The definition describes mastocytosis as a clonal neoplasm, meaning it arises from the expansion of abnormal mast cells. The packet does not certify associated genes, a molecular mechanism, or an inheritance pattern for this condition, so none are described.

Diagnostic methods, diagnostic criteria, and newborn screening information are not certified in this packet, so they are not described here.

The packet lists three products with active FDA-approved status in its approved-treatment records. Ayvakit (avapritinib) was approved in 2020 and Rydapt (midostaurin) in 2017, each through a new drug application reviewed by CDER. Gastrocrom (cromolyn sodium) was approved in 1996 through a new drug application also reviewed by CDER. The specific subtypes covered by each approval are not certified in this packet.

The packet also lists several other products with FDA orphan drug designations for mastocytosis. An orphan designation is not an approval, and one antibody designation is recorded as withdrawn. Foundational therapy and management guidance are not certified in this packet.

A natural history field is not certified in this packet. The definition notes that the condition ranges from cutaneous proliferations that may regress spontaneously to aggressive neoplasms associated with organ failure and short survival, indicating a wide range of disease courses.

Several certified active clinical trial records are present for mastocytosis, including studies of targeted drug therapies in systemic mastocytosis and observational studies. Active clinical trials for this condition are listed on ClinicalTrials.gov.