Summary:The severe phenotype of leukocyte adhesion deficiency is a rare, congenital disorder of leukocyte function that is usually fatal in the first few years of life. Allogeneic hematopoietic stem cell transplantation currently offers the only curative approach for this disease. We describe the first successful matched unrelated donor bone marrow transplant in an infant with leukocyte adhesion deficiency. Keywords: leukocyte adhesion; marrow transplant; unrelated Leukocyte adhesion deficiency type I (LAD-I) is a rare inherited immunodeficiency caused by a mutation in the gene encoding the beta-2 integrin, CD18. This adhesive protein, defined by a specific monoclonal antibody anti-CD18, is expressed by leukocytes, and is shared by three adhesive heterodimers, LFA-1, Mac-1 (CR3) and p150, 95.1 The disease is characterized by an inability of phagocytic cells to adhere to endothelial cells and thereafter to migrate to sites of infection.2 The disease can present as a severe or moderate phenotype based on the degree of expression of the adhesion molecules. In the severe phenotype, beta-2 integrins are expressed on fewer than 1% of leukocytes. Patients with the severe form of LAD-I usually die of infection before the age of 2 years. 3 In the moderate phenotype, beta-2 integrins are expressed on 10-30% of leukocytes. Although the quality of life is usually poor in the moderate phenotype, life expectancy can reach the fourth decade.1,4 Allogeneic hematopoietic stem cell transplantation is the only therapeutic option known to cure LAD. There have been several reports of successful matched and mismatched related stem cell transplants in patients with LAD, including a cord blood transplant from a histocompatible sibling. [3][4][5] We report the first successful matched unrelated donor BMT in this disease. Case reportThe patient was the first child of a non-consanguinous Mexican-American couple born at full term by spontaneous vaginal delivery. She was hospitalized at 1 week of age for omphalitis and severe diaper dermatitis which resolved during a 2 week treatment course with intravenous antibiotics. She was readmitted at 24 days of age with fever and irritability; her white blood cell count on admission was 62 600 with a left shift; despite broadspectrum antibiotic therapy, her condition deteriorated; she developed increasing abdominal distention with persistent fever and a rise in her WBC to 78 000/mm 3 . Pseudomonas aeruginosa and Enterococcus spp. were isolated from a perianal lesion. She was treated with intravenous antibiotics for her presumed peritonitis and perianal abscess but developed cellulitis at an intravenous catheter site in her left foot. P. aeruginosa was isolated from the fluid around her cellulitis. She went on to develop P. aeruginosa line sepsis and osteomyelitis of the left metatarsal bones. An immunologic evaluation revealed normal immunoglobulin G, A, M and E levels, normal C3, C4 and CH50, normal proportions of T and B cells and lymphocyte subsets. On flow cytometric evaluation, less than 1% of...

Bone marrow (BM) aspiration plays an important role in hematologic malignancies diagnosis. Access and cost of diagnostic flow cytometry remains a problem in low and middle-income countries. In this context, morphological diagnosis by BM smear often represents the only means to rapidly diagnose our patients. Therefore, in this context obtaining the highest quality sample possible during the procedure is paramount. Despite being a well-known problem, evidence-based recommendations to improve BM aspirate quality are few, with studies evaluating factors associated with poor quality samples lacking. Objectives To determine factors associated with poor quality BM aspirates defined by an aspicular or hemodiluted sample in a hematology referral center. Materials and methods We conducted a retrospective study in our University Hospital and analyzed the BM smear samples stored in our center performed from October 2014 to December 2018. We collected and analyzed data based on diagnosis, age, gender, recent chemotherapy, and the variables of a complete blood count performed just before each BM aspiration. The quality of the BM smear was defined in any of the following: aspicular (without spicules), pauciaspicular (1-3 spicules), spicular (> 3 spicules), defining aspicular BM smear as non-diagnostic samples. Univariate analysis was performed looking for diferences between operators (in a 3-year residency program). In the other hand, in the multivariate analysis we seek to reveal the factors associated with obtaining hemodiluted (aspicular) bone marrow aspirate-smears. Results A total of 1,073 BM aspirates were