Successful Bone Marrow Transplantation Research Articles

High Rate of Attendance at a Young Adult Clinic Embedded in an Adult Sickle Cell Center: If You Build It, They Will Come

Introduction The transition from pediatric to adult care for people with sickle cell disease (SCD) is associated with multifactorial vulnerabilities including life stage of affected individuals (emerging adulthood), accumulating SCD morbidities, lack of adult specialists to provide care, and limited resources to ensure successful integration into adult care. Much of the data on transition in SCD is reported from pediatrics. The purpose of this study is to describe attendance of young adults with SCD at a young adult clinic embedded in an adult SCD center. Methods The Johns Hopkins IRB approved this single-center, retrospective cohort study that included all patients scheduled with the hematologist at the Young Adult Clinic (YAC) in the Sickle Cell Center for Adults at Johns Hopkins between March 1, 2019 and June 31, 2023. YAC referrals come mostly from pediatric hematology centers or Center staff who refer patients <30 years old without an assigned hematologist. The YAC has an “infinite reschedule” policy: there is no limit on rescheduling missed appointments. We included all patients scheduled to see the YAC hematologist and captured patient demographic and SCD characteristics, referral sources, and clinic attendance. Patients' YAC referral indication was categorized as pediatric referral for aging out of care, internal referral, re-entering care, relocation, transfer from outside adult care, no prior care, or pregnancy. Subjects were classified as “attended” if they ever had a YAC visit and “never attended” if they never had a YAC visit. We compared subject characteristics using tests of proportions, non-parametric, or t-tests as appropriate. Results The 193 subjects with a scheduled visit were mean age 23.8+/-2.4 years, 61% female, 74% HbSS/HbSβ 0 genotypes, 58% were publicly insured, 56% received hydroxyurea or chronic transfusions, and 82% previously received care at a regional pediatric SCD care center (Table 1). For 40%, the appointment indication was pediatric referral for aging out of care. Most scheduled subjects (89%, 171/193) attended a YAC appointment; 11% (22/193) did not. Among those who attended a visit, 61% (105/171) attended the first scheduled visit and 39% (66/171) attended rescheduled visits. The median number of rescheduled visits was 1 (IQR 1). Median days from last pediatric to first YAC visit varied by indication; It was shortest for pediatric referrals (107 days, IQR 193), longer for relocation (304 days, IQR 1022), and longest for those re-entering care (1575 days, IQR 843). Referral from pediatrics for aging out of care was associated with 99% attendance (76/77 attended vs 1/77 never attended, p<0.001). Risks for not attending any visit included re-entering care (14/171 attended vs 7/22 never attended, p<0.001), relocation (11/171 attended vs 6/22 never attended, p<0.01), and longer time from last pediatric visit to first scheduled YAC visit (median 333 days attended vs 1533 days never attended, p<0.01). Visit attendance was not associated with age, sex, genotype, pediatric care site, insurance, SCD treatment, or reschedule attempts. Among 22 subjects who never attended a YAC visit, 7 were seen by a Center Advanced Practice Provider (APP). Among the 15 subjects never seen in the Center (Table 2), most (N=7) were re-entering care and last pediatric visit was over 3 years (median 1296 days, IQR 1855) before scheduled YAC appointment. In total 92% of YAC-eligible subjects engaged in SCD care, 171 with the YAC hematologist and 7 with an APP. In this cohort, there were 4 deaths and 4 successful bone marrow transplants. Conclusion Most young adults offered repeated opportunities to engage in comprehensive SCD care at the YAC ultimately pursue care. Rates of attendance at a specialty clinic for young adults with SCD were especially high, 99%, for referred from pediatric SCD care without no difference in attendance by sex, referral site or insurance, a proxy for socioeconomic status. Patients with care gaps are less likely to attend visits, but the low number of subjects who never attended limits conclusions. The outcomes underscore the high stakes of SCD care: most (57%) used SCD treatment, 2% died, and 2% were cured of SCD. There is overrepresentation of women in the cohort, but men and women are equally represented among clinic non-attenders. Sex-specific risk factors for continuing, or not, in SCD care need study. Resources are needed to identify and bring patients into care.

