Successful eradication of chronic myeloid leukemia in a child despite allogeneic graft rejection

Background Chronic myeloid leukemia (CML) is a rare disease in children and treated with tyrosine kinase inhibitors (TKI) and with allogeneic hematopoietic stem cell transplantation (HSCT) still in many cases. Case We describe an 8-year-old patient with CML treated with two different TKIs before proceeding to allogeneic HSCT. Despite successful engraftment, prompt rejection of the graft was followed by autologous reconstitution. TKI therapy was reintroduced post-rejection in anticipation of relapse but shortly discontinued due to low white blood cell and neutrophil counts. The patient has remained disease-free over 5 years after graft rejection and without further therapy. Conclusion This case suggests that even a short antileukemic effect by an allogeneic transplant may succeed in eradicating CML.Non peer reviewe

CAR-T-soluhoito - mitä ja millä hinnalla?

Teema : Hematologiset syövät. English summar

Lasten pienentyneet valkosolumäärät

Lasten pienentyneet valkosolumäärät johtuvat tavallisesti neutrofiilisten granulosyyttien määrän vähentymisestä. Yleensä kyseessä on ohimenevä virusinfektioihin liittyvä ilmiö. Imeväisiässä ja varhaislapsuudessa immuunivälitteiset neutropeniat ovat mahdollisia pitkittyneen leukopenian aiheuttajia. Harvinaisia leukopenian aiheuttajia on lukuisia, ja taustalla voi olla synnynnäinen luuydinsairaus, jokin oireyhtymä tai pahanlaatuinen veritauti. Neutropeniaan liittyvä infektioriski on suurentunut nimenomaan niillä potilailla, joiden neutropenian taustalla on luuytimen tuotantovika. Jatkotutkimukset ovat tarpeen, mikäli pienentyneisiin valkosoluarvoihin liittyy merkittäviä infektioita, muita hematologisia poikkeavuuksia tai poikkeavia kliinisiä löydöksiä

Early vascular toxicity after pediatric allogeneic hematopoietic stem cell transplantation

Treatment-related mortality and morbidity remain a challenge in hematopoietic stem cell transplantation (HSCT). In this retrospective, single-center study, we analyzed endothelial damage as a potential, common denominator and mechanism for the adverse effects. We evaluated the prevalence of key vascular complications and graft-versus-host disease among 122 pediatric patients with an allogeneic HSCT between 2001 and 2013. The spectrum and frequency of acute adverse events emergingPeer reviewe

Syöpälääketutkimus lasten lääketutkimuksen eturintamassa

Teema : lastenlääkkeet. English summar

Käänteishyljintä kantasolusiirroissa

English summar

Pediatric acute graft-versus-host disease prophylaxis and treatment : surveyed real-life approach reveals dissimilarities compared to published recommendations

Pediatric allogeneic hematopoietic cell transplantation (HCT) practices differ from those of adults, particularly the heterogeneity of transplantable nonmalignant diseases and the lower incidence of graft-versus-host disease (GVHD). Several guidelines regarding the management of acute (a) GVHD in adult HCT have been published. We aimed to capture the real-life approaches for pediatric aGVHD prophylaxis/treatment, and data from 75/193 (response rate 39%) EBMT centers (26 countries) were included, representing half (48%) of the pediatric EBMT-HCT activity. Results with >= 75% approval from respondents (74/75) for GVHD prophylaxis after myeloablative HCT for malignancies partially contradict published guidelines: Single-agent cyclosporine A (CsA) was used for matched sibling donor HCT in 47%; blood CsA levels were reported lower; the relapse risk in malignant diseases influenced GVHD prophylaxis with early withdrawal of CsA; distinct longer duration of CsA was employed in nonmalignant diseases. Most centers used additional anti-thymocyte globulin for matched unrelated and mismatched donor HCT, but not for matched siblings. Regarding prophylaxis in nonmyeloablative conditioning (mainly for nonmalignant diseases), responses showed broad heterogeneity. High conformity was found for first-line treatment; however, results regarding steroid-refractory aGVHD indicate an earlier diagnosis in children. Our findings highlight the need for standardized pediatric approaches toward aGVHD prophylaxis/treatment differentiated for malignant and nonmalignant underlying diseases.Peer reviewe

FASCIA Method in the Assessment of Lymphocyte Mitogen Responses in the Laboratory Diagnostics of Primary Immunodeficiencies

Peer reviewe

ABO incompatibile graft management in pediatric transplantation

Up to 40% of donor-recipient pairs in SCT have some degree of ABO incompatibility, which may cause severe complications. The aim of this study was to describe available options and survey current practices by means of a questionnaire circulated within the EBMT Pediatric Diseases Working Party investigators. Major ABO incompatibility (donor's RBCs have antigens missing on the recipient's cell surface, towards which the recipient has circulating isohemagglutinins) requires most frequently an intervention in case of bone marrow grafts, as immediate or delayed hemolysis, delayed erythropoiesis and pure red cell aplasia may occur. RBC depletion from the graft (82%), recipient plasma-exchange (14%) were the most common practices, according to the survey. Graft manipulation is rarely needed in mobilized peripheral blood grafts. In case of minor incompatible grafts (donor has isohemagglutinins directed against recipient RBC antigens), isohemagglutinin depletion from the graft by plasma reduction/centrifugation may be considered, but acute tolerability of minor incompatible grafts is rarely an issue. According to the survey, minor ABO incompatibility was either managed by means of plasma removal from the graft, especially when isohemagglutinin titer was above a certain threshold, or led to no intervention at all (41%). Advantages and disadvantages of each method are discussed.Peer reviewe