TREATMENT OF CHILDHOOD MASTOCYTOSIS

Özge Kangallı Boyacıoğlu

İzmir Bakırçay University, Çiğli Training and Research Hospital, Department of Pediatric Immunology and Allergy, İzmir, Türkiye

Kangallı Boyacıoğlu Ö. Treatment of Childhood Mastocytosis. In: Özdemir Ö, editor. Childhood Mastocytosis: New Developments in Diagnosis and Treatment. 1st ed. Ankara: Türkiye Klinikleri; 2025. p.141-148.

ABSTRACT

Mastocytosis is a condition characterized by an abnormal accumulation and clonal expansion of mast cells in the skin and various organs. The disease is broadly categorized into two forms: cutaneous mastocytosis and systemic mastocytosis.In pediatric cases, the disease is mostly limited to the skin, with systemic involvement being rare. The primary symptoms of cutaneous mastocytosis include pig- mented macules, papules, plaques, and itching. Diagnosis is based on clinical assessment, histopatho- logical examination, and serum tryptase level measurement.The treatment primarily aims to control symptoms through the use of antihistamines, topical corticosteroids, and mast cell stabilizers, while avoiding triggering factors is also recommended. Management is centered on therapies that minimize symptoms resulting from mast cell mediator release, and treatment is individualized based on the frequency and severity of symptoms. There is a need for novel therapeutic approaches and long-term follow-up, as the potential progression to systemic mastocytosis must be monitored. Despite the avail- able treatment options, there is currently no curative therapy for cutaneous mastocytosis, highlighting the necessity for ongoing research into more effective treatments.

Keywords: Child; Drug therapy; Mast cells; Mastocytosis; Skin lesions; Urticaria pigmentosa

