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Systemic mast cell disease(MASTSYS)

MedGen UID:
67436
Concept ID:
C0221013
Neoplastic Process
Synonym: Systemic mastocytosis
SNOMED CT: Systemic mast cell disease (397016004); Systemic mastocytosis (397016004); SMCD - systemic mast cell disease (397016004); Systemic mastocytosis (1255137009)
Modes of inheritance:
Not genetically inherited
MedGen UID:
988794
Concept ID:
CN307044
Finding
Source: Orphanet
clinical entity without genetic inheritance.
 
Monarch Initiative: MONDO:0016586
OMIM®: 154800
Orphanet: ORPHA2467

Definition

Systemic mastocytosis is a blood disorder that can affect many different body systems. Individuals with the condition can develop signs and symptoms at any age, but it usually appears after adolescence.

Signs and symptoms of systemic mastocytosis often include extreme tiredness (fatigue), skin redness and warmth (flushing), nausea, abdominal pain, bloating, diarrhea, the backflow of stomach acids into the esophagus (gastroesophageal reflux), nasal congestion, shortness of breath, low blood pressure (hypotension), lightheadedness, and headache. Some affected individuals have attention or memory problems, anxiety, or depression. Many individuals with systemic mastocytosis develop a skin condition called urticaria pigmentosa, which is characterized by raised patches of brownish skin that sting or itch with contact or changes in temperature. Nearly half of individuals with systemic mastocytosis will experience severe allergic reactions (anaphylaxis).

There are five subtypes of systemic mastocytosis, which are differentiated by their severity and the signs and symptoms. The mildest forms of systemic mastocytosis are the indolent and smoldering types. Individuals with these types tend to have only the general signs and symptoms of systemic mastocytosis described above. Individuals with smoldering mastocytosis may have more organs affected and more severe features than those with indolent mastocytosis. The indolent type is the most common type of systemic mastocytosis.

The severe types include aggressive systemic mastocytosis, systemic mastocytosis with an associated hematologic neoplasm, and mast cell leukemia. These types are associated with a reduced life span, which varies among the types and affected individuals. In addition to the general signs and symptoms of systemic mastocytosis, these types typically involve impaired function of an organ, such as the liver, spleen, or lymph nodes. The organ dysfunction can result in an abnormal buildup of fluid in the abdominal cavity (ascites). Aggressive systemic mastocytosis is associated with a loss of bone tissue (osteoporosis and osteopenia) and multiple bone fractures. Systemic mastocytosis with an associated hematologic neoplasm and mast cell leukemia both involve blood cell disorders or blood cell cancer (leukemia). Mast cell leukemia is the rarest and most severe type of systemic mastocytosis.

Individuals with the milder forms of the condition generally have a normal or near normal life expectancy, while those with the more severe forms typically survive months or a few years after diagnosis. [from MedlinePlus Genetics]

Professional guidelines

PubMed

Gotlib J
Hematol Oncol Clin North Am 2017 Aug;31(4):643-661. doi: 10.1016/j.hoc.2017.04.009. PMID: 28673393
Tefferi A
Leuk Res 2004 Mar;28(3):223-4. doi: 10.1016/j.leukres.2003.09.004. PMID: 14687615
Mohyi D, Tabassi K, Simon J
Maturitas 1997 Jul;27(3):203-14. doi: 10.1016/s0378-5122(97)83974-6. PMID: 9288692

Curated

NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines®), Systemic Mastocytosis, 2024

Recent clinical studies

Etiology

Kiladjian JJ, Mesa RA, Hoffman R
Blood 2011 May 5;117(18):4706-15. Epub 2011 Mar 9 doi: 10.1182/blood-2010-08-258772. PMID: 21389325
Pagano L, Valentini CG, Caira M, Rondoni M, Van Lint MT, Candoni A, Allione B, Cattaneo C, Marbello L, Caramatti C, Pogliani EM, Iannitto E, Giona F, Ferrara F, Invernizzi R, Fanci R, Lunghi M, Fianchi L, Sanpaolo G, Stefani PM, Pulsoni A, Martinelli G, Leone G, Musto P
Int J Hematol 2008 Dec;88(5):483-488. Epub 2008 Nov 26 doi: 10.1007/s12185-008-0166-4. PMID: 19034614
Pardanani A, Baek JY, Li CY, Butterfield JH, Tefferi A
Mayo Clin Proc 2002 Nov;77(11):1169-75. doi: 10.4065/77.11.1169. PMID: 12440552
Johnstone PA, Mican JM, Metcalfe DD, DeLaney TF
Am J Clin Oncol 1994 Aug;17(4):328-30. doi: 10.1097/00000421-199408000-00009. PMID: 7519391
Webb TA, Li CY, Yam LT
Cancer 1982 Mar 1;49(5):927-38. doi: 10.1002/1097-0142(19820301)49:5<927::aid-cncr2820490517>3.0.co;2-b. PMID: 6174198

