Global characteristics and outcomes of autologous hematopoietic stem cell transplantation for newly diagnosed multiple myeloma: A study of the worldwide network for blood and marrow transplantation (WBMT).

Autologous hematopoietic cell transplantation (AHCT) is a commonly used treatment in multiple myeloma (MM). However, real-world global demographic and outcome data are scarce. We collected data on baseline characteristics and outcomes from 61 725 patients with newly diagnosed MM who underwent upfront AHCT between 2013 and 2017 from nine national/international registries. The primary endpoint was overall survival (OS), and the secondary endpoints were progression-free survival (PFS), relapse incidence (RI) and non-relapse mortality (NRM). Median OS amounted to 90.2 months (95% CI 88.2-93.6) and median PFS 36.5 months (95% CI 36.1-37.0). At 24 months, cumulative RI was 33% (95% CI 32.5%-33.4%) and NRM was 2.5% (95% CI 2.3%-2.6%). In the multivariate analysis, superior outcomes were associated with younger age, IgG subtype, complete hematological response at auto-HCT, Karnofsky score of 100%, international staging scoring (ISS) stage 1, HCT-comorbidity index (CI) 0, standard cytogenetic risk, auto-HCT in recent years, and use of lenalidomide maintenance. There were differences in the baseline characteristics and outcomes between registries. While the NRM was 1%-3% at 12 months worldwide, the OS at 36 months was 69%-84%, RI at 12 months was 12%-24% and PFS at 36 months was 43%-63%. The variability in these outcomes is attributable to differences in patient and disease characteristics as well as the use of maintenance and macroeconomic factors. In conclusion, worldwide data indicate that AHCT in MM is a safe and effective therapy with an NRM of 1%-3% with considerable regional differences in OS, PFS, RI, and patient characteristics. Maintenance treatment post-AHCT had a beneficial effect on OS.

[Haematopoietic stem cell donation from minor donor: Respecting laws, assessing fitness, delivering information and good care (SFGM-TC)]. / Don de cellules souches hématopoïétiques à partir d'un donneur mineur : cadre réglementaire, information, évaluation de l'aptitude au don et parcours (SFGM-TC).

Haematopoietic stem cell collection from paediatric donors is a common and life-saving practice, as evidenced by the fact that there is a growing annual number of cases of transplants from minor donors among SFGM-TC centers over the last decade. Still, medical use of human tissue from a healthy and underage donor requires proper regulations and medical management. The guidelines below aim at underlining the importance of pondering the legal, medical and ethical aspects of using stem cells from healthy paediatric donors and stress out the importance of obtaining informed consent at the time of assessing HLA compatibility. Combined medical and psychological assessments are required before the donation, as well as one month later and one year later to ensure of the child's physical and mental wellbeing. Bone marrow harvest under general anaesthetics remains the preferred method of collection for children. Peripheral blood stem cell collection should only be considered for children who will not require a central venous access for collection. We aim at offering guidelines centered on the healthy child donating stem cells and his/her wellbeing, and these should be regularly reviewed as medical practices evolve.

[Pediatric aphereses (workshop SFGM-TC)]. / Cytaphérèses pédiatriques (atelier SFGM-TC).

Practice of pediatric aphereses - in particular when caring for low-weight children - differs from the practice of adult aphereses, since pediatric aphereses represent low numbers of procedures, which has practical implications in terms of practical training and retraining for involved healthcare personnel, as needed for habilitation and validation of ongoing competencies. A specific training is mandatory in order to ensure both the child and the staff safety during and after collection, as well as ensure high quality of the collected cell product and that its meets predefined specifications that depend on its intended use. Low and very low-weight children deserve a particular attention for a number of procedural and clinical aspects: the nature and quality of venous accesses to ensure proper operation of the cell separator, management of hemodynamic fluctuations in relation with the relative importance of the extracorporeal blood volume as compared to the total blood volume of the child, risks and clinical manifestations of citrate toxicity, minimization of stress during the procedure that may include but is not limited to pharmacological sedation. The full spectrum of competencies needed to deal with these aspects is rarely present within a single team of healthcare professionals; it most often requires the tight combination of expertise drawing from the collection facility, the pediatric department and possibly the pediatric intensive care unit ward, whether from the same or from different institutions. Interactions must be formalized in a document that accurately describes which category of actors is responsible for each category of acts (prescriptions, decisions), depending on their initial qualifications, specific competencies, and affiliations.