Beyond Busulfan: Building a Better Conditioner for Bone Marrow Transplantation

Beyond Busulfan: Building a Better Conditioner for Bone Marrow Transplantation

Atypical Presentations of Hypomorphic X-Linked SCID

RationaleIL2RG mutations are associated with X-linked Severe Combined Immunodeficiency (X SCID). There is increasing awareness of Hypomorphic SCID associated with IL2RG variants; we present 3 novel mutations of this gene with variable presentations. MethodsChart review was performed for patients with hypomorphic SCID due to pathogenic IL2RG variants. Plasmablast differentiation was performed from peripheral blood mononuclear cells using CD40L and IL-21 stimulation for 6 days followed by staining for plasmablast markers (CD19+/CD27+/CD38+) and surface immunoglobulins. ResultsPatient 1 is a 7-year-old Honduran boy with history of recurrent sinopulmonary infections including recent CMV pneumonitis, bronchiectasis, chronic lung disease, food allergies, unilateral retinal detachment, and hydrocephalus s/p VP shunt. Immunologic testing showed elevated lymphocyte numbers with decreased memory B cells, normal immunoglobulins, absent vaccine responses, and poor proliferation to mitogens. Whole genome testing showed two mosaic mutations in IL2RG: c. 568C>T (20%), c. 589T>C (87%). He is on prophylaxis with azithromycin and trimethoprim/sulfamethoxazole, immunoglobulin, and awaiting definitive treatment. Patient 2 is a 10-year-old with recurrent sinopulmonary disease with bronchiectasis. He had normal TRECs, normal lymphocyte numbers with normal mitogen proliferation to PHA, normal immunoglobulin levels, but absent vaccine responses, and abnormal plasmablast differentiation (0.12% CD27+/CD38+ at day 6 post in vitro activation) and profoundly decreased expression of surface IgG and IgA in vitro. Genetic testing showed a hemizygous IL2RG c.32T>C mutation, as well as CFTR mutations (c.1972dup and c.1977_1985del, in cis). He is maintained on immunoglobulin replacement. Patient 3 is a 40-year-old who had recurrent infections beginning in childhood, and extranodal B-cell lymphoma at age 26 years. He had pan-lymphopenia, and genetic testing showed IL2RG c.982C>T, p.R328X. He underwent successful bone marrow transplantation from a matched sibling at age 36. ConclusionsHypomorphic X-SCID due to novel or mosaic variants in IL2RG can present with variable symptoms including malignancy and variable lymphocyte phenotype including normal T, B, and NK cell counts, and should be considered in males with opportunistic or persistent infections. [Display omitted]

An Ayurvedic management of the Sickle Cell Anaemia: A Case Study

Sickle Cell Anaemia is a genetic disease-causing high morbidity and mortality. Quality of life hampers due to its chronic nature and painful crisis. Permanent cure is not possible till now, except successful bone marrow transplantation. Considering symptomatology and nature of disease, it can be correlated with Pandu Vyadhi, under the heading of Adibala Pavrutta Vyadhi. The chronicity indicates Asadhya nature of the disease. If some medication improves the quality of life and maintain the health of SCA patient, then such medications and efforts may become helpful to serve the society. A 22-year-old male patient diagnosed to besuffering with sickle Cell Anaemia with symptoms of severe pain in hip joints, knee joints body ache, weakness, anorexia, and weight loss came to the OPD, department of Kayachikita at Sane Guruji Arogya Kendra, Hadapsar, Pune. Mother and father are sickle Cell trait (HbAS). They are the residents of Northern Maharashtra lives in Pune for 8 years. Patient has tried modern medicine but no relief in pain crisis. So, they came here and treated with Ayurvedic medications for 6 months, during which he had not experienced any pain crises. Weight is improved from 47 kg to 53 kg. Patient is now playing outdoor games without any sickle Cell crisis. So, this case study is intended to study the pathophysiology and management of sickle Cell Anaemia from Ayurvedic perspective and to explore the probable mode of action of ayurvedic medications that are used in Sickle-Cell Disease (SCD).

