Haematological malignancy: are we measuring what is important to patients? A systematic review of quality of life instruments

© 2018 The Authors. This is an open access article under the terms of the Creative Commons Attribution‐NonCommercial License, which permits use, distribution and reproduction in any medium, provided the original work is properly cited and is not used for commercial purposes.The wide range of health‐related quality‐of‐life (HRQoL) instruments used in haematology makes it challenging for haematologists and other care team members in practice to select, use and understand the scoring system and finally interpret the results. The main objectives of this study were to: (a) provide a comprehensive list of quality‐of‐life issues important to patients suffering from haematological malignancies, identified through the literature; (b) provide a list of health‐related quality‐of‐life (HRQoL) instruments used in haematological malignancies in both daily clinical practice and research; and (c) evaluate the relevance and comprehensibility of the identified instruments in haematological malignancies. Systematic literature review of two databases, followed by addition of articles by manual searching, was carried out. The articles focusing on the primary studies, which have used semi‐structured/structured interviews or surveys to identify issues important to HM patients, and other studies describing the results of testing measurement properties, such as reliability, validity and responsiveness of the instruments currently used to evaluate the HRQoL in different HMs, were included. Fifty‐seven studies reported development and validation of 30 HRQoL instruments, which have been used in haematology. Twenty‐four studies were identified using qualitative methods to report HRQoL issues and symptoms from a patient's perspective. No identified instrument captured all the issues identified from the qualitative studies. None of the instruments reviewed appeared to have been developed for use in clinical practice and specifically for patients with HM, except MyPOS. Furthermore, measurement properties were established, largely, in clinical trial scenarios. There is a need for development of a new HRQoL instrument entirely based on involvement of patients with haematological malignancies.Peer reviewe

Multiple Myeloma : an update on disease biology and therapy

Multiple myeloma is a malignancy of immunoglobulin producing plasma cells. Clinical features include bone pain due to lytic bone lesions or pathological fractures, anemia, symptomatic hypercalcemia, renal insufficiency, recurrent infections and amyloidosis. In the last few years, there have been considerable advances in the understanding of the biology of this disease. While multiple myeloma is biologically diverse, several oncogenes are activated in this illness. In addition, the role of the bone marrow microenvironment to support the growth and survival of the malignant cells has been well described. In this review, we discuss recent developments in the molecular pathogenesis of myeloma. These recent observations are being translated into novel therapeutic approaches that target both the tumor cell as well as the stroma. Current therapeutic strategies are discussed.peer-reviewe

Role of Allogeneic Transplantation in Multiple Myeloma in the Era of New Drugs

High-dose melphalan with autologous stem cell rescue has been regarded as the standard of care for patients with newly diagnosed myeloma up to the age of 65–70 years. The recent development of agents with potent anti-tumor activity such as thalidomide, lenalidomide and bortezomib has further improved overall survival and response rates. However, relapse is a continuous risk

Mesenchymal stem cells and their use as cell replacement therapy and disease modelling tool.

Mesenchymal stem cells (MSCs) from adult somatic tissues may differentiate in vitro and in vivo into multiple mesodermal tissues including bone, cartilage, adipose tissue, tendon, ligament or even muscle. MSCs preferentially home to damaged tissues where they exert their therapeutic potential. A striking feature of the MSCs is their low inherent immunogenicity as they induce little, if any, proliferation of allogeneic lymphocytes and antigen-presenting cells. Instead, MSCs appear to be immunosuppressive in vitro. Their multilineage differentiation potential coupled to their immuno-privileged properties is being exploited worldwide for both autologous and allogeneic cell replacement strategies. Here, we introduce the readers to the biology of MSCs and the mechanisms underlying immune tolerance. We then outline potential cell replacement strategies and clinical applications based on the MSCs immunological properties. Ongoing clinical trials for graft-versus-host-disease, haematopoietic recovery after co-transplantation of MSCs along with haematopoietic stem cells and tissue repair are discussed. Finally, we review the emerging area based on the use of MSCs as a target cell subset for either spontaneous or induced neoplastic transformation and, for modelling non-haematological mesenchymal cancers such as sarcomas

Role of autologous and allogeneic stem cell transplantation in myeloma.

The treatment of multiple myeloma (MM), a largely incurable B-cell hematologic malignancy, is changing dramatically. Autologous stem cell transplantation (SCT) and the approval of two new classes of drugs, immunomodulators and proteosome inhibitors, have resulted in improved response rates and increased overall survivals. Thalidomide, bortezomib and lenalidomide have been combined with corticosteroids, alkylators and anthracyclines in front-line MM treatment. Phase 2 and preliminary phase 3 studies have reported very high response rates and complete response rates formerly seen only with SCT. When patients with MM who have received these new drugs then proceed to transplant, major response rates are further increased. Owing to limited follow-up, it is unclear whether these higher response rates translate into increased survival. Despite these improvements, the disease remains incurable for all but a small fraction of patients. Allogeneic SCT is potentially curative, due in part to a graft-versus-myeloma effect but is limited by mortality. Mortality can be reduced through the use of lower intensity conditioning regimens but this comes at a cost of higher rates of disease progression and relapse. Strategies to improve outcomes of allogeneic transplants include more intensive, yet non-myeloablative conditioning regimens, tandem transplants, peripheral blood cells, graft engineering, post-transplant maintenance and targeted conditioning therapies