Successful Allogeneic Peripheral Blood Stem Cell Transplantation in 4 Wiskott-Aldrich Syndrome Patients.

BACKGROUND: Allogeneic hematopoietic stem cell transplantation is a potential curative treatment in Wiskott-Aldrich syndrome (WAS). Here, we analyzed the outcomes in 4 WAS patients who underwent this procedure with peripheral blood stem cell (PBSC) in our center. PATIENTS AND METHODS: Four patients with severe WAS phenotype have received allogeneic hematopoietic stem cell transplantation between January 2014 and December 2019 from matched sibling donors with PBSC. Two different preparative conditioning regimens were provided: the first associated busulfan-cyclophosphamide (2 patients) and the second with busulfan-fludarabine administered to the others. Cyclosporine gave as preferred graft-versus-host disease prophylaxis with a short course of methotrexate. RESULTS: All patients achieved engraftment after PBSC with a median CD34+ cell count: 13.6×106/kg (8 to 24.9×106/kg). Chronic graft-versus-host disease developed in 2 patients treated by cyclosporine-steroids with complete resolution. Chimerism for all the patients was fully donor (>95% donor). After a median follow-up of 41 months (8 to 74 mo), all patients (100%) are alive, healthy, with complete clinical, immunologic, and hematologic recovery, without signs of WAS. CONCLUSION: This limited study with high-dose PBSC transplantation approach for WAS, demonstrated a safe and effective treatment option, with rapid engraftment, without complications, excellent long-term outcomes, independent of conditioning regimen.

Peripheral Blood Stem Cell Mobilization and Collection in Pediatric Healthy Sibling Donors Weighing 20 Kilograms or Less; Algerian Experience.

Peripheral blood stem cells (PBSC) are the source of allogeneic hematopoietic stem cell transplants currently used for malignant and non-malignant hematological diseases. PBSC harvest may be difficult in young children who are donors. Extracorporeal separator line priming by red blood cells or albumin alone is usually required to improve haemodynamic tolerance and efficacy of collection. We present our experience with 29 children weighing 20 kg or less mobilised between January 2005 and June 2018. The median age and weight at the time of apheresis were 5 years and 18 kg, respectively. A total of 54 PBSC were performed. The median cell yield per apheresis was 5.9 × 106 CD34+cells/kg (2,5-13,9) recipient body weight (RBW). Despite their low weight, insertion of a femoral catheter was avoided in 58.6% of children. Nineteen donors required 2 or 3 apheresis sessions without any major complication. Twenty-nine pts with hemopathies have successfully benefited from PBSC except one case of rejection with aplastic anemia.

The Global State of Hematopoietic Cell Transplantation for Multiple Myeloma: An Analysis of the Worldwide Network of Blood and Marrow Transplantation Database and the Global Burden of Disease Study.

Multiple myeloma (MM) is a plasma cell neoplasm characterized by destructive bony lesions, anemia, and renal impairment. Access to effective therapy is limited globally. We report the rates and utilization of hematopoietic cell transplantation (HCT) globally from 2006-2015 to better characterize access to HCT for patients with MM. This was an analysis of a retrospective survey of Worldwide Network of Blood and Marrow Transplant sites, conducted annually between 2006-2015. Incidence estimates were from the Global Burden of Disease study. Outcome measures included total number of autologous and allogeneic HCTs by world regions, and percentage of newly diagnosed MM patients who underwent HCT, calculated by the number of transplants per region in calendar year/gross annual incidence of MM per region. From 2006 to 2015, the number of autologous HCT performed worldwide for MM increased by 107%. Utilization of autologous HCT was highest in Northern America and European regions, increasing from 13% to 24% in Northern America, and an increase from 15% to 22% in Europe. In contrast, the utilization of autologous HCT was lower in the Africa/Mediterranean region, with utilization only changing from 1.8% in 2006 to 4% in 2015. The number of first allogeneic HCT performed globally for MM declined after a peak in 2012 by -3% since 2006. Autologous HCT utilization for MM has increased worldwide in high-income regions but remains poorly utilized in Africa and the East Mediterranean. More work is needed to improve access to HCT for MM patients, especially in low to middle income countries. © 2020 American Society for Transplantation and Cellular Therapy. Published by Elsevier Inc.

