Anti-bone mass loss supplements (Ca, Vit D,
bisphosphonates, exercise)
Insect venom immunotherapy Bee or wasp venom allergy
Multi-resistant allergic symptoms, refractory MCAS,
Additional Anti-IgE therapy confirmed IgE-dependent allergy in high-risk patients
(prophylaxis)
specific
Sao Epinephrine and other emergency drugs used in
UE Anaphylactic shock (MCAS event)
MCAS in patients with advanced SM (reducing target
cells and releasability)
®Because of the risk of secondary skin cancer, this therapy is no longer recommended for the treatment of cutaneous lesions in mastocytosis in most
Tyrosine kinase inhibitor
centers. The recommendation to use aspirin or a leukotriene receptor antagonist in patients with mastocytosis is often based on and supported by the
demonstration of an increase in the urinary prostaglandin D2 metabolite 23-dinor=11b=PGF2a and LTE4,
www.peervoice.com/NXY870 Copyright © 2010-2024, PeerVoice
Initial Approaches to Managing Symptoms of Confirmed ISM
Drug Class or Preventive Measure Indication
Avoidance of any triggers Prophylaxis against anaphylaxis
Basis x Prophylaxis against anaphylaxis, mediator-related
eect a Basic therapy with HRI blocker + HR2 blocker ES
[Addition of a proton pump inhibitor to the HR2
blocker
Addition of MC stabiliser Refractory skin or Gl symptoms
Gl symptoms not controlled by HR2 blocker
RMS Addition of MC stabiliser, aspirin, or anti-leukotriene Ha flushing, tachycardia, hypotension, or bone
measures. ‘Addition of ultraviolet light therapy* Refractory skin symptoms.
Addition of corticosteroids Refractory allergic symptoms, anaphylaxis, MCAS.
Anti-bone mass loss supplements (Ca, Vit D,
bisphosphonates, exercise)
Insect venom immunotherapy Bee or wasp venom allergy
Multi-resistant allergic symptoms, refractory MCAS,
Additional Anti-IgE therapy confirmed IgE-dependent allergy in high-risk patients
(prophylaxis)
specific
Sao Epinephrine and other emergency drugs used in
UE Anaphylactic shock (MCAS event)
MCAS in patients with advanced SM (reducing target
cells and releasability)
®Because of the risk of secondary skin cancer, this therapy is no longer recommended for the treatment of cutaneous lesions in mastocytosis in most
Tyrosine kinase inhibitor
centers. The recommendation to use aspirin or a leukotriene receptor antagonist in patients with mastocytosis is often based on and supported by the
demonstration of an increase in the urinary prostaglandin D2 metabolite 23-dinor=11b=PGF2a and LTE4,
www.peervoice.com/NXY870 Copyright © 2010-2024, PeerVoice
PeerVoice
Impact of ISM Disease on QoL
Impact on Patients’ Ability to Work
Reported reducing work hours due to SM 32
Reported having been on medical disability due to SM 14
Reported voluntarily quitting their job due to SM 15
Took early retirement 10
Reported that they do not work due to SM 12
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Impact of ISM Disease on QoL
Impact on Patients’ Ability to Work
Reported reducing work hours due to SM 32
Reported having been on medical disability due to SM 14
Reported voluntarily quitting their job due to SM 15
Took early retirement 10
Reported that they do not work due to SM 12
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PeerVoice
Assessments of ISM Disease Burden
Symptom burden ij MAS, MSAF, ISM-SAF
QoL impairment MC-QoL, MQLQ
Skin lesion assessment
BMD measurements, fractures
Anaphylaxis rating/grading
|: Biomarker assessment (tryptase,
KIT D816V VAF)
BM investigation (trephine, aspirate)
Organ size evaluation
(splenomegaly)
Copyright © 2010-2024, PeerVoice
Assessments of ISM Disease Burden
Symptom burden ij MAS, MSAF, ISM-SAF
QoL impairment MC-QoL, MQLQ
Skin lesion assessment
BMD measurements, fractures
Anaphylaxis rating/grading
|: Biomarker assessment (tryptase,
KIT D816V VAF)
BM investigation (trephine, aspirate)
