Background. Relapsed acute promyleocytic leukemia (APL) is treated with re-induction chemotherapy, commonly arsenic trioxide, and stem cell transplantation (SCT). The effect of arsenic trioxide on autologous peripheral blood stem cell collection is unknown. Procedure. Five pediatric patients with relapsed APL had PML-RARA negative peripheral blood stem cells mobilized (four after arsenic trioxide) and underwent autologous SCT after cyclophosphamide (60 mg/kg x 2) and total body irradiation (TBI-fractionated 1,200 cGy) conditioning. Results. All five patients remain in molecular remission a median of 20 months post-transplant. Conclusion. Autologous SCT performed during molecular remission is a treatment option for pediatric patients with relapsed APL and may provide durable leukemia-free survival without the complications of allogeneic transplantation.

Rapidly progressive diffuse systemic sclerosis (dSSc) is a life-threatening condition characterized by increased mortality with few effective therapies, typically only helpful in stabilizing disease. Autologous hematopoietic stem cell transplantation (AHSCT) is the only treatment that has demonstrated improved survival. Despite promising results from three randomized controlled trials (RCTs), best practice use of AHSCT in the real-world setting is not well established. The primary objective of this thesis was to summarize the clinical efficacy, limitations and utilization of AHSCT in the management of rapidly progressive dSSc. Specifically, we conducted (1) a systematic review to describe the efficacy of AHSCT in dSSc as well as practice variation in patient selection and treatment regimens; and (2) a multicenter retrospective cohort study to compare outcomes for subjects who received AHSCT in France compared to those who received conventional care in Canada. There was important variability in the criteria for patient selection and treatment protocols. While AHSCT is associated with improved overall survival, skin fibrosis and lung function, further studies are needed to understand its potential for expanded eligibility and effects on other disease manifestations.

Busulfan, cyclophosphamide, and etoposide (BuCyE) is a commonly used conditioning regimen for autologous stem cell transplantation (ASCT). This multicenter, phase II study examined the safety and efficacy of BuCyE with individually adjusted busulfan based on preconditioning pharmacokinetics. The study initially enrolled Hodgkin lymphoma (HL) and non-Hodgkin lymphoma (NHL) patients ages 18 to 80 years but was amended due to high early treatment-related mortality (TRM) in patients > 65 years. BuCyE outcomes were compared with contemporaneous recipients of carmustine, etoposide, cytarabine, and melphalan (BEAM) from the Center for International Blood and Marrow Transplant Research. Two hundred seven subjects with HL (n = 66) or NHL (n = 141) were enrolled from 32 centers in North America, and 203 underwent ASCT. Day 100 TRM for all subjects (n = 203), patients > 65 years (n = 17), and patients ≤ 65 years (n = 186) were 4.5%, 23.5%, and 2.7%, respectively. The estimated rates of 2-year progression-free survival (PFS) were 33% for HL and 58%, 77%, and 43% for diffuse large B cell lymphoma (DLBCL; n = 63), mantle cell lymphoma (MCL; n = 29), and follicular lymphoma (FL; n = 23), respectively. The estimated rates of 2-year overall survival (OS) were 76% for HL and 65%, 89%, and 89% for DLBCL, MCL, and FL, respectively. In the matched analysis rates of 2-year TRM were 3.3% for BuCyE and 3.9% for BEAM, and there were no differences in outcomes for NHL. Patients with HL had lower rates of 2-year PFS with BuCyE, 33% (95% CI, 21% to 46%), than with BEAM, 59% (95% CI, 52% to 66%), with no differences in TRM or OS. BuCyE provided adequate disease control and safety in B cell NHL patients ≤ 65 years but produced worse PFS in HL patients when compared with BEAM.