Successful Autologous Bone Marrow Transplantation in Active COVID-19 Patients: Case Report

BackgroundPatients with hematologic malignancies are considered at high risk for COVID-19 infection either from the disease or the treatment. Hematopoietic stem cell transplantation, one of the approved therapies for hematologic malignancies, was performed worldwide during the COVID-19 era with some regulations, such as COVID-19 testing, before proceeding with transplantation or cellular therapy. To the authors’ knowledge, none have reported the result of autologous hematopoietic stem cell transplantation in an active COVID-19 patient. Case PresentationWe describe a successful clinical course of autologous bone marrow transplantation for 2 lymphoma patients who tested positive for COVID-19. A thorough discussion was conducted between multidisciplinary hemato-oncology, intensive care, and infectious diseases teams. The decision was to proceed toward bone marrow transplantation with some modifications in the transplantation protocol and close patient monitoring. ConclusionOur cases lend credence that successful autologous bone marrow transplantation is possible among active COVID-19 patients. The obstacles we faced could be overcome with collaboration between a highly qualified multidisciplinary team. Despite the potential complications, the benefits of bone marrow transplantation among patients with a high risk of relapse and who are still COVID-19-positive outweigh the risks. However, further studies are still recommended to support our inference.

Current Trends in Transplantation of Patients with Hematological Malignancies

Hematopoietic stem cell transplantation (HSCT) is widely used to treat hematological malignancies and nonmalignant hematological disorders. Many advances have been made in improving the success of bone marrow transplantation. However, lack of available matched related or matched sibling donors has posed a significant challenges to the availability and proper utilization of hematopoietic stem cell transplantation in patients with hematological malignancies. The rising availability of alternative donors, particularly haploidentical donors (HIDs), has propelled the rapid rise of allo-HSCT. The aim of this review is to highlight and evaluate the recent advances in the development of HSCT, focusing mainly on the selection of suitable hematopoietic stem cell donors, HID-HSCT and some current advances in the use of umbilical cord blood cells as an alternative graft source. Haplo-HSCT has shown a promising result and better outcomes due to the number of advantages, including a wide range of stem cell sources, increased GVL effects, improved immunologic reconstitution, and positive clinical outcomes. In the near future, haplo-HSCT is likely to be considered as a better option for patient donor selection particularly in AML patients while HLA matching is unlikely to be the most important factor in AML patient donor selection. In addition, the use of umbilical cord blood cells has shown a promising role as an alternative source of HSCT even with other available sources such as bone marrow and peripheral blood. Other transplantation procedures, such as T-cell depletion allografts, Post-transplant cyclophosphamide (PTCY), G-CSF-mobilized allografts, and antithymocyte globulin, have contributed to significant improvements in haplo-HSCT outcomes in recent decades.

Challenges of bone marrow transplant for sickle cell disease in resource limited setting

Sickle cell disorders (SCD) are a group of inherited disorders that results from both parent being carriers, haemoglobin AS resulting in haemoglobin (SS), causing anemia, infections, pain, stroke, priapism, sequenstration crisis, multiple organ dysfunctions etc. There are several ways of managing sickle cell disorder but the best way, which is considered a gold standard cure for the disease is a successful bone marrow transplant of haematopoietic stem cells (HSCT). Bone marrow (BM), Peripheral blood (PBSC) and Umbilical cord blood (UCB) are rich in stem cells. In order to have a good bone marrow transplant, without graft rejection, the laboratory plays a vital role especially in compatibility testing of donor and recipients at the various stages of the transplant, which includes initial stage, during the transplant and follow up testing to ensure tolerance to the new graft and testing for chimerism. There are various types of transplant which include Autologous, Allogeneic (Sibling/ Unrelated Donor), Parent or relative, etc. The various pre transplant tests includes: Sickling test, High Performance Liquid Chromatograph (HPLC), Genetic studies, RBC Indices Hb – 6-9 gm/dL, Peripheral Smear, Retic count Reticulocytosis, ESR, Bone marrow analysis. Post-transplant test Includes Engraftment Analysis, Complete Blood counts, kidney function, liver function, Cholesterol, HIV, Hepatitis B, Hepatitis C, additional infectious studies (Endemic testing), Urinalysis etc. Haemopoietic transplantation challenges in a resource limited settings like Nigeria are enormous and they includes: Danger of serious illness associated with donor-to-patient stem cell transplant, lack of appropriate donors, Graft-versus-host disease (GVHD), Inadequate Human resource, Inadequate equipped facility, cost of the service, Corruption, Lack of political will, Leadership incompétence, Lack of strategic planing, Policy inconsistency, Lack of qualified personnel, Poor healthcare administration, Conflict of interest among health workers. The cost of Bone Marrow Transplantation is highly exorbitant to Nigerians and government need to play vital role towards funding part of the cost and alleviating the pain of sickle cell. Effective implementation of National Health Insurance Scheme and address corruption are the most efficient ways the cost would be subsidized. Appropriate information campaigns largely championed by maternal and child health-care professionals would significantly contribute to raising the level of awareness and acceptance of Cord Blood donation. Training and re training of medical personnel cannot be over emphasized.