Organ size evaluation
(splenomegaly)
Copyright © 2010-2024, PeerVoice
PeerVoice
Mastocytosis Quality of Life (MC-QoL
Number of items:
‘Assessment, period:
Suited for:
Evaluation:
Max points:
Max sum:
Language:
15
Mild
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27 items
Retrospective, 14 d recall
All adult (218 y) patients with cutaneous and
indolent systemic mastocytosis
- Symptoms:
- Functioning/social life
- Emotions
- Skin
Each item scores from O to 4 points
108 (points are normalised to 100 max total score)
Domain scores (0-100 each), total score (0-100)
37 adopted versions
Moderate
Is eesse
Copyright © 2010-2024, PeerVoice
Mastocytosis Quality of Life (MC-QoL
Number of items:
‘Assessment, period:
Suited for:
Evaluation:
Max points:
Max sum:
Language:
15
Mild
www.peervoice.com/NXY870
27 items
Retrospective, 14 d recall
All adult (218 y) patients with cutaneous and
indolent systemic mastocytosis
- Symptoms:
- Functioning/social life
- Emotions
- Skin
Each item scores from O to 4 points
108 (points are normalised to 100 max total score)
Domain scores (0-100 each), total score (0-100)
37 adopted versions
Moderate
Is eesse
Copyright © 2010-2024, PeerVoice
PeerVoice
Clinical Case: Assessing QoL and Disease Activity
Additional Symptoms
Medical History
+ Skin lesions for 15 years
+ Pruritus: 7/10 (NRS)
+ Darier's sign: Positive
+ No allergic reactions
* Vertebral compression
fractures (2 years ago)
Abdominal cramping
Diarrhoea
Bone pain
‘Brain fog’
Osteoporosis
ase Burden (PROMs)
» MC-QoL = 81
» MAS=58
» MCT=3
Moderate-to-severe symptom
burden and QoL impairment
Initial Treatment Plan
2nd-generation antihistamine
(4-fold dose)
H2 blocker
Disodium cromoglycate
Alendronate
Calcium + vitamin D
Images used with patient permission; Courtesy of Frank Siebenhaar, PD.
www.peervoice.com/NXY870
Copyright © 2010-2024, PeerVoice
Clinical Case: Assessing QoL and Disease Activity
Additional Symptoms
Medical History
+ Skin lesions for 15 years
+ Pruritus: 7/10 (NRS)
+ Darier's sign: Positive
+ No allergic reactions
* Vertebral compression
fractures (2 years ago)
Abdominal cramping
Diarrhoea
Bone pain
‘Brain fog’
Osteoporosis
ase Burden (PROMs)
» MC-QoL = 81
» MAS=58
» MCT=3
Moderate-to-severe symptom
burden and QoL impairment
Initial Treatment Plan
2nd-generation antihistamine
(4-fold dose)
H2 blocker
Disodium cromoglycate
Alendronate
Calcium + vitamin D
Images used with patient permission; Courtesy of Frank Siebenhaar, PD.
www.peervoice.com/NXY870
Copyright © 2010-2024, PeerVoice
PeerVoice
Abbreviations and References
Indolent Systemic Mastocytosis: Spectrum of Signs and Symptoms
Abbreviation(s): SM: systemic mastocytosis.
Reference(s): European Medical Journal. Transforming Management of Indolent Systemic Mastocytosis With Precision
Medicine. https://www.emjreviews.com/allergy-immunology/symposium/transforming-management-of-indolent-
systemic-mastocytosis-with-precision-medicine-j010124/. Published 11 July, 2024. Accessed 24 August, 2024.
Pyatilova P, Siebenhaar F. Immunol Allergy Clin North Am. 2023;43:751-762.
Siebenhaar F et al. Allergy. 2018:73:1489-1496.
Initial Approaches to Managing Symptoms of Confirmed ISM
Abbreviation(s): Ca: calcium; HR: histamine receptor; IgE: immunoglobulin E; ISM: indolent systemic mastocytosis;
LTE4: leukotriene E4; MC: mast cell; MCAS: mast cell activation syndrome.
Reference(s): Valent P et al. J Allergy Clin Immunol Pract. 2022:10:1999-2012.
Impact of ISM Disease on QoL
Reference(s): Mesa R et al. ISPOR-The Professional Society for Health Economics and Outcomes Research Europe 2023;
Poster number: PCRI36.
www.peervoice.com/NXY870 Copyright © 2010-2024, PeerVoice
Abbreviations and References
Indolent Systemic Mastocytosis: Spectrum of Signs and Symptoms
Abbreviation(s): SM: systemic mastocytosis.
