To update the ASCO guideline on use of hematopoietic colony-stimulating factors (CSFs) in patients with cancer.

Regenerative medicine is an evolving medical subspecialty dedicated to enhancing the body's natural healing mechanisms to repair or replace damaged tissues. By using autologous or allogeneic biologics, it offers the potential to restore function where conventional therapies have shown limited success. While this field holds great promise and continues to generate enthusiasm among both patients and clinicians, it remains in early stages of clinical validation. Therefore, it must be approached with careful optimism and responsible application, ensuring that its presentation, promotion, and use in clinical settings are grounded in evidence and ethical standards.

CMV is one of the main complications associated with transplantation, whether of solid organs or hematopoietic stem cells, and is related to an increase in post-transplant morbidity and mortality. Due to its significant impact, prevention is recommended, either through prophylaxis or other preventive therapies. The latter require viral monitoring of the patient, as viremia serves as a predictor of of the disease, allowing for an early detection and timely preventive treatment. The gold standard for viral monitoring is the quantitative determination of nucleic acids. Laboratories often face practical challenges regarding the type of sample to be used, its transport and storage conditions, the appropriate methods of nucleic acid extraction and quantification, the frequency of monitoring and establishment of cut-off points to ensure the accuracy and reproducibility of CMV viral load test results. The Argentine Society of Virology has formed a working group specifically dedicated to addressing these difficulties. This document aims to provide recommendations on the aforementioned points based on the available scientific evidence and the expertise of the working group.

Acute graft-versus-host disease (GVHDa) is one of the leading causes of morbidity and mortality after allogeneic hematopoietic stem cell transplant (HSCT) patients. While the first-line consensus treatment has been based on systemic corticosteroid therapy for many years, ruxolitinib has recently been approved and has become the standard second-line treatment. Nevertheless, the effectiveness of ruxolitinib remains limited to 40 % of cortico-resistant patients, raising the crucial question of selecting a third-line treatment. Among the therapeutic modalities described, this workshop selected fecal microbiota transplantation (FMT), mesenchymal stromal cells (MSC) injection, and extracorporeal photopheresis (ECP) as the most promising or with a benefit/risk balance that favors their prescription at this stage. The workshop also highlighted the importance of research aimed at identifying markers or score calculations that guide toward a risk-adapted approach as early as possible. To date, aside from calprotectin, no marker or score is routinely used, but all are the subject of intense research. Finally, measures associated with specific treatment remain crucial, and new developments in dietary contributions, infection prophylaxis, and tissue regeneration are also addressed.

This document is the tool through which the knowledge developed by biomedical research is transferred to daily clinical practice. It does not offer standards of care to which one can refer in acritical and decontextualized manner: these standards must be able to be expressed, for each individual case, on the basis of available clinical information, preferences of the patients and other contextual situations, accurately examined in light of the expertise of healthcare professionals. It is therefore up to the expertise and judgement of the professionals, who carefully listen to particular requests and consider the values expressed by patients, to establish which procedures or treatments are more appropriate to manage individual clinical cases.The Good Practice (GP) is organized in two large sections: a main section which, for each topic of interest, reports a short specific introduction and continues with recommendations for clinical practice in order to allow the reader to access the interpretation of the evidence and the recommendations made by the Authors. No. 4 Enclosures where the following is reported: Enclosure 1 - transfusion yield; Enclosure 2 - perioperative management and prevention of postoperative complications; Enclosure 3 - donation of blood, hematopoietic stem cells (HSCs), organs and tissues; Enclosure 4 - table of matching levels.

The aim of this article is to discuss alternatives to COBE2991® (Terumo BCT©) for the preparation of cell therapy products, in view of its planned cessation of commercialization in 2025. Cell therapy units need to find alternative methods in the face of regulatory restrictions and equipment obsolescence. Currently, COBE2991® (Terumo BCT©) is widely used in France, but its replacement will require multiple validations and adjustment of practices, as there is no single equivalent equipment. Recommendations for harmonizing these practices were discussed at a specific workshop in September 2024, following a survey of 25 French-speaking cell therapy centers.

How should fertility preservation in child and adolescent males receiving gonadotoxic therapies be managed?

In 2019, The Practice Guidelines Committee of the American Society for Transplantation and Cellular Therapy partnered with its Transplant Infectious Diseases Special Interest Group to update the 2009 compendium-style infectious disease guidelines for hematopoietic cell transplantation (HCT). The new format is now structured around frequently asked questions (FAQs), concise tables, and figures to better support clinical providers. Here, a panel of experts in HCT and infectious diseases identified relevant FAQs, which they graded based on the strength of clinical practice recommendations and the level of supporting evidence, as described herein. In the ninth set of guidelines in the series, the focus is on parainfluenza virus and human metapneumovirus, with FAQs addressing epidemiology, incidence, clinical manifestations, risk factors, diagnosis, prevention (including vaccines), and therapeutic management in recipients of HCT and chimeric antigen receptor T cells (cellular therapy). Special considerations for pediatric patients, unmet needs, and future research directions are conveyed in the guidelines.