CT-282 Case Report: Successful Management of Severe Veno-Occlusive Disease after Allogeneic Stem Cell Transplantation Using a Transjugular Intrahepatic Portosystemic Shunt

Veno-occlusive disease (VOD), also called sinusoidal obstruction syndrome (VOD/SOS), of the liver is a complication of allogeneic bone marrow or peripheral stem cell transplantation and is known to be an unpredictable, life-threatening complication with high mortality. We report here a case of a 24-year-old male patient who was diagnosed in 2019 at Gustave Roussy institute with AML. The patient was treated with the AML protocol 3+7 in December 2019 with a complete cytologic response and a positive MRD. Leukemia relapse was observed in September 2020, treated by gemtuzumab ozogomycin and cytarabine with a negative MRD. A successful allogeneic bone marrow transplantation from an HLA-matched pheno-identical related donor was conducted on December 22, 2020, using a myeloablative conditioning regimen. The patient was always controlled by a daily blood test and controlled blood transfusions, and a weekly repeated Doppler ultrasound examination did not indicate a high-risk profile of VOD. However, 12 days post transplant, the patient's condition deteriorated with the appearance of abdominal ascites, weight gain of 6 kg (per base 76 kg), and liver cytolysis with ASAT 72 UI/L, ALAT 86 UI/L, and normal bilirubin. Worsening cytolysis was observed, with ASAT 659 UI/L, ALAT 557 UI/L, and normal bilirubin. A Doppler ultrasound found 5 positive criteria of VOD (hepatomegaly, portal dilation, enlargement of the gall bladder wall, splenomegaly, ascites, decrease in the diameter of the subhepatic veins). Defibrotide was initiated. The patient's condition worsened and was refractory to medical therapy. Transjugular intrahepatic portosystemic shunting (TIPS) was performed 13 days after transplantation without technical complications in any case. The median hepatic venous pressure gradient prior to the procedure was 20 mmHg with a drop to 12 mmHg. An abdominal CT scan showed an under hepatic hematoma with hemorrhagic syndrome. Ascites puncture with abdominal drain installation was done. In summary, following TIPS, the patient showed clinical improvement, and VOD resolved subsequently with a complete remission post allogeneic transplant.

Organ transplantation in Nepal: Ethical, legal, and practical issues.

In Nepal, live donor organ transplantation is only 14 years old with the first successful kidney transplant made in 2008 and a successful liver and bone marrow transplant made in 2016. However, transplantation of cadaveric cornea dates back to 1998. There are still no cases of animal-to-human organ transplantation in Nepal. There are stringent laws to regulate human body organ transplantation in Nepal which are amended from time to time. However, there is a racket of human traffickers who lure rural people from this low-income country into the illegal organ trade. Furthermore, there is a substantial lack of awareness of organ donation among the general public. This article focuses on the stipulations of ethical, legal, and practical issues of obtaining organs procured from living and brain-dead donors that support the process of transplantation in Nepal. In addition, the article also explores the legal and practical issues of organ trafficking and organ donation awareness in Nepal on the basis of factual data and findings from other studies.