Reference(s): European Medical Journal. Transforming Management of Indolent Systemic Mastocytosis With Precision
Medicine. https://www.emjreviews.com/allergy-immunology/symposium/transforming-management-of-indolent-
systemic-mastocytosis-with-precision-medicine-j010124/. Published 11 July, 2024. Accessed 24 August, 2024.
Pyatilova P, Siebenhaar F. Immunol Allergy Clin North Am. 2023;43:751-762.
Siebenhaar F et al. Allergy. 2018:73:1489-1496.
Initial Approaches to Managing Symptoms of Confirmed ISM
Abbreviation(s): Ca: calcium; HR: histamine receptor; IgE: immunoglobulin E; ISM: indolent systemic mastocytosis;
LTE4: leukotriene E4; MC: mast cell; MCAS: mast cell activation syndrome.
Reference(s): Valent P et al. J Allergy Clin Immunol Pract. 2022:10:1999-2012.
Impact of ISM Disease on QoL
Reference(s): Mesa R et al. ISPOR-The Professional Society for Health Economics and Outcomes Research Europe 2023;
Poster number: PCRI36.
www.peervoice.com/NXY870 Copyright © 2010-2024, PeerVoice
PeerVoice
Abbreviations and References (Cont'd)
Assessments of ISM Disease Burden
Abbreviation(s): BM: bone marrow; BMD: bone mineral density; ISM-SAF: ISM Symptom Assessment Form;
MAS: Mastocytosis Activity Score; MC-QoL: Mastocytosis Quality of Life Questionnaire; MQLQ: Mastocytosis Quality of
Life Questionnaire; MSAF: Mastocytosis Symptom Assessment Form; VAF: variant allele frequency.
Reference(s): Pyatilova P et al. J Allergy Clin Immunol Pract. 2022:10:2015-2024.
Mastocytosis Quality of Life (MC-QoL)
Reference(s): Siebenhaar F et al. Allergy. 2016:71:869-877.
Clinical Case: Assessing QoL and Disease Activity
Abbreviation(s): H: histamine; MCT: Mastocytosis Control Test; NRS: numeric rating scale; PROMs: patient-reported
outcome measures.
www.peervoice.com/NXY870 Copyright © 2010-2024, PeerVoice
Abbreviations and References (Cont'd)
Assessments of ISM Disease Burden
Abbreviation(s): BM: bone marrow; BMD: bone mineral density; ISM-SAF: ISM Symptom Assessment Form;
MAS: Mastocytosis Activity Score; MC-QoL: Mastocytosis Quality of Life Questionnaire; MQLQ: Mastocytosis Quality of
Life Questionnaire; MSAF: Mastocytosis Symptom Assessment Form; VAF: variant allele frequency.
Reference(s): Pyatilova P et al. J Allergy Clin Immunol Pract. 2022:10:2015-2024.
Mastocytosis Quality of Life (MC-QoL)
Reference(s): Siebenhaar F et al. Allergy. 2016:71:869-877.
Clinical Case: Assessing QoL and Disease Activity
Abbreviation(s): H: histamine; MCT: Mastocytosis Control Test; NRS: numeric rating scale; PROMs: patient-reported
outcome measures.
www.peervoice.com/NXY870 Copyright © 2010-2024, PeerVoice
PeerVoice
Part 3 of 3: Escalating Treatment in Insufficiently Controlled ISM:
Recommended Therapeutic Approaches
Frank Siebenhaar, PD Vito Sabato, MD, PhD
Doctor of Medicine and Head of Outpatient Clinics Professor of Allergy and Clinical Immunology
Charité Institute of Allergology University of Antwerp
Berlin, Germany Senior Staff Member
‘Antwerp University Hospital
Antwerp, Belgium
Copyright © 2010-2024, PeerVoice
Part 3 of 3: Escalating Treatment in Insufficiently Controlled ISM:
Recommended Therapeutic Approaches
Frank Siebenhaar, PD Vito Sabato, MD, PhD
Doctor of Medicine and Head of Outpatient Clinics Professor of Allergy and Clinical Immunology
Charité Institute of Allergology University of Antwerp
Berlin, Germany Senior Staff Member
‘Antwerp University Hospital
Antwerp, Belgium
Copyright © 2010-2024, PeerVoice
PeerVoice
Frank Siebenhaar, PD, has a financial interest/relationship or affiliation in the form of.