At the 10th European Conference on Infections in Leukaemia (ECIL), the guidelines for antifungal prophylaxis in pediatric and adult patients with hematological malignancies (HM) were updated and some changes introduced. Regarding acute myeloid leukemia (AML) and myelodysplastic syndromes (MDS) patients undergoing remission induction chemotherapy, a B-II grading has been assigned to isavuconazole, micafungin, and caspofungin, based on non-randomized studies that have shown efficacy in preventing invasive fungal diseases (IFD). Regarding high-risk MDS patients treated with azacytidine, prophylaxis with posaconazole during the first four cycles of treatment is supported in the literature. Prophylaxis is not indicated in patients treated for myeloproliferative neoplasms (NPM), acute lymphoid leukemia (ALL), and Hodgkin lymphoma (HL). For patients with chronic lymphocytic leukemia (CLL) and non-Hodgkin lymphoma (NHL), prophylaxis is not generally indicated. For patients with multiple myeloma (MM), prophylaxis is not indicated and the limited epidemiological data available do not support the use of prophylaxis in subjects treated with bispecific antibodies. For patients undergoing allogeneic hematopoietic stem cell transplantation (HSCT), no substantial changes were made, apart from the addition of isavuconazole with grading B-II in the post-engraftment period. In patients undergoing auto-HSCT, antifungal prophylaxis is not indicated. Previous ECIL guidelines did not include CAR-T cells. The expert panel proposes to endorse the use of anti-mold prophylaxis in high-risk patients during pre-infusion and post-infusion, while in low-risk patients, anti-yeast prophylaxis can be recommended (B-II). For pediatric hematology patients, based on newly published data, caspofungin received a B-I grading as mold-active prophylaxis. Moreover, patients with ALL with insufficient treatment response during induction therapy, and children older than 12 y.o are now considered at high risk for IFD and are recommended to receive antifungal prophylaxis.

For patients with myelodysplastic neoplasm/syndrome (MDS), allogeneic hematopoietic cell transplantation (allo-HCT) represents the only potentially curative treatment, capable of eradicating disease-related mutant hematopoietic cells and establishing normal donor hematopoiesis. Biologic-assignment clinical trials have indicated that in eligible patients, allo-HCT is associated with superior clinical outcomes compared with nontransplant therapy. However, this therapeutic option is only available to a subset of patients, and the outcome is influenced by multiple factors inherent to the patient, the MDS subtype, and the allo-HCT procedure itself. In 2017, the European Society for Blood and Marrow Transplantation (EBMT) published recommendations for allo-HCT in MDS to guide practical decision making. In the contemporary era, genomic profiling has become routine clinical practice in many centers, and the most recent classification systems include MDS entities that are defined by genetic abnormalities. In particular, the molecular International Prognostic Scoring System offers more precise prognostication across all clinical end points and currently represents the standard tool for estimating patient survival in the absence of disease-modifying treatment. Evidence from multiple sources increasingly indicates that allo-HCT should be considered at the time of diagnosis in all eligible patients with MDS. Therefore, genomic profiling for somatic mutations and testing for germ line predisposition variants are integral to determining a patient's eligibility for transplantation. Although all patients with higher-risk MDS are potential candidates for immediate transplantation, a subset of those with lower-risk MDS may also derive benefit from this procedure at an earlier disease stage. Comprehensive recommendations on behalf of an expert international panel for clinical practice and future clinical studies of relevance are presented.

Paroxysmal nocturnal hemoglobinuria (PNH) is a rare acquired clonal disorder of hematopoietic stem cells characterized by intravascular hemolysis, bone marrow failure, and a high risk of thrombosis. Although PNH is a benign intravascular hemolytic disease, severe cases may be life-threatening. In recent years, remarkable progress has been made in the diagnosis and treatment of PNH, particularly in the field of complement inhibitor therapy. To further standardize and improve the diagnosis and treatment level of PNH in China, the Red Blood Cell Disease (Anemia) Group of the Chinese Society of Hematology updated the 2013 PNH Expert Consensus by combining the latest diagnosis and treatment progress of PNH, consulting relevant foreign guidelines/consensus and China's national conditions, and soliciting expert advice and opinions to formulate the Chinese Guidelines for the Diagnosis and Treatment of Paroxysmal Nocturnal Hemoglobinuria (2024) .