Two Successful Bone Marrow Transplantations Improved Lung Functions in Patients With Chronic Granulomatous Disease

Chronic Granulomatous Disease (CGD) is a rare inherited primary immune deficiency disorder with defective respiratory burst activity in phagocytes, resulting in recurrent pyogenic infections. In this study, we described two CGD patients who had done bone marrow transplantation (BMT). As Bone marrow transplantation (BMT) is the definitive treatment of the disease, we evaluated the function of their lungs before and after BMT. In both patients, the BMT was from their siblings. In case 1, the patient’s pulmonary function (PFT) before BMT was: FEV1: 34, FVC: 40, FEV1 / FVC: 72%, and after BMT was: FEV1: 66, FVC: 40 by 49, FEV1 / FVC: 64%. In case 2, the patient’s PFT before BMT was: FEV1: 22, FVC: 36, FEV1 / FVC: 41%, and after BMT was: FEV1: 47, FVC: 33, FEV1/FVC: 43%. BMT significantly improved their Pulmonary Problems and Preclinical (PFT). In addition, after BMT, both patients’ well-tolerated clinical signs and the infection rate, and the number of hospitalizations in both patients decreased.

Role of β3-Adrenergic Receptor in Bone Marrow Transplant as Therapeutical Support in Cancer.

β3-adrenergic receptor (β3-AR) is the last β-adrenoceptor subtype identified. β3-AR is widely expressed and regulates numerous physiological processes, and it is also a potential target for the treatment of many diseases, including cancers. For some types of cancers, bone marrow transplant (BMT) represents a valid therapeutic support, especially in the case of the necessity of high-dose chemotherapy and radiotherapy. For a successful BMT, it is necessary that a donor’s hematopoietic stem cells (HSCs) correctly reach the staminal niche in the recipient’s bone marrow (BM) and contribute to restore normal hematopoiesis in order to rapidly repopulate BM and provide all the healthy blood cells of which the patient needs. Generally, it takes a long time. Control and accelerate homing and engraftment of HSCs could represent a helpful approach to avoid the complications and undesirable effects of BMT. The evidence that the β-adrenergic system has a role in the BM can be found in different studies, and this leads us to hypothesize that studying this field could be interesting to meliorate the most critical aspects of a BMT. Here, we collected the data present in literature about the role of β3-AR in the BM with the purpose of discovering a possible utility of β3-AR modulation in regulating HSC trafficking and hematopoiesis.

A case of RCVS with PRES diagnosed by thunderclap headache, result in successful allogeneic bone marrow transplantation with continuing tacrolimus

A case of RCVS with PRES diagnosed by thunderclap headache, result in successful allogeneic bone marrow transplantation with continuing tacrolimus

Simultaneous Administration of NOD-2 (MDP) and TLP-4 (LPS) Ligands to Bone Marrow Donors 24 h before Transplantation Increases the Content of Multipotent Stromal Cells (MSCs) in Bone Marrow Grafts in CBA Mice Compared to the Total Result of Their Isolated Administration.

In 3-month bone marrow transplants of CBA mice from bone marrow donors receiving single injections of TLR-4 ligand (LPS) or NOD-2 ligand (muramyl dipeptide, MDP) 24 h before transplantation, an increase in the total number of MSCs (by 2.6 and 1.9 times, respectively), as well as a slight increase in the number of nuclear cells and the mass of bone capsules (by 1.3 and 1.2 times) were observed. After combined administration of MDР and LPS to donors, the total content of MSCs in the grafts was higher by 1.6 times in comparison with the total result of their isolated administration (and by 7.2 times in comparison with the control). At the same time, the concentration of osteogenic MSCs in the grafts of all groups was almost the same and corresponded to the control level. The number of nuclear cells and the mass of bone capsules of the grafts after combined administration of LPS and MDP were close (~80%) to the sum of the results of their isolated administration. These findings suggest that activation of the stromal tissue and the success of bone marrow transplantation depend on the intensity of innate immune responses. These data can be useful for the development of optimal methods of tissue transplantation.