Diagnosis

Gotlib J
Hematol Oncol Clin North Am 2017 Aug;31(4):643-661. doi: 10.1016/j.hoc.2017.04.009. PMID: 28673393
Horny HP, Ruck P, Kröber S, Kaiserling E
Histol Histopathol 1997 Oct;12(4):1081-9. PMID: 9302569
Mohyi D, Tabassi K, Simon J
Maturitas 1997 Jul;27(3):203-14. doi: 10.1016/s0378-5122(97)83974-6. PMID: 9288692
Huang TY, Yam LT, Li CY
Br J Radiol 1987 Aug;60(716):765-70. doi: 10.1259/0007-1285-60-716-765. PMID: 3664176
MUTTER RD, TANNENBAUM M, ULTMANN JE
Ann Intern Med 1963 Dec;59:887-906. doi: 10.7326/0003-4819-59-6-887. PMID: 14082741

Therapy

Gotlib J
Hematol Oncol Clin North Am 2017 Aug;31(4):643-661. doi: 10.1016/j.hoc.2017.04.009. PMID: 28673393
Tefferi A, Pardanani A
Curr Opin Hematol 2004 Jan;11(1):58-64. doi: 10.1097/00062752-200401000-00009. PMID: 14676628
Nieva J, Saven A
Leuk Res 2004 Feb;28(2):113-4. doi: 10.1016/s0145-2126(03)00258-3. PMID: 14654073
Pardanani A, Elliott M, Reeder T, Li CY, Baxter EJ, Cross NC, Tefferi A
Lancet 2003 Aug 16;362(9383):535-6. doi: 10.1016/s0140-6736(03)14115-3. PMID: 12932387
Debeuckelaere S, Schoors DF, Devis G
Acta Clin Belg 1991;46(4):226-32. PMID: 1683083

Prognosis

Tefferi A, Pardanani A
Curr Opin Hematol 2004 Jan;11(1):58-64. doi: 10.1097/00062752-200401000-00009. PMID: 14676628
Pardanani A, Baek JY, Li CY, Butterfield JH, Tefferi A
Mayo Clin Proc 2002 Nov;77(11):1169-75. doi: 10.4065/77.11.1169. PMID: 12440552
Horny HP, Ruck P, Kröber S, Kaiserling E
Histol Histopathol 1997 Oct;12(4):1081-9. PMID: 9302569
Debeuckelaere S, Schoors DF, Devis G
Acta Clin Belg 1991;46(4):226-32. PMID: 1683083
Travis WD, Li CY, Bergstralh EJ, Yam LT, Swee RG
Medicine (Baltimore) 1988 Nov;67(6):345-68. PMID: 3054417

Clinical prediction guides

Kiladjian JJ, Mesa RA, Hoffman R
Blood 2011 May 5;117(18):4706-15. Epub 2011 Mar 9 doi: 10.1182/blood-2010-08-258772. PMID: 21389325
Hesselmann S, Micke O, Schaefer U, Willich N
Strahlenther Onkol 2002 May;178(5):275-9. doi: 10.1007/s00066-002-0928-5. PMID: 12082688
Johnstone PA, Mican JM, Metcalfe DD, DeLaney TF
Am J Clin Oncol 1994 Aug;17(4):328-30. doi: 10.1097/00000421-199408000-00009. PMID: 7519391
Travis WD, Li CY, Bergstralh EJ, Yam LT, Swee RG
Medicine (Baltimore) 1988 Nov;67(6):345-68. PMID: 3054417
Webb TA, Li CY, Yam LT
Cancer 1982 Mar 1;49(5):927-38. doi: 10.1002/1097-0142(19820301)49:5<927::aid-cncr2820490517>3.0.co;2-b. PMID: 6174198

Supplemental Content

Table of contents

    Clinical resources

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    • PubMed
      See practice and clinical guidelines in PubMed. The search results may include broader topics and may not capture all published guidelines. See the FAQ for details.
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      See practice and clinical guidelines in NCBI Bookshelf. The search results may include broader topics and may not capture all published guidelines. See the FAQ for details.

    Curated

    • NCCN, 2024
      NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines®), Systemic Mastocytosis, 2024

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