Referanslar

  1. Hartmann K, Henz BM. Mastocytosis: recent advances in defining the disease. Br J Dermatol. 2001 Apr;144(4):682-95. [Crossref]  [PubMed]
  2. Valent P, Akin C, Metcalfe DD. Mastocytosis: 2016 updated WHO classification and novel emerging treatment concepts. Blood. 2017 Mar 16;129(11):1420-1427. [Crossref]  [PubMed]  [PMC]
  3. Castells M, Metcalfe DD, Escribano L. Diagnosis and treatment of cutaneous mastocytosis in children: practical recommendations. Am J Clin Dermatol. 2011 Aug 1;12(4):259-70. [Crossref]  [PubMed]  [PMC]
  4. Heide R, Beishuizen A, De Groot H, Den Hollander JC, Van Doormaal JJ, De Monchy JG, et al; Dutch National Mastocytosis Work Group. Mastocytosis in children: a protocol for management. Pediatr Dermatol. 2008 Jul-Aug;25(4):493-500. [Crossref]  [PubMed]
  5. Carter MC, Metcalfe DD, Komarow HD. Mastocytosis. Immunol Allergy Clin North Am. 2014 Feb;34(1):181-96. Epub 2013 Oct 7. [Crossref]  [PubMed]  [PMC]
  6. Hartmann K, Escribano L, Grattan C, Brockow K, Carter MC, Alvarez-Twose I, et al. Cutaneous manifestations in patients with mastocytosis: Consensus report of the European Competence Network on Mastocytosis; the American Academy of Allergy, Asthma & Immunology; and the European Academy of Allergology and Clinical Immunology. J Allergy Clin Immunol. 2016 Jan;137(1):35-45. [Crossref]  [PubMed]
  7. Lange M, Hartmann K, Carter MC, Siebenhaar F, Alvarez-Twose I, Torrado I, et al. Molecular Background, Clinical Features and Management of Pediatric Mastocytosis: Status 2021. Int J Mol Sci. 2021 Mar 4;22(5):2586. [Crossref]  [PubMed]  [PMC]
  8. Cardet JC, Akin C, Lee MJ. Mastocytosis: update on pharmacotherapy and future directions. Expert Opin Pharmacother. 2013 Oct;14(15):2033-45. [Crossref]  [PubMed]  [PMC]
  9. Carter MC, Metcalfe DD, Matito A, Escribano L, Butterfield JH, Schwartz LB, et al. Adverse reactions to drugs and biologics in patients with clonal mast cell disorders: A Work Group Report of the Mast Cells Disorder Committee, American Academy of Allergy, Asthma & Immunology. J Allergy Clin Immunol. 2019 Mar;143(3):880-893. [Crossref]  [PubMed]
  10. Tiano R, Krase IZ, Sacco K. Updates in diagnosis and management of paediatric mastocytosis. Curr Opin Allergy Clin Immunol. 2023 Apr 1;23(2):158-163. [Crossref]  [PubMed]
  11. Azaña JM, Torrelo A, Matito A. Update on Mastocytosis (Part 2): Categories, Prognosis, and Treatment. Actas Dermosifiliogr. 2016 Jan-Feb;107(1):15-22. English, Spanish. [Crossref]  [PubMed]
  12. Castells M, Butterfield J. Mast Cell Activation Syndrome and Mastocytosis: Initial Treatment Options and Long-Term Management. J Allergy Clin Immunol Pract. 2019Apr;7(4):1097-1106. [Crossref]  [PubMed]
  13. Giona F. Pediatric Mastocytosis: An Update. Mediterr J Hematol Infect Dis. 2021 Nov 1;13(1):e2021069. [Crossref]  [PubMed]  [PMC]
  14. Vlieg-Boerstra BJ, van der Heide S, Oude Elberink JN, Kluin-Nelemans JC, Dubois AE. Mastocytosis and adverse reactions to biogenic amines and histamine-releasing foods: what is the evidence? Neth J Med. 2005 Jul-Aug;63(7):244-9. [PubMed]
  15. Maintz L, Novak N. Histamine and histamine intolerance. Am J Clin Nutr. 2007 May;85(5):1185-96. [Crossref]  [PubMed]
  16. Guyer AC, Saff RR, Conroy M, Blumenthal KG, Camargo CA Jr, Long AA, et al. Comprehensive allergy evaluation is useful in the subsequent care of patients with drug hypersensitivity reactions during anesthesia. J Allergy Clin Immunol Pract. 2015 Jan-Feb;3(1):94-100. [Crossref]  [PubMed]
  17. Hussain SH. Pediatric mastocytosis.Curr Opin Pediatr. 2020Aug;32(4):531-538. [Crossref]  [PubMed]
  18. Schwartz LB, Irani AM, Roller K, Castells MC, Schechter NM. Quantitation of histamine, tryptase, and chymase in dispersed human T and TC mast cells. J Immunol. 1987Apr15;138(8):2611-5. [Crossref]  [PubMed]
  19. Falus A, Merétey K. Histamine: an early messenger in inflammatory and immune reactions. Immunol Today.1992May;13(5):154-6. [Crossref]  [PubMed]
  20. Inoue I, Yanai K, Kitamura D, Taniuchi I, Kobayashi T, Niimura K, et al. Impaired locomotor activity and exploratory behavior in mice lacking histamine H1 receptors. Proc Natl Acad Sci U S A. 1996 Nov 12;93(23):13316-20. [Crossref]  [PubMed]  [PMC]
  21. Siebenhaar F, Akin C, Bindslev-Jensen C, Maurer M, Broesby-Olsen S. Treatment strategies in mastocytosis. Immunol Allergy Clin North Am. 2014 May;34(2):433-47. [Crossref]  [PubMed]
  22. Johnson KB, Johns Hopkins Hospital Children's Medical and Surgical Center. The Harriet Lane Handbook : A Manual for Pediatric House Officers. 13th ed. Mosby; 1993.
  23. Zuberbier T, Aberer W, Asero R, Bindslev-Jensen C, Brzoza Z, Canonica GW, et.al; Dermatology Section of the European Academy of Allergy and Clinical Immunology; Golbal Allergy and Asthma European Network; European Dermatology Forum; World Allergy Organization. Methods report on the development of the 2013 revision and update of the EAACI/GA2 LEN/EDF/WAO guideline for the definition, classification, diagnosis, and management of urticaria. Allergy. 2014 Jul;69(7):e1-29. [PubMed]
  24. Arock M, Akin C, Hermine O, Valent P. Current treatment options in patients with mastocytosis: status in 2015 and future perspectives. Eur J Haematol. 2015 Jun;94(6):474-90. [Crossref]  [PubMed]
  25. Broesby-Olsen S, Dybedal I, Gülen T, Kristensen TK, Møller MB, Ackermann L, et al. Multidisciplinary Management of Mastocytosis: Nordic Expert Group Consensus. Acta Derm Venereol. 2016 Jun 15;96(5):602-12. [Crossref]  [PubMed]
  26. Siebenhaar F, Förtsch A, Krause K, Weller K, Metz M, Magerl M, et al. Rupatadine improves quality of life in mastocytosis: a randomized, double-blind, placebo-controlled trial. Allergy. 2013 Jul;68(7):949-52. [Crossref]  [PubMed]
  27. Worobec AS. Treatment of systemic mast cell disorders. Hematol Oncol Clin North Am. 2000 Jun;14(3):659-87, vii. [Crossref]  [PubMed]
  28. Verma KK, Bhat R, Singh MK. Bullous mastocytosis treated with oral betamethasone therapy. Indian J Pediatr.2004Mar;71(3):261-3. [Crossref]  [PubMed]
  29. Yankova R, Abadjieva T, Belovezhdov V. Cutaneous Mastocytosis with Persistent Blistering: Successful Treatment with Methylprednisolone and 3-Year Follow-Up Management. Dermatol Ther (Heidelb). 2015 Jun;5(2):145-50. [Crossref]  [PubMed]  [PMC]
  30. Mashiah J, Harel A, Bodemer C, Hadj-Rabia S, Goldberg I, Sprecher E, et al. Topical pimecrolimus for paediatric cutaneous mastocytosis. Clin Exp Dermatol. 2018 Jul;43(5):559-565. [Crossref]  [PubMed]
  31. Correia O, Duarte AF, Quirino P, Azevedo R, Delgado L. Cutaneous mastocytosis: Two pediatric cases treated with topical pimecrolimus. Dermatol Online J. 2010 May 15;16(5):8. [Crossref]  [PubMed]
  32. Guhl S, Hartmann K, Tapkenhinrichs S, Smorodchenko A, Grützkau A, Henz BM, et al. Ultraviolet irradiation induces apoptosis in human immature, but not in skin mast cells. J Invest Dermatol. 2003 Oct;121(4):837-44. [Crossref]  [PubMed]
  33. Escribano L. Successful management of a case of diffuse cutaneous mastocytosis with recurrent anaphylactoid episodes and hypertension.J Allergy Clin Immunol. 2004;113(2): S335. [Crossref]
  34. Brazzelli V, Grassi S, Merante S, Grasso V, Ciccocioppo R, Bossi G, et al. Narrow-band UVB phototherapy and psoralen-ultraviolet A photochemotherapy in the treatment of cutaneous mastocytosis: a study in 20 patients. Photodermatol Photoimmunol Photomed. 2016 Sep;32(5-6):238-246. [Crossref]  [PubMed]