R. Callens MD

Practical recommendations for the management of late relapsed and refractory multiple myeloma in 2024

SUMMARY

Despite significant advances in therapeutic modalities, the treatment of relapsed and refractory multiple myeloma (RRMM) is still challenging. In this publication, we aim to provide an update on therapeutic modalities for RRMM in Belgium. First, novel combinations of well-established therapeutic agents will be discussed. Second, T-cell redirection therapies will be addressed. These include bispecific antibodies, both anti-BCMA x CD3 and anti-GPRC5D x CD3, as well as chimeric antigen receptor (CAR) T cell therapy. Third, we discuss novel modalities such as antibody-drug conjugates, selinexor, venetoclax, melflufen and CELMoDs. Finally, a general flowchart regarding overall treatment sequencing will be proposed, providing an integrated treatment recommendation from frontline to relapse.

(BELG J HEMATOL 2024;15(6):225–32)

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VEXAS-syndrome: A state of the art

SUMMARY

VEXAS (Vacuoles, E1 enzyme, X-linked, Autoinflammatory, Somatic) syndrome is an acquired, late-onset disorder, almost exclusively described in male patients. This new clinical entity is associated with autoinflammation and haematological abnormalities, such as Myelodysplastic Syndrome, Monoclonal Gammopathy of Undetermined Significance (MGUS) and Multiple Myeloma (MM). Common laboratory abnormalities are chronic inflammation, macrocytic anaemia, thrombocytopenia and lymphopenia. The diagnosis is genotype-based by the identification of myeloid-restricted somatic mutations in the UBA1 gene, exclusively found on the X-chromosome. A bone marrow aspirate and trephine biopsy are crucial in the diagnostic work-up, demonstrating the typical finding of vacuoles. Clear scientific support comparing different treatment strategies in VEXAS syndrome is still lacking. Currently, corticosteroid treatment remains the cornerstone in the control of inflammatory flare-ups. Corticosteroid-sparing regimens such as methotrexate, tumour necrosis factor inhibitors, anti-interleukine-6, and anti-interleukine-1 agents have only been able to demonstrate a short-term response. While an allogeneic haematopoietic stem cell transplantation (allo-HSCT) seems to be the only long-lasting curative treatment to eradicate the causing pathogenic UBA1 clones, ideal candidate selection and timing for allo-HSCT remain unclear. Recently, some case reports have demonstrated promising results when integrating the use of hypomethylating agents or ruxolitinib in the treatment of patients with VEXAS syndrome. As VEXAS syndrome remains a fatal disease with a mean 5-year mortality of up to 40%, clinicians should be aware of its existence, clinical work-up and possible treatment strategies.

(BELG J HEMATOL 2023;14(6):236–44)

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Adolescents/young adults and their caregiver on the onco-haematology ward

SUMMARY

In Belgium, over 1,800 adolescents and young adults (AYAs) aged 15–39 are diagnosed annually with cancer. Of all yearly new cancer diagnoses in Belgium, AYA cancers are rare because they are rare in absolute numbers, or because they are rare examples of common cancers occurring outside of the usual age range. Leukaemia and lymphoma’s represent the most common AYA haematological cancers among the AYA population. Apart from the treatment(s) of cancer, the specific needs of young people with haematological malignancies are defined as much, or more, by their age and developmental stage as their life-threatening disease. In June 2018, an AYA interest group under the guidance of “Kom op tegen Kanker” published a blueprint for age-specific care for young people with cancer to highlight the current and future needs of AYA specific cancer care. Current healthcare professional education, training programs and healthcare settings do not address AYA-specific issues. Cure and care is currently exclusively approached from paediatric or adult care perspective. This compartmentalised approach to cancer care can result in a blind spot for AYA comprehensive age developmental cancer care for youngsters and their caregivers. Between the current paediatric and adult silos of care, there is an unmet need for comprehensive AYA cancer care. This care should focus on specific topics to support young people with haematological cancer during treatment, into survivorship care or with early integration of palliative care, providing comprehensive support for AYA patient with limited-life expectations.

(BELG J HEMATOL 2020;11(7):275-81)

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Big Children or Small Adults? Leukaemia Treatment in Adolescence and Young Adulthood

SUMMARY

The outcome of adolescents and young adults (AYAs) with acute lymphoblastic leukaemia (ALL) has improved dramatically over the last decades by using paediatric and paediatric-inspired protocols in this age group. The outcome of different paediatric, paediatric-inspired and adult-based regimens are compared in this review. Despite pre-existing fear among clinicians to use these high-intensity paediatric regimens in AYAs, toxicities seem manageable, with treatment-related mortality comparable to that seen with adult protocols. In paediatric protocols, the use of allogeneic stem cell transplantation is restricted to certain high-risk groups and prophylactic cranial irradiation is omitted. In recent years, evaluation of minimal residual disease is increasingly used as prognostic marker and as a tool to guide therapy. In Philadelphia-positive ALL, the use of tyrosine-kinase inhibitors has completely changed prognosis and therapeutic decisions.

(BELG J HEMATOL 2020;11(3):88–97)

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P.21 Disseminated nocardiosis in a patient with multiple myeloma treated with bortezomib, thalidomide and dexamethasone

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