An Ayurvedic Management of Sickle Cell Anemia in Children: A case study

Introduction:- Sickle cell anemia is a term for a group of genetic diseases characterized by the production of hemoglobin (Hb) "S". This is the result of replacing the base pair thymine at the 6th position of the β-globin gene on chromosome 11 with adenine and replacing valine with glutamic acid. A single amino acid substitution is the reason for the profound changes in the stability and solubility of the hemoglobin "S" molecule. Due to its chronic nature and painful crisis, children’s quality of life is difficult. From now on, there is no final treatment other than a successful bone marrow transplant. Its symptoms and the nature of the disease can be compared with Pandu. If a drug improves the quality of life and maintains the health of the patient, then the drug and efforts will be beneficial to society. Material and Methods:- A 9-year-old male patient was diagnosed with sickle cell anemia. Symptoms include joint and limb pain, anorexia, fatigue, loss of appetite, and weight loss. approached the OPD, department of Kaumarbhritya, Parul Institute of Ayurved, Vadodara. Observations and Results:-The patient is taking modern medicine (1 mg folic acid once a day). The Childs parents also wanted to take Ayurvedic medicine, so they came here for 2 months of Ayurvedic medicine treatment, during which he did not have a pain crisis.Weight improved From 16 kg to 18 kg.The quality of life of a patient is also improved. Conclusion:-Therefore, the purpose of this case study is to study the pathophysiology and treatment of sickle cell anemia from an Ayurvedic perspective and to study the possible mechanism of action of the drug.

Some clinical lessons from one Blood and Marrow Transplantation Unit in Medellin, Colombia

After more than 60 years of the first successful bone marrow transplant (BMT) by D.E. Thomas for the treatment of hematological malignant diseases and more than 46 years since the first bone marrow transplant by Alberto Restrepo-Mesa in Medellin-Colombia for the treatment of a female triplet patient with paroxysmal nocturnal hemoglobinuria and aplastic anemia, in Colombia only around 750 bone marrow transplants are performed annually.
 With the experience accumulated during these years by each one of us, the León XIII Clinic of the Universidad de Antioquia began the hematopoietic stem cell transplantation (HSCT) program for adults in 2014. In this review, we report some clinical lessons drawn from the different phases of the HSCT in 109 adult patients with hematological malignancies. The progression-free survival (PFS) and the five-year overall survival (OS) were for autologous stem cell transplantation (ASCT) (87% and 70%), allogeneic stem cell transplantation (Allo SCT) (50% and 40%) and haploidentical stem cell transplantation (Haplo SCT) (25% and 18%) respectively.

A Study to Identify Correlation of Hematopoietic Progenitor Cell(XN-HPC) By Sysmex Cell Counters with Final Yield of CD34 + Stem Cells

Successful bone marrow transplantation depends on the collection and infusion of enough numbers of peripheral blood stem cells. Several predictors of stem cell yield are used now, including white blood cell (TLC) count and CD34 in peripheral blood. This study evaluated the utility of the new automated hematopoietic progenitor cell count(HPC) available on Sysmex XN hematology analyzers (XN-HPCs) in stem cell harvest. STUDY DESIGN AND METHODSThe characteristics of XN-HPC, CD34+ stem cells, and TLC analysis were compared using 217 matched peripheral blood and apheresis bag samples. RESULTSGood correlation was observed between HPC and CD34+ stem cell counts in peripheral blood (r = 0.90; slope, 0.82) and apheresis collections (r = 0.93; slope, 0.91). There is a good corelation that peripheral blood HPC and CD34 analysis showed ability to predict successful stem cell harvests (≥ 2 × 106 CD34+ cells/kg). Fixing a cutoff of 25 × 106 HPC/L, peripheral blood XN- HPC and CD34 analysis both showed negative predictive values (NPVs) of 100% and positive predictive values (PPVs) of 66% and 80%, respectively. In contrast, the correlation between peripheral blood TLC and CD34 analysis was poor (r = 0.39), and the peripheral blood WBC count (cutoff, 20 × 109/L) was a poor predictor of PBPC harvest (NPV 55%, PPV 38.7%). CONCLUSIONHematopoietic Progenitor cell (HPC) compares well with both peripheral blood and bag CD34 analysis and may act as a cheap surrogate marker for CD34 analysis to predict optimal timing of stem cell harvest especially in Autologous bone marrow transplant receipients.This will help us in reducing costs in a resource limited developing country like India. DisclosuresNo relevant conflicts of interest to declare.