The advanced practice nurse (APN) has been introduced in France, following the 2016 health law and implementing decrees published in 2018. In this context, the French Society for Bone Marrow Transplantation and Cellular Therapy (SFGM-TC) has already issued guidelines regarding the allocation of APNs' new clinical competences and their collaboration with physicians. It is now providing new recommendations on the transversal activities that can be fulfilled by APNs, such as research, leadership, training and teaching. Additionally, the guidelines outline how APNs can cooperate with other professionals in departments of haematology and cellular therapy, including nurses, coordinators and health managers.

First-line treatments of autoimmune systemic diseases (ARD) are based on the use of various types of immunosuppressive or immunomodulatory drugs, either alone or in association, according to standardized reference protocols. Prolonged use of these drugs in severe or refractory ARD is associated with high morbidity and increased mortality. Innovative cell therapies represent a new promising approach for patients with ARDs, with the recent clinical use of: a) mesenchymal stromal cells (MSCs), based on their immunomodulatory, antifibrotic and pro-angiogenic properties and b) Chimeric Antigen Receptors (CAR) T cell therapies T lymphocytes, where genetically modified expression of a chimeric antigen receptor (CAR-T cells). Therapeutic use of MSC or CAR-T cells, remains indications of exception in patients with severe ARDs resistant to prior standard therapies with new prerequisite and organisation of health-care pathways as compared to traditional drugs, not only for the Cell and Gene Therapy (CGT) product definition and delivery process, but also for the patient clinical management before and after administration of the CGT product. The aim of this workshop under the auspices of the French Speaking Society of Bone Marrow and Cell transplantation (SFGM-TC) working group on autoimmune diseases (MATHEC) is to describe: a) the prerequisite for French hospitals to set-up the specific health-care pathways for MSC or CART therapy in ARDs patients, in accordance with regulatory and safety needs to perform academic or industry sponsored clinical trials, and b) the care-pathway for ARD patients treated with CGT, highlighting the importance of working in tandem between the ARD and the CAR-T cell specialist all along the indication, procedures and follow-up of ARDs. Patient safety considerations are central to guidance on patient selection to be validated collectively at the multidisciplinary team meeting (MDTM) based on recent (less than 3 months) thorough patient evaluation. MSC and CAR-T procedural aspects and follow-up are then carried out within appropriately experienced and SFGM-TC accredited centres in close collaboration with the ADs specialist.

Management of acquired aplastic anemia (AA) in emerging countries depends on the means of prognostic stratification, treatment and logistics available. During the 13th annual harmonization workshop of the francophone Society of bone marrow transplantation and cellular therapy (SFGM-TC), a designated working group reviewed the literature in order to elaborate unified guidelines for allogeneic hematopoietic cell transplantation (Allo-HCT) in this disease. In terms of practice, the conclusions are as follows; The use of anti-tymocyte globuline (ATG) is mainly from rabbit and very little from horse. Access to bone marrow graft, total body irradiation, and the international unrelated donor registries is limited, which justifies the use of peripheral blood stem cells, chemotherapy-based conditioning, and related alternative donor. The workshop recommends matched sibling allo-HCT in all patients aged less than 40 years with acquired severe or very severe AA. For patients aged over than 40 years, or who lack an HLA-identical donor, treatment with the combination of cyclosporin, horse ATG, eltrombopag or cyclosporine, eltrombopag is recommended. If horse ATG and eltrombopag are not available, matched sibling allo-HCT may be indicated as first-line therapy in patients aged between 40-60 years, and good performance status. Although, in patients who have failed immunosuppressive treatments and thrombopoietin agonists, and in the absence of HLA-matched donor, a haplo-identical allo-HCT with modified Baltimore conditioning is recommended.

These guidelines represent a GRADE-method revision of the recommendations produced by the Italian Society of Hemapheresis and Cell Manipulation (SIDEM) and the Italian Transplant Group for Bone Marrow Transplantation, Hematopoietic Stem Cells and Cell Therapy (GITMO) in 2013. Since 2013 several studies have been published that have strengthened the role of ECP in the management of GVHD. Thus, it was deemed appropriate to proceed with an update, with the aim to define uniform criteria for the application of ECP in adult and pediatric patients affected by GVHD throughout the national territory, in line with international guidelines, in maintaining of high standards of safety for patients and quality of the procedures provide. Post-HSCT GvHD therapies other than ECP and ECP therapy of other diseases, such as CTCL, are not covered by these guidelines.The development panel for this guideline includes professionals from various specialties who routinely interact in the management of the patient with GVHD, namely the transfusionist, the adult and pediatric hematologist, and the hospital pharmacist. A hematologist experienced in systematic reviews and GRADE guideline development ccordinated the development process, and an experienced transfusionist coordinated the assignment of tasks and reporting. External reviewers of the guideline included a patient representative.