evaluated. Hematology fellows performed 97% of BM aspirates; the remaining 3% were performed by attending physicians. In our analysis, 301 aspirates were aspicular, constituting 28.1% of the total number of aspirate smears. Most BM aspirates were performed for a diagnostic evaluation (66.3%) with the rest of the procedures for subsequent hematologic malignancy response assessments. In the univariate analysis, no differences were observed between operators. In a multivariate analysis the presence of an age >65 years (OR 3.1, 95% CI 2.3 to 4.1) and hemoglobin <6.0 g/dL (OR 2.7, 95% CI 1.4 to 4.5) at the time of the procedure were significantly associated to obtaining a non-diagnostic sample. Diagnosis, WBC count, platelet count, operator experience or other variables did not show statistical relevance. In our center, 18.81% of diagnostic patient samples that had acute leukemia were diagnosed without flow cytometry and through BM aspirate morphology alone. A second procedure to reach a diagnosis was necessary in 7.97% of the patients due to aspicular samples, obtaining a mean of diagnostic-treatment delay of 18.3 days (±5.7 days). Conclusions We found no differences between operators, emphasizing that there are other factors to consider in addition to a correct BM aspiration technique or operator experience. We believe this is crucial to recognize, especially in developing countries where morphological diagnosis remains the only means for the diagnosis or response evaluation of our patients. BM aspirate sample quality is multifactorial, being age and hemoglobin important factors. In addition, obtaining aspicular or hemodiluted samples represents not only a diagnostic challenge, but also delays the treatment of our patients. Disclosures Gomez-Almaguer: Celgene: Consultancy, Speakers Bureau; Takeda: Consultancy, Speakers Bureau; Teva: Consultancy, Speakers Bureau; Amgen: Consultancy, Speakers Bureau; Janssen: Consultancy, Speakers Bureau.

Introduction. Hematopoietic stem cell donors (HSCD) frequently suffer from anxiety and feelings that are usually unnoticed by the physician o their relatives. The origin is multifactorial: fear of pain, fear of the procedure, adverse effects of growth factor, feelings of responsibility regarding the success of the transplant, family pressure, etc. HSCD could show ambivalence of feelings about the donation, which may be related to the moral obligation to help a sick relative and fear of the donation procedure, thus originating anxiety and moral distress (MD). We evaluated the incidence of MD, anxiety, and other symptoms in a group of HSCD regarding donor related allogeneic transplantation. Materials and methods. A prospective observational study was carried out through a survey applied to 60 consecutive HSCD from April, 2014 to January, 2017. Protocol was approved by Institutional Ethics Review Committee and was carried out according to the Declaration of Helsinki. HSCD completed 3 written self-answered questionnaires (Questionnaire to assess moral distress (MDQ) elaborated by the researcher, State Trait Anxiety Index (STAI®) and Edmonton Symptom Assessment System (ESAS)) in three stages: right before initiating stem cell mobilization, immediately before the apheresis procedure, and 24 hours after the donation. Cell mobilization was done with granulocyte colony-stimulating factor (G-CSF) 10 µg/kg/day/4 days while the apheresis collection was performed on the fifth day as an outpatient basis in all cases. Results. Sixty donors, 36 male and 24 female with a mean age of 38.2 years (SD = 13.6) were included. HSCD was a brother/sister of the patient in 39 cases (65%), and one parent in 21 cases. Regarding donor educational level, only 24 (40%) donors had university level. MDQ: Most donors felt happy to be the donor (97%), but 28% said they were not given the opportunity to accept or refuse being the donor, 10% would have preferred that another person had been the donor, and 5% mentioned that donation worsened their family relationship and they would not donate cells again if necessary. The number of donors with negative feelings regarding the donation decreased through the 3MD questionnaires (Table 1). STAI: In the first, second, and third questionnaire, the mean score for the state of anxiety was 12.1 (SD = 7.6), 10.3 (SD = 6.6), and 7.9 (SD = 5.7), respectively. In first STAI, mild to moderate anxiety was observed in 33% of the donors and severe anxiety was observed in 7%, observing a higher score in the first questionnaire, just before starting cell mobilization; however, that score gradually decreased in the following questionnaires. ESAS: In the first questionnaire, although no procedure