Successful Salvage Haploidentical Bone Marrow Transplantation in a Child With Hemophagocytic Lymphohistiocytosis, When the Previously Matched Unrelated Donor Tested Positive for SARS-CoV-2 on the Day of Stem Cells Collection

The coronavirus disease 2019 pandemic has made us adjust our standards and cope with unpredictable circumstances affecting the whole world, including the medical field.A 2-year-old boy diagnosed with X-linked lymphoproliferative disease type 2 with concomitant positive polymerase chain reaction test for Epstein-Barr virus–DNA was admitted to our transplant ward. His treatment scheme had to be modified at the last moment because of a donor disqualification due to a positive polymerase chain reaction result for severe acute respiratory syndrome coronavirus 2 just before the apheresis. We decided to perform salvage haploidentical bone marrow transplant from the patient's mother because it was the only possible option. Now, in a 5-month observation period after the hematopoietic stem cell transplantation, our patient is in good general condition. His case convinced us to redirect our approach to transplant procedure preparation. Following the European Group of Blood and Marrow Transplantation recommendations, we use cryopreserved apheresis materials to ensure the availability of stem cell products before the start of a conditioning regimen.

Predictors of a short hospitalization in bone marrow transplantation patients presenting to the emergency department.

Despite the advantages of bone marrow transplantation (BMT), patients receiving this intervention visit the emergency department (ED) frequently and for various reasons. Many of those ED visits result in hospitalization, and the length of stay varies. The objective of our study was to identify the patients who were only briefly hospitalized and were thus eligible for safe discharge from the ED. This was a retrospective cohort study conducted on all adult patients who have completed a successful BMT and had an ED visit that resulted in hospitalization. Our study included 115 unique BMT with a total number of 357 ED visits. Around half of those visits resulted in a short hospitalization. We found higher odds of a short hospitalization among those who have undergone autologous BMT (95%CI [1.14-2.65]). Analysis of the discharge diagnoses showed that patients with gastroenteritis were more likely to have a shorter hospitalization in comparison to those diagnosed with others (95%CI [1.10-3.81]). Furthermore, we showed that patients who presented after a month from their procedure were more likely to have a short hospitalization (95%CI [1.04-4.87]). Another significant predictor of a short of hospitalization was the absence of Graft versus Host Disease (GvHD) (95%CI [2.53-12.28]). Additionally, patients with normal and high systolic blood pressure (95%CI [2.22-6.73] and 95%CI [2.81-13.05]; respectively), normal respiratory rate (95%CI [2.79-10.17]) and temperature (95%CI [2.91-7.44]) were more likely to have a shorter hospitalization, compared to those presenting with abnormal vitals. Likewise, we proved higher odds of a short hospitalization in patients with a quick Sepsis Related Organ Failure Assessment score of 1-2 (95%CI [1.29-5.20]). Moreover, we demonstrated higher odds of a short hospitalization in patients with a normal platelet count (95%CI [1.39-3.36]) and creatinine level (95%CI [1.30-6.18]). In our study, we have shown that BMT patients visit the ED frequently and many of those visits result in a short hospitalization. Our study showed that patients presenting with fever/chills are less likely to have a short hospitalization. We also showed a significant association between a short hospitalization and BMT patients without GvHD, with normal RR, normal T °C and a normal platelet count.

Autoimmune Liver Disease in Patients With Sickle Cell Disease.

Hepatic and biliary tract diseases are common in sickle cell disease (SCD) patients, likely due to sickling, hemosiderosis, viral hepatitis, or cholelithiasis. Literature is lacking on associations between SCD, autoimmune hepatitis (AIH), and/or sclerosing cholangitis (SC)-together, autoimmune liver disease (AILD). We aimed to better understand the relationship of these diseases in pediatric patients. A retrospective analysis of patients with SCD and AILD at the Children's Hospital of Philadelphia (January 2008 to August 2015). Seven patients, ages 8 to 23 years (3 males), were identified. Three had AIH, 2 SC, and 2 AIH/SC overlap, known as autoimmune SC. All patients with AIH treated with azathioprine significantly improved their liver enzymes. One patient with SC and inflammatory bowel disease underwent successful bone marrow transplant. Two SC patients died from SCD complications. In this cohort, there seems to be an association between SCD and AILD; SC in this population was severe. Physicians should be aware of this and evaluate patients with SCD and elevated liver enzymes for AILD.

Impact of bone marrow transplantation on resting energy expenditure of oncohematological patients

Rationale: The assessment of Resting Energy Expenditure (REE) is important for early nutritional intervention and improves the success of bone marrow transplantation.