Two decades of experience in a combined adult/pediatric allogeneic hematopoietic stem cell transplantation center in Algiers, Algeria.

Hematopoietic stem cell transplantation (HSCT) has evolved from an experimental to a successful treatment modality reaching worldwide 80.000 HSCT/year. Distribution and trends of HSCT, however, remain heterogeneous. Activities range from none to more than 511/10 million population between countries and regions. Here, we report on a successful autologous and allogeneic HSCT program for adult and pediatric patients started two decades ago in Northern Africa. From 1998 to December 2017, a total of 2828 HSCT was performed of which 2059 were allo-HSCT (1474 adults and 585 children). The activities were analyzed according to indication, donor type, stem cell source, and trends over time. There was a significant difference in indications according to age. Adult patients were transplanted more often for hematological malignancies. In children, the indications were distributed equally between malignant and non-malignant diseases. Overall activities increased substantially in AML and to a lower extent in ALL and CLL despite sharp reduction of activity in CML after 2005. Finally, a higher transplantation rate (33/10 million population) was reached as compared to most regions of the world except Europe and USA/Canada. Overall survival in children with AML was 56.0% at 15 years, in adults 61.3% at 5 years, and in patients with CML 55.5% at 15 years without difference between reduced intensity condition (RIC) and myeloablative conditioning (MAC). Patients with Ph+ ALL had the lowest survival reaching 26.7% at 5 years. Highest survival was observed in patients with aplastic anemia, Fanconi anemia, and thalassemia reaching 77.3%, 73.5%, and 75.7% at 15 years respectively. Long distances and late referral remain a challenge for this large country.

Special report: Summary of the first meeting of African Blood and Marrow Transplantation (AfBMT) group, Casablanca, Morocco, April 19-21, 2018 held under the auspices of the Worldwide Network for Blood and Marrow Transplantation (WBMT).

The first meeting of the African Blood and Marrow Transplantation (AfBMT) was held in Casablanca from April 19, 2018 to April 21, 2018, with the aim of fostering hematopoietic stem cell transplantation (HSCT) activity in Africa. Out of the 54 African countries, HSCT is available only in six (Algeria, Egypt, Morocco, Nigeria, South Africa, and Tunisia). During this meeting, African teams and international experts from the Worldwide Network for Blood and Marrow Transplantation (WBMT) gathered to share their experience and discussed ways to help fill the gap. Nurses and patients held their meeting in parallel. International support and collaboration can help by providing expertise adapted to local resources and regional population needs. Local engagement including government and private participants are necessary to initiate and develop local HSCT capability.

[Perception of doctors in different specialties of iron deficiency and iron deficiency anemia in Algeria in 2016: the SUPFER DZ survey]. / Perception de la carence martiale et de l'anémie ferriprive par les médecins de différentes spécialités en Algérie en 2016: enquête SUPFER DZ.

INTRODUCTION: diagnostic methods and management of iron deficiency and iron deficiency anemia in clinical practice in Algeria is poorly known. METHODS: we conducted a cross-sectional survey among doctors in different specialties treating patients with iron deficiency anemia in 2016. RESULTS: data analysis was based on 349 questionnaires which were validated (anesthesia/resuscitation: 39; obstetrics and gynaecology: 111; oncology/Hematology: 71; hepato-gastroenterology: 64; cardiology: 36; internal medicine: 28). All specialties combined, 73% (254/349) of physicians thought that at least 30% of their patients had iron deficiency anemia; 65% of physicians (226/349) thought that at least 30% of their patients had iron deficiency. Iron deficiency was investigated systematically by 57% (63/111) of physicians of the group obstetrics and gynaecology, but only by 11% (26/238) of the remaining doctors; indeed, 82% (195/238) of physicians investigated it only in patients with anemia. The assessment of iron deficiency showed that the hemoglobin (Hb) was almost always determined (89%; 310/349) while laboratory tests to explore iron metabolism were inadequate: 70% (244/349) of physicians performed serum ferritin test and only 37% (128/349) performed transferrin saturation. Patients with iron deficiency (with or without anemia) received oral iron therapy (prescribed by 92% (322/349) of physicians) and iron injections therapy depending on Hb level (prescribed by 36% (127/349) of physicians). CONCLUSION: this survey shows that iron deficiency is evaluated only in patients with anemia. In particular, laboratory tests to measure iron deficiency are insufficiently prescribed.