Consultant for Cogent Biosciences, Inc; Granular Therapeutics; Invea Therapeutics, Inc;
and Vintura.
Other Financial or Material Support from Blueprint Medicines Corporation; Cogent
Biosciences, Inc; Granular Therapeutics; Invea Therapeutics, Inc.; Noucor; Novartis AG;
and Sanofi.
Vito Sabato, MD, PhD, has a financial interest/relationship or affiliation in the form of:
Grant/Research Support from Blueprint Medicines Corporation and Cogent Biosciences,
Inc.
Speakers Bureau participant with Blueprint Medicines Corporation and Cogent
Biosciences, Inc.
Advisory Board for Blueprint Medicines Corporation; Cogent Biosciences, Inc; Novartis
AG; and Telios Pharma, Inc.
Speaker or participant in accredited CME/CPD for Blueprint Medicines Corporation and
Cogent Biosciences, Inc.
www.peervoice.com/NXY870 Copyright © 2010-2024, PeerVoice
Frank Siebenhaar, PD, has a financial interest/relationship or affiliation in the form of.
Consultant for Cogent Biosciences, Inc; Granular Therapeutics; Invea Therapeutics, Inc;
and Vintura.
Other Financial or Material Support from Blueprint Medicines Corporation; Cogent
Biosciences, Inc; Granular Therapeutics; Invea Therapeutics, Inc.; Noucor; Novartis AG;
and Sanofi.
Vito Sabato, MD, PhD, has a financial interest/relationship or affiliation in the form of:
Grant/Research Support from Blueprint Medicines Corporation and Cogent Biosciences,
Inc.
Speakers Bureau participant with Blueprint Medicines Corporation and Cogent
Biosciences, Inc.
Advisory Board for Blueprint Medicines Corporation; Cogent Biosciences, Inc; Novartis
AG; and Telios Pharma, Inc.
Speaker or participant in accredited CME/CPD for Blueprint Medicines Corporation and
Cogent Biosciences, Inc.
www.peervoice.com/NXY870 Copyright © 2010-2024, PeerVoice
PeerVoice
Proportion of Patients With ISM Whose Symptoms Are
Insufficiently Controlled
PIONEER Baseline BSC Use by Drug Class QoL and Symptom Burden
100% Or 60
50
80%
8 655% 5
a ¥ 40
E 60% $
2 = 30
a 40% 310% Br o
248% so 7%
10
o
ESOS ee 2 None Mild Moderate Severe
PPO EES EE :
US SE m QoL impairment (MC-QoL)
g x
x e aie oe oF m Symptom burden (MAS)
> oe
www.peervoice.com/NXY870 Copyright © 2010-2024, PeerVoice
Proportion of Patients With ISM Whose Symptoms Are
Insufficiently Controlled
PIONEER Baseline BSC Use by Drug Class QoL and Symptom Burden
100% Or 60
50
80%
8 655% 5
a ¥ 40
E 60% $
2 = 30
a 40% 310% Br o
248% so 7%
10
o
ESOS ee 2 None Mild Moderate Severe
PPO EES EE :
US SE m QoL impairment (MC-QoL)
g x
x e aie oe oF m Symptom burden (MAS)
> oe
www.peervoice.com/NXY870 Copyright © 2010-2024, PeerVoice
PeerVoice
Avapritinib (PIONEER): Individual Symptom Scores of ISM-SAF
Mean TSS Absolute Change From Baseline up to 48 wk, Individual ISM-SAF,
by Treatment Group
IM Avapritinib 25 mg QD, 24 wk,n=131 M Placebo, 24wkn=66 IM Avapritinib 25 mg QD, 48 wk
LU
20
Change From Baseline
25
30
Flushing Itching Spots Brain Dizziness Headache
fog
Neurocognitive
Bone
pain
Systemic
Abdominal Diarthoea Nausea
pain sensitivity
Gastrointestinal
Skin
www.peervoice.com/NXY870 Copyright © 2010-2024, PeerVoice
Avapritinib (PIONEER): Individual Symptom Scores of ISM-SAF
Mean TSS Absolute Change From Baseline up to 48 wk, Individual ISM-SAF,