Class of 2012 Abstract / Specific Aims: Autologous stem cell transplant (ASCT) is the current gold standard following induction therapy to improve survival of multiple myeloma (MM). Lenalidomide (LEN) is used for treatment of MM before ASCT, but exposure may impair autologous peripheral blood stem cell (PBSC) mobilization. Chemomobilization with cyclophosphamide (CTX) has not been evaluated in this setting. CTX + filgrastim was investigated to determine if LEN-associated mobilization impairment can be abrogated. Methods: 36 pts (group A=12 pts who received ≥2 cycles of LEN and group B=24 pts without LEN) were analyzed retrospectively. Baseline characteristics were matched (p>0.05 for all variables). All pts received CTX (median group B, 1.5g/m2; median group A, 3gm/m2(p=0.18)) and filgrastim 10µg/kg/day. Primary outcomes include number of CD34+ cells collected and number of leukapheresis sessions. Secondary outcomes include failure to collect CD34+ cells and total CD34+ cells collected after second leukapheresis. Main Results: Total median number of CD34+ cells collected in group B=9.15x106/kg CD34+ cells and group A=7.43x106/kg CD34+ cells (p=0.159). Median number of apheresis sessions in group B=2 and group A=3 (p=0.42). Two of 12 pts with antecedent LEN usage failed to collect while no patient without previous LEN exposure failed to collect (p=0.105). Total number of CD34+ cells collected after 2 apheresis sessions for group B=8.13x106/kg CD34+ cells and group A=3.34x106/kg CD34+ cells (p=0.06). Conclusions: Chemomobilization with CTX + filgrastim yields robust PBSC collections irrespective of antecedent lenalidomide. There was a trend towards lesser PBSC collection in LEN-treated pts.

No description available.

Stem cell transplant is often considered the last hope for the survival for many cancer patients. The CD34+ cell content of a collection of stem cells has appeared as the most reliable indicator of the quantity of desired cells in a peripheral blood stem cell harvest and is used as a surrogate measure of the sample quality. Factors predicting the yield of CD34+ cells in a collection are not yet fully understood. Throughout the literature, there has been conflicting evidence with regards to age, gender, disease status, and prior radiation. In addition to the factors that have already been explored, we are interested in finding a cancer-chemotherapy interaction and to develop a predictive model to better identify which patients will be good candidates for this procedure. Because the amount of CD34+ cells is highly skewed, most traditional statistical methods are inappropriate without some transformation. A Bayesian generalized regression model was used to explain the variation of CD34+ collected from the sample by the cancer chemotherapy interaction. Missing data was modeled as unknown parameters to include the entire data set in the analysis. Posterior estimates are obtained using Markov chain methods. Posterior distributions identified weight and gender as well as some cancer-chemotherapy interactions as significant factors. Predictive posterior distributions can be used to identify which patients are good candidates for this procedure.

No description available.