Allogeneic transplantation of haematopoietic stem cells is still the only curative treatment for certain haematological malignancies. This treatment can be responsible for a number of side-effects, leading to multiple and interdependent physical and psychological deficiencies that affect patients' quality of life and social participation, and can be experienced as a handicap, sometimes for several years after the transplant. For several years now, the integration of post-transplant rehabilitation pathways has been becoming more widespread, and initiatives to provide multidisciplinary care at an increasingly early stage are being studied. The aim of this early management is to improve the patient's overall functional state before, during and after the transplant, in order to limit the impact of the treatment and ensure the quickest possible return to a life that is as satisfying as possible. The international literature and the experiments carried out throughout the French-speaking world describe heterogeneous practices. Based on this literature and experience, the aim of this study is to issue homogenous recommendations for good clinical practice and to identify areas for further research into pre-transplant, per-transplant and post-transplant rehabilitation of haematopoietic stem cells.

Transcranial brain parenchyma sonography (TCS) has been recommended as a tool for the early and differential diagnosis of Parkinson's disease (PD) in German and European clinical guidelines. Still, the brain structures to be examined for the diagnostic questions and the requirements for being a qualified investigator were not specified in detail. These issues have now been addressed in the 2023 update of the clinical guideline on PD by the German Society of Neurology (DGN).

Treatment of pediatric high-risk acute myeloid leukemia (AML), defined either on molecular or cytogenetic features, relies on bone marrow transplant after cytologic remission. However, relapse remains the first post-transplant cause of mortality. In this 13th session of practice harmonization of the francophone society of bone marrow transplantation and cellular therapy (SFGM-TC), our group worked on recommendations regarding the management of post-transplant relapse in AML pediatric patients based on international literature, national survey and expert opinion. Overall, immunomodulation strategy relying on both measurable residual disease (MRD) and chimerism evaluation should be used for high-risk AML. In very high-risk (VHR) AML with a 5-year overall survival ≤30 %, a post-transplant maintenance should be proposed using either hypomethylating agents, combined with DLI whenever possible, or FLT3 tyrosine kinase inhibitors if this target is present on leukemia cells. In the pre-emptive or early relapse settings (< 6 months post-transplant), treatments combining DLI, Azacytidine and Venetoclax should be considered. Access to phase I/II trails for targeted therapies (menin, IDH or JAK inhibitors) should be discussed in each patient according to the underlying molecular abnormalities of the disease.

Conditioning regimen prior to hematopoietic stem cell transplantation have an impact on patient fertility through the use of gonadal irradiation and/or bifunctional alkylating agents. Their impact on fertility depends mainly on the dose used and, in women, on age at the time of treatment. All patients should benefit before treatment from a consultation informing them of the potential impact on fertility and of fertility preservation techniques. In the absence of contraindications, the major toxicity of myeloablative conditioning regimen justifies fertility preservation. There are few data concerning fertility after reduced-intensity conditioning. Despite lower theoretical gonadotoxicity, we also recommend fertility preservation, if possible before transplantation. The fertility preservation techniques used depend on the patient's age, pathology and conditioning. In the event of subsequent use of harvested gonadal tissue in the context of acute leukemia or aggressive lymphoma, it is advisable to assess the risk of reintroduction of tumor cells. Finally, it is recommended to assess gonadal function after transplant, especially after reduced conditioning. If there is persistent residual gonadal function, post-treatment fertility preservation should be discuss.

Acquired aplastic anemia (AA) is a rare heterogeneous disorder characterized by pancytopenia and hypoplastic bone marrow. The incidence is 2-3 per million population per year in the Western world, but 3 times higher in East Asia. Survival in severe aplastic anemia (SAA) has improved significantly due to advances in hematopoietic stem cell transplantation (HSCT), immunosuppressive therapy, biologic agents, and supportive care. In SAA, HSCT from a matched sibling donor (MSD) is the first-line treatment. If a MSD is not available, options include immunosuppressive therapy (IST), matched unrelated donor, or haploidentical HSCT. The purpose of this guideline is to provide health care professionals with clear guidance on the diagnosis and management of pediatric patients with AA. A preliminary evidence-based document prepared by a group of pediatric hematologists of the Bone Marrow Failure Study Group of the Italian Association of Pediatric Hemato-Oncology (AIEOP) was discussed, modified and approved during a series of consensus conferences that started online during COVID 19 and continued in the following years, according to procedures previously validated by the AIEOP Board of Directors.