by Treatment Group
IM Avapritinib 25 mg QD, 24 wk,n=131 M Placebo, 24wkn=66 IM Avapritinib 25 mg QD, 48 wk
LU
20
Change From Baseline
25
30
Flushing Itching Spots Brain Dizziness Headache
fog
Neurocognitive
Bone
pain
Systemic
Abdominal Diarthoea Nausea
pain sensitivity
Gastrointestinal
Skin
www.peervoice.com/NXY870 Copyright © 2010-2024, PeerVoice
PeerVoice
Avapritinib (PIONEER): MC-QoL Measure
Change in Mean MC-QoL Component Score From MC-Qol Total Score
Baseline to Wk 24 in ITT Population ITT Patients Part 2 and Part 3
s Social tel su Rollover to open-label extension
toms Functioning Emotions Skin
di 704 Double-blind pg Open-iabel y
® $ es] * treatment period ”* extension (ongoing)
‘ ©
£ 60
5 E
H gs All patients on
E ¿so avapritinib (ongoing)
8
7 30 379
A 3
a En AS
ö 3 35
0052 To
seras Pr 3°
M Avapritinib 25 mg QD M Placebo =
aos 8 2 16 20 M 2 2 36 40 44 a8
Avapriti i
GENK 25mg QD (n = 141) Visit, wk
M Avapritinib 25 mg QD M Placebo
Mean % change a cask
nooo. (-399%0-287) (-251t0-108) 000 Im Placebo group crossing over to receive avapritinib 25 mg QD
www.peervoice.com/NXY870 Copyright © 2010-2024, PeerVoice
Avapritinib (PIONEER): MC-QoL Measure
Change in Mean MC-QoL Component Score From MC-Qol Total Score
Baseline to Wk 24 in ITT Population ITT Patients Part 2 and Part 3
s Social tel su Rollover to open-label extension
toms Functioning Emotions Skin
di 704 Double-blind pg Open-iabel y
® $ es] * treatment period ”* extension (ongoing)
‘ ©
£ 60
5 E
H gs All patients on
E ¿so avapritinib (ongoing)
8
7 30 379
A 3
a En AS
ö 3 35
0052 To
seras Pr 3°
M Avapritinib 25 mg QD M Placebo =
aos 8 2 16 20 M 2 2 36 40 44 a8
Avapriti i
GENK 25mg QD (n = 141) Visit, wk
M Avapritinib 25 mg QD M Placebo
Mean % change a cask
nooo. (-399%0-287) (-251t0-108) 000 Im Placebo group crossing over to receive avapritinib 25 mg QD
www.peervoice.com/NXY870 Copyright © 2010-2024, PeerVoice
PeerVoice
Avapritinib (PIONEER): Safety Profile
Majority of AEs were Grade 1 or 2 in the treatment group and no
higher than in the placebo group
Copyright © 2010-2024, PeerVoice
Avapritinib (PIONEER): Safety Profile
Majority of AEs were Grade 1 or 2 in the treatment group and no
higher than in the placebo group
Copyright © 2010-2024, PeerVoice
PeerVoice
Novel Disease-Modifying Approaches in ISM
Inhibition of Mediators and Mast Cell Silencing
ntelimab
Ye iglec-8) ">
02008 y, sc Pa
Jo
Fey Mast coll
Activation, À
Sarilumab Ibrutinib
Anti-IL-6 BTK-inhibitors
Inhibition
In clinical development;
® Not approved for the treatment of ISM in the EU
www.peervoice.com/NXY870
Ki
ar
Barzolvolimab
@ntiKIT)
= _Spoptosie) „
Y Avapritinib (TKI)
Bezuclastinib (TKI)?
Elenestinib (TKI)?
KT Masitinib (TKI)*>
Midostaurin (TKI)?
Inhibition
and/or
& apoptosis
Mast Cell Depletion
Copyright © 2010-2024, PeerVoice
Novel Disease-Modifying Approaches in ISM
Inhibition of Mediators and Mast Cell Silencing
ntelimab
Ye iglec-8) ">
02008 y, sc Pa
Jo
Fey Mast coll
Activation, À
Sarilumab Ibrutinib
Anti-IL-6 BTK-inhibitors
Inhibition
In clinical development;
® Not approved for the treatment of ISM in the EU
www.peervoice.com/NXY870
Ki
ar
Barzolvolimab
@ntiKIT)
= _Spoptosie) „
Y Avapritinib (TKI)
Bezuclastinib (TKI)?
