Controversies in hybrid banking: attitudes of Swiss public umbilical cord blood donors toward private and public banking

Purpose: Umbilical cord blood (UCB) stored in public inventories has become an alternative stem cell source for allogeneic stem cell transplantation. The potential use of autologous UCB from private banks is a matter of debate. In the face of the limited resources of public inventories, a discussion on "hybrid” public and private UCB banking has evolved. We aimed to explore the attitudes of the donating parents toward public and private UCB banking. Study design and methods: A standardized, anonymous questionnaire was sent to the most recent 621 public UCB donors including items regarding satisfaction with recruitment process, the need for a second consent before release of the UCB unit for stem cell transplantation, and the donors' views on public and private UCB banking. Furthermore, we asked about their views on UCB research. Results: Of the questionnaires, 48% were returned, and 16% were lost due to mail contact. Of our donors, 95% would donate to the public bank again. As much as 35% of them were convinced that public banking was useful. Whereas 27% had never heard about private UCB banking, 34% discussed both options. Nearly 70% of donors opted for public banking due to altruism and the high costs of private banking. Of our public UCB donors, 81% stated that they did not need a re-consent before UCB release for stem cell transplantation. In case of sample rejection, 53.5% wanted to know details about the particular research project. A total of 9% would not consent. Conclusions: Almost all donors would choose public banking again due to altruism and the high costs of private banking. Shortly after donation, mail contact with former UCB donors was difficult. This might be a relevant issue in any sequential hybrid bankin

Quality-adjusted survival analysis shows differences in outcome after immunosuppression or bone marrow transplantation in aplastic anemia

Bone marrow transplantation (BMT) and immunosuppression (IS) have improved the prognosis of aplastic anemia; both treatments have specific advantages and drawbacks but similar survival rates. Analysis of additional endpoints may help in treatment decisions. In a single-center study, patients with aplastic anemia treated with IS (n=155) or BMT (n=52) were compared for survival, event-free survival, and quality-adjusted time without symptoms and toxicity (Q-TWiST). Probability of overall and event-free survival at 15 years was similar among both groups (BMT 51±15% and 25±14%, IS 53±10% and 27±8%), with more early deaths in the transplant group and more late deaths in the IS group. There were differences in terms of mean duration of seven analyzed health states: time with symptoms from treatment-related toxicity (IS 0.36 years, BMT 0.27), transfusion dependency (IS 0.66 years, BMT 0.1 years), partial remission (IS 3.27 years, BMT 1.42), and secondary clonal disorder (IS 0.68 years, BMT 0.04) was significantly longer for IS compared to BMT (p≤0.001). Patients treated with BMT spent more time with extensive chronic graft-versus-host disease (GvHD) (IS 0 years, BMT 0.96, p<0.023) and in CR without drugs (IS 1.22 years, BMT 2.43, p=0.056). In conclusion, survival, event-free survival, and Q-TWiST are similar. BMT-treated patients had longer periods free from symptoms, while IS-treated patients needed closer medical care, transfusion support, and medication

Aplastic anemia and concomitant autoimmune diseases

The association of aplastic anemia (AA) with other autoimmune diseases (AID) has been described but so far not systematically evaluated. We assessed the incidence and the outcome of concomitant AID in a retrospective, single-center study of 243 patients with severe AA treated between 1974 and 2006 with either immunosuppression (186) or hematopoietic stem cell transplantation (57) and a median follow-up time of 9.3years (0-33). Clinically manifest AID were observed in 24 out of 243 (10 ± 3.7%) patients. Age at diagnosis of AA was significantly younger in patients without AID compared to patients with AID (median, 20 versus 52years; P < 0.001). In 12 patients where the diagnosis of AID was done before AA therapy, response to antithymocyte globulin was good for AA (ten out of 12) but not for AID (2 out of 12). In 13 patients in which AID occurred after first-line therapy, the median time to the AID was 7years (range 3months-27.5years

Klinisches Management einer Thalassämie beim erwachsenen Patienten

The clinical management of patients with thalassemia has changed in the last 40 years. Increasing knowledge of the underlying pathophysiology of the disease, as well as the introduction of modern transfusion policies, together with a consequent iron-chelation strategy, clearly improves the life expectancy for patients with thalassemia. Nowadays children with correctly treated thalassemia major will survive to adulthood. As a consequence emerging complications are expected. The management of symptomatic thalassemia requires a highly specialized multidisciplinary healthcare provider team where the hematologist plays a central role. General practitioners will be more frequently involved in thalassemia patient handling. In this manuscript we will focus on the most relevant complications such are iron overload, bone complications, extramedullary hematopoiesis, hypercoagulability, impaired fertility and pregnancy

Thrombotic complications after haematopoietic stem cell transplantation : early and late effects

Haematopoietic stem cell transplantation is currently the only curative option for many haematological malignancies, but is characterized by a wide spectrum of complications, including haemostatic changes. Bleeding and thrombotic events occur in the early and late phases after transplantation. In the early phase, thrombotic events have a variable clinical picture and present either as venous thrombosis, mainly at the site of central venous lines, veno-occlusive disease (also known as sinusoid occlusion syndrome) or transplant-associated microangiopathy. The latter two occur in the context of an acute graft-vs-host reaction, which involves various organs including the endothelium. In the late phase, years or decades after transplantation, thrombotic events present either as common venous thromboses or as arterial occlusions because of the development of premature atherosclerosis combined with diabetes, hypertension and dyslipidaemia, all of which are accelerated under the influence of the post-transplant treatment. This chapter will discuss the incidence, possible causative associations and treatment options of early and late thrombotic events after haematopoietic stem cell transplantation

Immunosuppressive treatment for aplastic anemia : are we hitting the ceiling?

The combination of antithymocyte globulin of horse origin and cyclosporine A is the standard treatment for aplastic anemia in patients not eligible for bone marrow transplantation. In this perspective article, Drs. Passweg and Tichelli discuss the current immunosuppressive therapy of aplastic anemia. See related article on page 348