was still performed, all donors had symptoms, including insomnia (33%), anxiety (33%), and tiredness (30%). In the second ESAS questionnaire, symptoms worsened, mainly pain (78%), tiredness (63%), and insomnia (55%), which was probably related to G-CSF. In the third ESAS questionnaire, there was a significant decrease in the symptoms: pain (60%), tiredness (50%), and discomfort (40%). The average global score for 10 symptoms evaluated was 10.5 (SD = 14.2), 20.1 (SD = 16.0), and 12.3 (SD = 14.4) for the first, second, and third ESAS questionnaires, respectively. According to the score given to each symptom, the most frequent disabling symptoms in each questionnaire were hyporexia (10%), pain (23%), and discomfort (15%) in ESAS questionnaire 1, 2, and 3, respectively. Higher scores for MD correlated with higher scores in STAI questionnaires as well as with lower levels of education (r=0.456, p< .005). Conclusions MD in the donor can manifest both clinically and socially through insomnia, fatigue, loss of appetite, anxiety, and ambivalence of feelings (happy to be the donor but feel morally obligated to donate) which may affect family relationships. Although there is no instrument to measure the MD before performing any procedure in the present study, the donors had different symptoms, such as negative feelings, anxiety, tiredness, difficulty to sleep, and even pain; all of them without an apparent physical reason. It is desirable that donors receive bioethical counseling and psychological guidance before the donation process in order to provide them with the necessary information regarding the experience they are about to endure. Disclosures Gomez-Almaguer: Amgen: Consultancy, Speakers Bureau; Teva: Consultancy, Speakers Bureau; Janssen: Consultancy, Speakers Bureau; Takeda: Consultancy, Speakers Bureau; Celgene: Consultancy, Speakers Bureau.

Introduction: Numerous clinical trials have been conducted in order to reduce or prevent the appearance of mucositis in patients undergoing cancer treatment and some of these have appeared in the clinical guidelines as recommendations (Keefe,DM Cancer 2007). However, to date no molecule has been shown to prevent appearance of this kind of lesions. Selenium, a trace element in the organism, cofactor of multiple biological processes, has been used previously for mucositis chemoprevention in head and neck tumors. We conducted a study to know if selenium supplementation has an impact on the development of mucositis. Objective: We aim to know the impact of Selenium in patients undergoing hematopoietic stem cell transplantation, in the presentation and duration of mucositis comparing against placebo. Methods: We performed a double blind clinical trial in a single center pilot study approved by our ethics committee. All patients candidates for autologous or allogeneic HSCT above 18 years were submitted for our study previous informed consent by our institution. Patients were randomized into one of two arms: a) selenium methionine, b) placebo. Previously randomized, patients were administered supplementation with oral selenium methionine at a dose of 400mcg/day or placebo starting 7 days prior transplantion. We determined a baseline measurement of serum and urinary selenium. Subsequent measurements were made each week for a total of 28 days. Evaluations were made with validated SMAQ questionnaires which also included scales of mucositis severity, using the WHO mucositis scale. All of them performed by an observer blinded to what supplementation were being administered to each patient. Results: We have recruited a total of 31 patients, of whom 16 were allogeneic and 15 autologous. We observed that 38.7% developed some degree of mucositis, most of them on the allogeneic arm (10 patients). Selenium consumption arm was associated with a median decrease of 32% (1.9 vs 2.8) in the WHO scale in the severity of mucositis compared to the placebo arm (p=0.046), however we didn't find difference in mucositis resolution time between groups (p=0.162). On the other hand, selenium supplementation was associated with shorter platelet engraftment time (p=0.041), without any significant difference in neutrophil engraftment time. With a follow up of 112 days, there are no difference about mortality or other analized variables. Conclusions: Supplementation with selenium was associated with a slight decrease in the severity of mucositis in comparison with placebo. Without any relevant findings in relation to mortality or other variables analized. Disclosures Gomez-Almaguer: Novartis: Consultancy; AbbVie: Consultancy. Herrera-Rojas:Janssen: Employment.

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