Elenestinib (TKI)?
KT Masitinib (TKI)*>
Midostaurin (TKI)?
Inhibition
and/or
& apoptosis
Mast Cell Depletion
Copyright © 2010-2024, PeerVoice
PeerVoice
Clinical Case: Assessing QoL and Disease Activity
Medical History
+ Skin lesions for 15 years
+ Pruritus: 7/10 (NRS)
+ Darier's sign: Positive
+ No allergic reactions
+ Vertebral compression
fractures (2 years ago)
Additional Symptoms
- Abdominal cramping
+ Diarrhoea
* Bone pain
+ ‘Brain fog’
+ Osteoporosis
ease Burden (PROM:
* MC-QoL = 81
+ MAS =58
+ MCT=3
Moderate-to-severe symptom
burden and QoL impairment
Initial Treatment Plan
+ 2nd-generation antihistamine
(4-fold dose)
* H2 blocker
* Disodium cromoglycate
» Alendronate
+ Calcium + vitamin D
Images used with patient permission; Courtesy of Frank Siebenhaar, PD.
www.peervoice.com/NXY870
+ MC-QoL = 58
+ MAS =32
+ MCT=6
Continued moderate-to-severe
symptom burden and QoL.
impairment, uncontrolled disease
Copyright © 2010-2024, PeerVoice
Clinical Case: Assessing QoL and Disease Activity
Medical History
+ Skin lesions for 15 years
+ Pruritus: 7/10 (NRS)
+ Darier's sign: Positive
+ No allergic reactions
+ Vertebral compression
fractures (2 years ago)
Additional Symptoms
- Abdominal cramping
+ Diarrhoea
* Bone pain
+ ‘Brain fog’
+ Osteoporosis
ease Burden (PROM:
* MC-QoL = 81
+ MAS =58
+ MCT=3
Moderate-to-severe symptom
burden and QoL impairment
Initial Treatment Plan
+ 2nd-generation antihistamine
(4-fold dose)
* H2 blocker
* Disodium cromoglycate
» Alendronate
+ Calcium + vitamin D
Images used with patient permission; Courtesy of Frank Siebenhaar, PD.
www.peervoice.com/NXY870
+ MC-QoL = 58
+ MAS =32
+ MCT=6
Continued moderate-to-severe
symptom burden and QoL.
impairment, uncontrolled disease
Copyright © 2010-2024, PeerVoice
PeerVoice
Abbreviations and References
Proportion of Patients With ISM Whose Symptoms Are Insufficiently Controlled
Abbreviation(s): BSC: best supportive care; H: histamine; IgE: immunoglobin E; ISM: indolent systemic mastocytosis;
MAS: Mastocytosis Activity Score; MC-QoL: Mastocytosis Quality of Life Questionnaire; PPI: proton pump inhibitor.
Reference(s): Siebenhaar F et al. Allergy. 2018;73:1489-1496.
Siebenhaar F et al. European Academy of Allergy and Clinical Immunology (EAACI) Congress 2023. Symptom and
Quality of Life Burden in Patients with Indolent Systemic Mastocytosis.
Avapritinib (PIONEER): Individual Symptom Scores of ISM-SAF
Abbreviation(s): ISM-SAF: ISM Assessment Form; TSS: total symptom score.
Reference(s): Castells M et al. 2023 American Academy of Allergy, Asthma and Immunology (AAAAI) Annual Meeting.
Abstract 627.
Avapritinib (PIONEER): MC-QoL Measure
Abbreviation(s): ITT: intention-to-treat
Reference(s): Castells M et al. 2023 AAAAI Annual Meeting. Abstract 627.
Avapritinib (PIONEER): Safety Profile
Reference(s): Castells M et al. 2023 AAAAI Annual Meeting. Abstract 627.
www.peervoice.com/NXY870 Copyright © 2010-2024, PeerVoice
Abbreviations and References
Proportion of Patients With ISM Whose Symptoms Are Insufficiently Controlled
Abbreviation(s): BSC: best supportive care; H: histamine; IgE: immunoglobin E; ISM: indolent systemic mastocytosis;
MAS: Mastocytosis Activity Score; MC-QoL: Mastocytosis Quality of Life Questionnaire; PPI: proton pump inhibitor.
