Systemic Mastocytosis Clinical Trial
— EvaTryMSOfficial title:
Evaluation of the Diagnostic Value of Level of Bone Marrow Tryptase in Adult Systemic Mastocytosis
Verified date | July 2019 |
Source | University Hospital, Toulouse |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Interventional |
The hypothesis of the study is that Bone Marrow Tryptase (MT) level is a diagnostic marker of
Systemic Mastocytosis (SM). Determination of the bone marrow tryptase in Bone Marrow Aspirate
(BMA) could be a new diagnostic criteria for systemic mastocytosis with sensitivity close to
100% and a low false negative rate. This new test could be useful to improve the ability to
diagnose accurately systemic mastocytosis (in particular the indolent forms). Because of its
limited invasiveness compared to bone marrow biopsy, it could also be considered as a test
performed before bone marrow biopsy. Only patients with high bone marrow tryptase would then
undergo bone marrow biopsy.
In the future and if validated by this study, bone marrow tryptase could be a useful marker
of mast cell load and help to monitor the efficacy of treatment in systemic mastocytosis.
Status | Completed |
Enrollment | 250 |
Est. completion date | July 2019 |
Est. primary completion date | July 2019 |
Accepts healthy volunteers | No |
Gender | All |
Age group | 18 Years and older |
Eligibility |
Inclusion Criteria: - Suspicion of systemic mastocytosis, for whom bone marrow sampling (bone marrow biopsy and/or bone marrow aspiration) is carried out as part of normal workup of the disease in the center of reference and centers of competence in mastocytosis. - Patient whose written informed consent has been obtained. Exclusion Criteria: - Patients with a contra-indication to marrow sampling (anticoagulant and/or anti-clotting treatments, - Thrombocytopenia < 50 000/mm2) for whom it is impossible to formally conclude the final type of mast cell disease: SM, CM, mast cell activation syndrome. |
Country | Name | City | State |
---|---|---|---|
France | CHU Bordeaux Hôpital Haut-Lévêque, service de dermatologie | Bordeaux | |
France | CHU de Caen, service d'hématologie | Caen | |
France | CHU Dupuytren service d'hématologie | Limoges | |
France | CHU Lyon Sud, service de médecine interne | Lyon | |
France | Hôpital Necker, service d'hématologie | Paris | |
France | Hôpital Pité Salpétrière | Paris | |
France | CHU Toulouse, Hôpital Larrey, service de dermatologie | Toulouse | |
Martinique | CHU Fort de France, service de dermatologie | Fort-de-France |
Lead Sponsor | Collaborator |
---|---|
University Hospital, Toulouse |
France, Martinique,
Akin C, Valent P, Metcalfe DD. Mast cell activation syndrome: Proposed diagnostic criteria. J Allergy Clin Immunol. 2010 Dec;126(6):1099-104.e4. doi: 10.1016/j.jaci.2010.08.035. Epub 2010 Oct 28. — View Citation
Alvarez-Twose I, González de Olano D, Sánchez-Muñoz L, Matito A, Esteban-López MI, Vega A, Mateo MB, Alonso Díaz de Durana MD, de la Hoz B, Del Pozo Gil MD, Caballero T, Rosado A, Sánchez Matas I, Teodósio C, Jara-Acevedo M, Mollejo M, García-Montero A, Orfao A, Escribano L. Clinical, biological, and molecular characteristics of clonal mast cell disorders presenting with systemic mast cell activation symptoms. J Allergy Clin Immunol. 2010 Jun;125(6):1269-1278.e2. doi: 10.1016/j.jaci.2010.02.019. — View Citation
Barete S, Assous N, de Gennes C, Grandpeix C, Feger F, Palmerini F, Dubreuil P, Arock M, Roux C, Launay JM, Fraitag S, Canioni D, Billemont B, Suarez F, Lanternier F, Lortholary O, Hermine O, Francès C. Systemic mastocytosis and bone involvement in a cohort of 75 patients. Ann Rheum Dis. 2010 Oct;69(10):1838-41. doi: 10.1136/ard.2009.124511. Epub 2010 Jun 22. — View Citation
Paul C, Sans B, Suarez F, Casassus P, Barete S, Lanternier F, Grandpeix-Guyodo C, Dubreuil P, Palmérini F, Mansfield CD, Gineste P, Moussy A, Hermine O, Lortholary O. Masitinib for the treatment of systemic and cutaneous mastocytosis with handicap: a phase 2a study. Am J Hematol. 2010 Dec;85(12):921-5. doi: 10.1002/ajh.21894. — View Citation
Valent P, Akin C, Escribano L, Födinger M, Hartmann K, Brockow K, Castells M, Sperr WR, Kluin-Nelemans HC, Hamdy NA, Lortholary O, Robyn J, van Doormaal J, Sotlar K, Hauswirth AW, Arock M, Hermine O, Hellmann A, Triggiani M, Niedoszytko M, Schwartz LB, Orfao A, Horny HP, Metcalfe DD. Standards and standardization in mastocytosis: consensus statements on diagnostics, treatment recommendations and response criteria. Eur J Clin Invest. 2007 Jun;37(6):435-53. Review. — View Citation
Valent P, Horny HP, Escribano L, Longley BJ, Li CY, Schwartz LB, Marone G, Nuñez R, Akin C, Sotlar K, Sperr WR, Wolff K, Brunning RD, Parwaresch RM, Austen KF, Lennert K, Metcalfe DD, Vardiman JW, Bennett JM. Diagnostic criteria and classification of mastocytosis: a consensus proposal. Leuk Res. 2001 Jul;25(7):603-25. Review. — View Citation
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Bone marrow tryptase level | Aliquot of the bone marrow aspirate sample will be used after centrifugation for measurements of bone marrow tryptase level. | Day 4 after bone marrow aspiration | |
Secondary | The bone marrow tryptase level for differential diagnosis between systemic mastocytosis and cell mast activation syndrome | Aliquot of the bone marrow aspirate sample will be used after centrifugation for measurements of bone marrow tryptase level. | Day 4 after bone marrow aspiration | |
Secondary | The bone marrow tryptase/serum tryptase ratio for diagnosis of systemic mastocytosis with and without mastocytosis in skin | Day 4 after bone marrow aspiration | ||
Secondary | The absolute and corrected bone marrow tryptase level for diagnosis of systemic mastocytosis with and without mastocytosis in skin | The degree of dilution of the bonne marrow aspirate by peripheral blood will be estimated by comparing the percentage of CD16bright vs CD16low granulocytes in bone marrow samples and used to adjust the bone marrow tryptase. | Day 4 after bone marrow aspiration | |
Secondary | Flow cytometry performed on cells collected by bone marrow aspirate and maintained in a preservative solution (TransFix®) to detect mast cells expressing CD25 and/or CD2 for diagnosis of systemic mastocytosis with and without mastocytosis in skin | Immunophenotyping of bone marrow mastocytes will be performed on bone marrow aspiration stabilized by Transfix using a pre-defined mixture of anti-CD45/CD117/CD34/CD2/CD25/CD16 antibodies. | Day 4 after bone marrow aspiration | |
Secondary | Quantification of KIT mutations-positive cells fraction in peripheral blood for systemic mastocytosis diagnosis,correlation with results of medullar tryptase level, medullar/systemic tryptase ratio and absolute and corrected medullar tryptase level | Genomic DNA from total leucocytes will be purified from the peripheral blood samples and used for the quantification of the kit mutation by qPCR | Day 4 after bone marrow aspiration |
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