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Medications for Systemic Mastocytosis

Other names: Mastocytosis, Systemic; Systemic Mast Cell Disease

Systemic mastocytosis is a rare disease characterized by uncontrolled proliferation and activation of mast cells in tissues and organs such as the skin, bone marrow, gastrointestinal tract, liver, spleen, or lymph nodes.

In most cases of systemic mastocytosis, there is a mutation in the gene encoding the tyrosine kinase receptor KIT causing a D816V substitution and autoactivation of the receptor.

There are five subtypes of systemic mastocytosis:

  • indolent systemic mastocytosis (ISM);
  • smoldering systemic mastocytosis (SSM);
  • systemic mastocytosis with an associated hematological neoplasm (SM-AHN);
  • aggressive systemic mastocytosis (ASM); and
  • mast cell leukemia (MCL).

The majority of patients with systemic mastocytosis have the non-advanced forms (ISM and SSM).  Advanced systemic mastocytosis (AdvSM) is the term collectively used for the three most aggressive forms of the disease (SM-AHN, ASM, and MCL).

Signs and symptoms of systemic mastocytosis include fatigue, skin flushing, nausea, abdominal pain, bloating, diarrhea, gastroesophageal reflux, nasal congestion, shortness of breath, hypotension, lightheadedness, and headache. Nearly half of individuals with systemic mastocytosis will experience severe allergic reactions.

 

Drugs used to treat Systemic Mastocytosis

The following list of medications are in some way related to or used in the treatment of this condition.

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Drug name Rating Reviews Activity ? Rx/OTC Pregnancy CSA Alcohol
avapritinib 1.5 2 reviews for avapritinib to treat Systemic Mastocytosis
Rx N
Generic name:
avapritinib systemic
Brand name:
Ayvakit
Drug class:
multikinase inhibitors
For consumers:
dosage, interactions, side effects
For professionals:
AHFS DI Monograph
Ayvakit Rate Add review
Rx N
Generic name:
avapritinib systemic
Drug class:
multikinase inhibitors
For consumers:
dosage, interactions, side effects
For professionals:
Prescribing Information
cromolyn 7.2 10 reviews for cromolyn to treat Systemic Mastocytosis
Rx B N
Generic name:
cromolyn systemic
Brand name:
Gastrocrom
Drug class:
mast cell stabilizers
For consumers:
dosage, interactions, side effects
For professionals:
AHFS DI Monograph, Prescribing Information
Gastrocrom 6.7 3 reviews for Gastrocrom to treat Systemic Mastocytosis
Rx B N
Generic name:
cromolyn systemic
Drug class:
mast cell stabilizers
For consumers:
dosage, interactions, side effects
For professionals:
Prescribing Information
Gleevec Rate Add review
Rx D N
Generic name:
imatinib systemic
Drug class:
BCR-ABL tyrosine kinase inhibitors
For consumers:
dosage, interactions, side effects
For professionals:
Prescribing Information
imatinib Rate Add review
Rx D N
Generic name:
imatinib systemic
Brand name:
Gleevec
Drug class:
BCR-ABL tyrosine kinase inhibitors
For consumers:
dosage, interactions, side effects
For professionals:
AHFS DI Monograph, Prescribing Information
lansoprazole Rate Add review
Rx/OTC B N
Generic name:
lansoprazole systemic
Brand names:
Prevacid, Prevacid OTC, Prevacid SoluTab
Drug class:
proton pump inhibitors
For consumers:
dosage, interactions, side effects
For professionals:
AHFS DI Monograph, Prescribing Information
midostaurin 8.3 3 reviews for midostaurin to treat Systemic Mastocytosis
Rx N
Generic name:
midostaurin systemic
Brand name:
Rydapt
Drug class:
multikinase inhibitors
For consumers:
dosage, interactions, side effects
For professionals:
AHFS DI Monograph
omeprazole Rate Add review
Rx/OTC C N
Generic name:
omeprazole systemic
Brand name:
Prilosec
Drug class:
proton pump inhibitors
For consumers:
dosage, interactions, side effects
For professionals:
AHFS DI Monograph, Prescribing Information
Prevacid Rate Add review
Rx B N
Generic name:
lansoprazole systemic
Drug class:
proton pump inhibitors
For consumers:
dosage, interactions, side effects
For professionals:
Prescribing Information
Prevacid OTC Rate Add review
OTC B N
Generic name:
lansoprazole systemic
Drug class:
proton pump inhibitors
For consumers:
dosage, interactions, side effects
Prevacid SoluTab Rate Add review
Rx B N
Generic name:
lansoprazole systemic
Drug class:
proton pump inhibitors
For consumers:
dosage, interactions, side effects
Prilosec Rate Add review
Rx C N
Generic name:
omeprazole systemic
Drug class:
proton pump inhibitors
For consumers:
dosage, interactions, side effects
For professionals:
Prescribing Information
Rydapt 7.5 2 reviews for Rydapt to treat Systemic Mastocytosis
Rx N
Generic name:
midostaurin systemic
Drug class:
multikinase inhibitors
For consumers:
dosage, interactions, side effects
For professionals:
Prescribing Information