Introduktion Autolog stamcellstransplantation är idag en vanlig behandling vid myelom och högmaligna lymfom hos patienter <65 år utan omfattande komorbiditet. Behandlingen delas upp i fem faser: induktionsbehandling, stamcellsmobilisering, stamcellsskörd, konditionering med högdoscytostatika och stamcellsåtergivning/transplantation. Initialt behandlades alla patienter som genomgick autolog stamcellstransplantation inom slutenvården under den sista behandlingsfasen, det vill säga i samband med konditionering och stamcellsåtergivning. Sedan 1990-talet har man dock på många håll i världen övergått till poliklinisk vårdform för denna patientgrupp. Detta innebär att patienten genomgår stamcellstransplantationen inom slutenvården men efter detta behandlas som öppenvårdspatient med fasta återbesök på sin hemklinik under posttransplantfasen. Poliklinisk vårdform har visat sig vara en säker, uppskattad och kostnadseffektiv vårdform som inte medför större risker för patienten och som inte ökar mortalitet och/eller morbiditet i samband autolog stamcellstransplantation. Syfte Syftet med denna studie är att undersöka om det föreligger skillnad i grad av cytostatikarelaterat fördröjt illamående och kräkningar mellan patienter som vårdats polikliniskt jämfört med patienter som vårdats inneliggande på vårdavdelning efter autolog stamcellstransplantation. Metod Studiepopulationen utgörs av 91 patienter varav 33 vårdades polikliniskt och 58 vårdades inom slutenvården efter autolog stamcellstransplantation. Patienterna fyllde i en illamåendedagbok i samband med behandlingen varpå dessa analyserades utifrån variabler gällande cytostatikarelaterat fördröjt illamående och kräkningar. Resultat Resultatet visar att de polikliniserade patienterna mår generellt bättre än de icke- polikliniserade patienterna vad gäller cytostatikarelaterat fördröjt illamående och kräkningar. Sammanfattning Föreliggande studie indikerar ett positivt samband mellan poliklinisk vårdform och lägre incidens av cytostatikarelaterat fördröjt illamående och kräkningar hos patienter som genomgår autolog stamcellstransplantation. / Introduction Treatment for myeloma and lymphoma today typically involves autologous stem cell transplantation for patients <65 years without coexisting comorbidity. The treatment consists of five stages: induction treatment, stem cell mobilisation, stem cell harvest, conditioning with high dose chemotherapy and stem cell rescue (transplantation). Historically all patients treated with autologous stem cell transplantation received treatment as inpatients but this practice has since the 1990ies, due to for instance financial reasons, gradually shifted into an outpatient approach to this line of care. Thus, for the patient the outpatient approach entails myeloablative conditioning and stem cell transplantation as inpatient followed by post transplant care as outpatient part of the home clinic’s outpatient program. Outpatient care following autologous stem cell transplantation has proven to be a safe, highly appreciated and cost effective method of care without any adverse effects on behalf of the patients with regards to clinical outcome, mortality and/or comorbidity. Objectives The aim of this study is to ascertain whether or not there is a difference in degree of chemotherapy-induced delayed nausea and vomiting between an outpatient population and an inpatient population following autologous stem cell transplantation. Methods A total of 91 patients, 33 of whom were included in an outpatient program while remaining 58 were treated as regular inpatients, participated in the study. Patients each day filled out a diary with regards to nausea and emesis during the entire treatment phase. Submitted data was then analysed concerning parameters related to chemotherapy-induced delayed nausea and vomiting. Results The result shows that the outpatient population suffers less in general than the inpatient population in terms of chemotherapy-induced delayed nausea and vomiting. Conclusion To conclude, this study suggests a positive correlation between outpatient care following autologous stem cell transplantation and a lower incidence of chemotherapy-induced delayed nausea and vomiting.

No description available.

Background: Ewing sarcoma is one of the most frequent soft-tissue tumors in pediatric patients. The current treatment protocols recommend stem cell apheresis (SCA) after completion of the second course of induction therapy with vincristine, ifosfamide, doxorubicine, and etoposide (VIDE). The feasibility of SCA and graft compositions in adult patients with Ewing sarcoma have not been previously analyzed. Methods and Materials: The authors analyzed 29 stem cell collections of 19 adult patients (9 male, 10 female) at a median age of 27 (range 19–53) years mobilized after VIDE (n = 17), cyclophosphamide/topotecan (n = 1) or vincristine, dactinomycin and ifosfamide (n = 1) chemotherapy. All patients were mobilized with filgrastim 5 μg/kg twice daily from day +7 of chemotherapy. The collections were performed if CD34+ cell count in peripheral blood was >10/μL. The target yields were ≥4106 CD34+ cells/kg body weight. Results: Median CD34+ cells/μL in peripheral blood before SCA were 45.8 (range 6.7–614.4)/μL. The median cumulative yields were 10.6 (range 1.5–38.8) CD34+ cells/kg body weight and ≥2106 in all but two patients (89%). CD34, CD3, and CD56 yields in collections after the third VIDE and after later courses did not differ. Four patients underwent high-dose therapy with autologous transplantation, and all were engrafted. Discussion: Stem cell mobilization is feasible in most Ewing sarcoma patients. Additionally, the present study's data suggest that it is safe to postpone stem cell collection to a later VIDE chemotherapy cycle if medically indicated