Reference(s): Siebenhaar F et al. Allergy. 2018;73:1489-1496.
Siebenhaar F et al. European Academy of Allergy and Clinical Immunology (EAACI) Congress 2023. Symptom and
Quality of Life Burden in Patients with Indolent Systemic Mastocytosis.
Avapritinib (PIONEER): Individual Symptom Scores of ISM-SAF
Abbreviation(s): ISM-SAF: ISM Assessment Form; TSS: total symptom score.
Reference(s): Castells M et al. 2023 American Academy of Allergy, Asthma and Immunology (AAAAI) Annual Meeting.
Abstract 627.
Avapritinib (PIONEER): MC-QoL Measure
Abbreviation(s): ITT: intention-to-treat
Reference(s): Castells M et al. 2023 AAAAI Annual Meeting. Abstract 627.
Avapritinib (PIONEER): Safety Profile
Reference(s): Castells M et al. 2023 AAAAI Annual Meeting. Abstract 627.
www.peervoice.com/NXY870 Copyright © 2010-2024, PeerVoice
PeerVoice
Abbreviations and References (Cont'd)
Novel Disease-Modifying Approaches in ISM
Abbreviation(s): BTK: Bruton's tyrosine kinase; CD: cluster of differentiation; FeyR: Fe receptor (IgG); FceR: Fc receptor
(IgE); in-6; SCF: stem cell factor; siglec: sialic acid immunoglobulin-like lectin; TKI: tyrosine kinase inhibitor.
Reference(s): Kolkhir P et al. Nat Rev Immunol. 2022;22:294-308.
Gotlib J et al. NEJM Evid. 2023;2:EVIDoa2200339.
Clinicaltrial gov. NCT05186753. Last update posted 29 August, 2024, Accessed 10 September, 2024.
Modena B et al. 2024 AAAAI Annual Meeting. Poster 694.
Clinicaltrial.gov. NCTO4910685. Last update posted 1 July, 2024. Accessed 10 September, 2024.
Tashi T et al. 65th American Society of Haematology (ASH) Annual Meeting and Exhibition. 2023. Abstract 76.
Siebenhaar F et al. Allergy. 2018;73:1489-1496.
Terhorst-Molawi D et al. Allergy. 2022:78:1269-1279.
Clinical Case: Assessing QoL and Disease Activity
Abbreviation(s): MCT: Mastocytosis Control Test; NRS: numeric rating scale; PROMs: patient-reported outcome
measures.
www.peervoice.com/NXY870 Copyright © 2010-2024, PeerVoice
Abbreviations and References (Cont'd)
Novel Disease-Modifying Approaches in ISM
Abbreviation(s): BTK: Bruton's tyrosine kinase; CD: cluster of differentiation; FeyR: Fe receptor (IgG); FceR: Fc receptor
(IgE); in-6; SCF: stem cell factor; siglec: sialic acid immunoglobulin-like lectin; TKI: tyrosine kinase inhibitor.
Reference(s): Kolkhir P et al. Nat Rev Immunol. 2022;22:294-308.
Gotlib J et al. NEJM Evid. 2023;2:EVIDoa2200339.
Clinicaltrial gov. NCT05186753. Last update posted 29 August, 2024, Accessed 10 September, 2024.
Modena B et al. 2024 AAAAI Annual Meeting. Poster 694.
Clinicaltrial.gov. NCTO4910685. Last update posted 1 July, 2024. Accessed 10 September, 2024.
Tashi T et al. 65th American Society of Haematology (ASH) Annual Meeting and Exhibition. 2023. Abstract 76.
Siebenhaar F et al. Allergy. 2018;73:1489-1496.
Terhorst-Molawi D et al. Allergy. 2022:78:1269-1279.
Clinical Case: Assessing QoL and Disease Activity
Abbreviation(s): MCT: Mastocytosis Control Test; NRS: numeric rating scale; PROMs: patient-reported outcome
measures.
www.peervoice.com/NXY870 Copyright © 2010-2024, PeerVoice
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