Articles

Advanced systemic mastocytosis: An uncommon cause of chronic diarrhoea and weight loss

BJH - volume 13, issue 2, march 2022

B. Sciot MD, T. Devos MD, PhD, T. Tousseyn MD, PhD, N. Boeckx MD, PhD, L. Michaux MD, PhD, P. Vandenberghe MD, PhD

SUMMARY

Introduction: Advanced systemic mastocytosis is a rare myeloproliferative disorder of mast cells, damaging the function of various organs and tissues. The diagnosis can be challenging due to its protean manifestations and rareness. Treatment options have improved over the last years. Currently, avapritinib, a novel tyrosine kinase inhibitor with activity against p.D816V mutated KIT, is under investigation.

Case: We report a case of a 64-year old man with chronic diarrhoea, fatigue, weight loss and ascites with hepatomegaly, developing an upper gastro-intestinal bleeding with multiple duodenal ulcers. Diagnostic work-up revealed hepatosplenomegaly and portal hypertension, a vertebral compression fracture and multiple 18F-FDG avid supra- and infradiaphragmatic lymph nodes and bone marrow. Based on the 2016 WHO criteria of systemic mastocytosis, and a concomitant chronic myelomonocytic leukaemia, the diagnosis of an aggressive systemic mastocytosis with an associated haematological neoplasm was made. The patient was consecutively treated with midostaurin, cladribine and avapritinib, the latter inducing a complete biochemical and molecular response.

Conclusion: This case illustrates the challenging clinical presentation of systemic mastocytosis. A deep response to avapritinib was observed despite prior use of midostaurin and cladribine, underlining its promise in advanced systemic mastocytosis.

(BELG J HEMATOL 2022;13(2):84–91)

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Tyrosine kinase inhibitor discontinuation in patients with chronic myelogenous leukaemia: A retrospective study and review of the literature

BJH - 2021, issue 2, march 2021

P. Beuselinck MD, Ir J. Van Ham , N. Boeckx MD, PhD, T. Devos MD, PhD, P. Vandenberghe MD, PhD, G. Verhoef MD, PhD

ABSTRACT

BACKGROUND: Tyrosine kinase inhibitors (TKIs) have improved the survival of patients with chronic myeloid leukaemia (CML). TKIs can be successfully discontinued in some CML patients who have achieved a stable deep molecular response.

OBJECTIVE: The purpose of this article is twofold. On the one hand, this review provides an overview of current use and discontinuation of TKIs in patients with CML. On the other hand, we retrospectively investigated the use and possible discontinuation of TKIs in a specific patient population with CML at the University Hospital of Leuven.

METHODS: A literature search was carried out in May 2019 to identify all relevant articles. Articles were searched on PubMed, Embase, Web of Science and Cochrane Library. Additionally, the articles found in the reference list were used.

RESULTS: This review included ten articles (two on imatinib, four on dasatinib, four on nilotinib), with 970 patients. Treatment free remission (TFR) ratio varied from 41–68% after one year. One study published the results of TFR after three years. In UZ Leuven, the TFR ratio was 60% after 106 weeks.

CONCLUSION: Tyrosine kinase inhibitor (TKI) therapy can be safely terminated in selected patient groups. About half of the patients retain the molecular remission after discontinuation of TKI therapy.

(BELG J HEMATOL 2020;12(2):52-8)

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Chimeric antigen receptor T-cells: a new therapeutic option for relapsed/refractory B-cell malignancies and beyond

BJH - volume 10, issue 8, december 2019

T. Feys MBA, MSc, G. Roex , Y. Beguin MD, PhD, T. Kerre MD, PhD, X. Poiré MD, PhD, P. Lewalle MD, PhD, P. Vandenberghe MD, PhD, D. Bron MD, PhD, S. Anguille MD, PhD

Chimeric antigen receptor (CAR) T-cell therapy is a new cancer immunotherapy targeting specific cell surface antigens. This type of adoptive cell immunotherapy has been a breakthrough in the treatment of aggressive B-cell lymphoma and B-cell precursor acute lymphoblastic leukaemia (ALL) and is currently also being studied in other cancer types, including multiple myeloma and chronic lymphocytic leukaemia. This review will discuss the recent clinical developments and future perspectives of CAR T-cell therapy, with a focus on the clinical trials that led to the FDA and EMA approval of tisagenlecleucel (Kymriah®, Novartis) and axicabtagene ciloleucel (Yescarta®, Gilead) for the treatment of childhood/adult relapsed/refractory (r/r) B-cell precursor ALL and aggressive B-cell non-Hodgkin lymphoma.