Frequently asked questions

View more FAQ

Learn more about Systemic Mastocytosis

Mayo Clinic reference

Legend

Rating For ratings, users were asked how effective they found the medicine while considering positive/adverse effects and ease of use (1 = not effective, 10 = most effective).
Activity Activity is based on recent site visitor activity relative to other medications in the list.
Rx Prescription only.
OTC Over-the-counter.
Rx/OTC Prescription or Over-the-counter.
Off-label This medication may not be approved by the FDA for the treatment of this condition.
EUA An Emergency Use Authorization (EUA) allows the FDA to authorize unapproved medical products or unapproved uses of approved medical products to be used in a declared public health emergency when there are no adequate, approved, and available alternatives.
Expanded Access Expanded Access is a potential pathway for a patient with a serious or immediately life-threatening disease or condition to gain access to an investigational medical product (drug, biologic, or medical device) for treatment outside of clinical trials when no comparable or satisfactory alternative therapy options are available.
Pregnancy Category
A Adequate and well-controlled studies have failed to demonstrate a risk to the fetus in the first trimester of pregnancy (and there is no evidence of risk in later trimesters).
B Animal reproduction studies have failed to demonstrate a risk to the fetus and there are no adequate and well-controlled studies in pregnant women.
C Animal reproduction studies have shown an adverse effect on the fetus and there are no adequate and well-controlled studies in humans, but potential benefits may warrant use in pregnant women despite potential risks.
D There is positive evidence of human fetal risk based on adverse reaction data from investigational or marketing experience or studies in humans, but potential benefits may warrant use in pregnant women despite potential risks.
X Studies in animals or humans have demonstrated fetal abnormalities and/or there is positive evidence of human fetal risk based on adverse reaction data from investigational or marketing experience, and the risks involved in use in pregnant women clearly outweigh potential benefits.
N FDA has not classified the drug.
Controlled Substances Act (CSA) Schedule
M The drug has multiple schedules. The schedule may depend on the exact dosage form or strength of the medication.
U CSA Schedule is unknown.
N Is not subject to the Controlled Substances Act.
1 Has a high potential for abuse. Has no currently accepted medical use in treatment in the United States. There is a lack of accepted safety for use under medical supervision.
2 Has a high potential for abuse. Has a currently accepted medical use in treatment in the United States or a currently accepted medical use with severe restrictions. Abuse may lead to severe psychological or physical dependence.
3 Has a potential for abuse less than those in schedules 1 and 2. Has a currently accepted medical use in treatment in the United States. Abuse may lead to moderate or low physical dependence or high psychological dependence.
4 Has a low potential for abuse relative to those in schedule 3. It has a currently accepted medical use in treatment in the United States. Abuse may lead to limited physical dependence or psychological dependence relative to those in schedule 3.
5 Has a low potential for abuse relative to those in schedule 4. Has a currently accepted medical use in treatment in the United States. Abuse may lead to limited physical dependence or psychological dependence relative to those in schedule 4.
Alcohol
X Interacts with Alcohol.

Further information

Always consult your healthcare provider to ensure the information displayed on this page applies to your personal circumstances.