(BELG J HEMATOL 2019;10(8):301–10)

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NGS: investing in deeper evaluation and optimised therapeutic decisions in myeloid neoplasms

BJH - volume 10, issue 6, october 2019

P. Vandenberghe MD, PhD

Editorial for the contribution of E. Van Valckenborgh et al., entitled: Diagnostic testing in myeloid malignancies by next-generation sequencing: recommendations from the Commission Personalised Medicine (BELG J HEMATOL 2019;10(6):241–9)

(BELG J HEMATOL 2019;10(6):229–30)

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Diagnostic testing in myeloid malignancies by next-generation sequencing: recommendations from the Commission Personalised Medicine

BJH - volume 10, issue 6, october 2019

E. Van Valckenborgh PhD, M. Bakkus PhD, E. Boone PhD, A. Camboni MD, PhD, J-P. Defour PhD, B. Denys MD, H. Devos MD, L. Dewispelaere MD, G. Froyen PhD, A. Hébrant PhD, P. Heimann MD, PhD, P. Hermans MD, PhD, E. Heylen PhD, K. Jacobs PhD, F. Lambert MD, M. Le Mercier Apr, PhD, E. Lierman PhD, H. Louagie MD, PhD, B. Maes MD, PhD, M-B. Maes PhD, G. Martens MD, PhD, L. Michaux MD, PhD, F. Nollet PhD, MSc, H.A. Poirel MD, PhD, G. Raicevic PhD, P. Saussoy MD, PhD, T. Tousseyn MD, PhD, M. Van Den Bulcke PhD, P. Vandenberghe MD, PhD, K. Vandepoele PhD, P. Vannuffel PhD, T. Venken PhD, K. Vermeulen PhD

SUMMARY

Molecular diagnostics have an increasing impact on diagnosis, risk stratification and targeted treatment in haemato-oncology. In the framework of a pilot study for the implementation of next-generation sequencing in the Belgian healthcare system, the Commission of Personalised Medicine was founded to give professional and evidence-based advice on the molecular analysis in haemato-oncology. This paper describes its recommendations for NGS analysis in myeloid malignancies. In addition, the minimally required set of genes that must be analysed is defined and algorithms for molecular workflow in myeloid malignancies are proposed.

(BELG J HEMATOL 2019;10(6):241–9)

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Causes, diagnosis and management of congenital or acquired neutropaenia

BJH - volume 10, issue 3, may 2019

S. van Hecke MD, P. Vandenberghe MD, PhD, A. Janssens MD, PhD

Neutropaenia is a common incidental finding on routine blood studies. This manuscript will focus on the possible causes, challenging differential diagnosis and appropriate management of neutropaenia. Different mechanisms may explain a decreased production, impaired development or increased destruction of neutrophilic granulocytes. We distinguish between congenital and acquired causes. The former includes benign ethnic neutropaenia, severe congenital neutropaenia and cyclic neutropaenia. For the latter, infections, drugs, auto-immune reactions, nutritional deficiencies as well as haematological malignancies are all possible reasons of neutropaenia. The risk of infection in those with non-chemotherapy-induced neutropaenia mainly depends on the bone marrow reserve. Asymptomatic patients with mild or moderate neutropaenia can be observed with serial blood counts at increasing intervals. Infections should always be treated according to the severity of neutropaenia. Therapy with growth factors, drug discontinuation and immunosuppressive therapy can be considered depending on the underlying cause.

(BELG J HEMATOL 2019;10(3):103–12)

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02 Unravelling the landscape of copy number aberrations in Hodgkin Lymphoma: a joint KU Leuven and Lysa study on circulating cell-free dna

BJH - volume 10, issue Abstract Book BHS, february 2019

J. Finalet Ferreiro , T. Tousseyn MD, PhD, O. Gheysens MD, PhD, G. Verhoef MD, PhD, M. André MD, PhD, P. Vandenberghe MD, PhD, L. Buedts , L.M. Fornecker , J. Lazarovici , R. Casasnovas , C. Copie , L. Marcelis MD, PhD

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