Autologous Bone Marrow Infusion Research Articles

Autologous bone marrow infusion via portal vein combined with splenectomy for decompensated liver cirrhosis: A retrospective study.

In a previous study, autologous bone marrow infusion (ABMI) was performed in patients with decompensated liver cirrhosis (DLC) and acquired immunodeficiency syndrome and achieved good results, but whether splenectomy affected outcome was unclear. To investigate the efficacy of ABMI combined with splenectomy for treatment of DLC. Eighty-three patients with DLC were divided into an intervention group (43 cases) and control group (40 cases) according to whether splenectomy was performed. The control group was treated with ABMI through the right omental vein, and the intervention group was additionally treated with splenectomy. After ABMI, the prothrombin time, serum total bilirubin levels, ascites volume and model for end-stage liver disease score in both groups were significantly lower, while the albumin levels were significantly higher than before ABMI (P < 0.01), but there were no significant differences between the groups (P > 0.05). After ABMI, the white blood cell and platelets counts in both groups were significantly higher than before ABMI (P < 0.01), and the counts in the intervention group were significantly higher than in the control group (P < 0.01). After ABMI the CD4+ and CD8+ T cell counts in both groups were significantly higher than before ABMI (P < 0.01). The CD8+ T cell counts in the intervention group increased continuously and the increase had a shorter duration compared with control group. ABMI through the portal vein in patients with DLC can significantly improve liver synthetic and secretory functions, and splenectomy promotes improvement of bone marrow hematopoietic and cellular immune functions.

Infusion of autologous bone marrow derived mononuclear stem cells potentially reduces urinary markers in diabetic nephropathy

Diabetic nephropathy (DN) is the leading cause of end-stage renal disease worldwide. Previous studies demonstrated safety and efficacy of autologous bone marrow-derived mononuclear cells (ABM-MNCs) in induced type-1 diabetes mellitus (T1DM) rats. However, the effect of ABM-MNCs on urinary markers of DN in humans is not well studied. We evaluated the therapeutic effect of ABM-MNCs on the urinary markers microalbuminuria (MAU), urinary type-IV collagen and urinary neutrophil gelatinase-associated lipocalin (uNGAL) in T1DM patients with and without nephropathy. This prospective open-label pilot study included 15 patients with T1DM, who had completed 2 visits within 6 months. Patients were divided into two groups according to the presence (DN, n = 7) and absence of nephropathy (T1DM, n = 8). ABM-MNCs were injected at each visit as per study protocol. Routine laboratory data, diabetes tests (fasting serum C-peptide and insulin, glycated hemoglobin, fasting and postprandial glucose), 24-h MAU and urinary type-IV collagen were measured at each visit. uNGAL levels were studied before and after 3days of ABM-MNCs infusion at each visit. Mean age of patients was 29.2 ± 10.4 years, 33% were male, and 27% of the overall group had hypertension. MAU was significantly reduced in the overall group (- 26.0%, p = 0.037), including in DN (- 83.2%, p = 0.021). A short-term significant reduction of uNGAL levels was observed 3days after ABM-MNCs administration during the both the 1st visit (median 13.4 vs. 9.5ng/ml, p = 0.027) and 2nd visit (median 8.8 vs. 6.4ng/ml, p = 0.042) in both groups. However this reduction did not remain significant at the 6-month follow-up. Urinary type-IV collagen did not respond significantly to ABM-MNCs infusion. Infusion of autologous bone marrow-derived mononuclear cells significantly reduced levels of MAU in DN patients. Further studies with larger sample size are needed to confirm these observations.

Intraportal therapy of autologous bone marrow mononuclear cells in patients on the waiting list for liver transplantation

This study was carried out to explore the methodology of intraportal infusion of autologous bone marrow mononuclear cells in patients in the waiting list for liver transplantation. The study included eight patients. The Model for End-Stage Liver Disease (MELD) score as a predictor of hepatic failure exceeds 10 points, i.e., 12-22 points. According to Child-Turcotte-Pugh (CTP) classification, the severity of cirrhosis ranged from class B to C. The bone marrow volume of 224-320 ml was harvested by bilateral aspiration from the posterior iliac crest. The total volume of the mononuclear cells (MNC) was 47.1-58.3 ml (median, 47 ml). The absolute viable count of isolated MNC was 1.24 · 109-5.84 · 109 and CD34+ cells 2.54 · 106-48.20 · 106. After portal vein catheterization, direct transhepatic portography was performed, and portosystemic collaterals were found in all cases. Portal flowmetry was performed to determine the optimal selective perfusion rate of the segmental branches of the portal vein. Portal vein infusion was performed at 0.3-1.5 ml/sec. Serious side effects were not detected during bone marrow harvesting and MNC infusion. All patients had stable condition (no deterioration was initially noticed) after MNC infusion according to the MELD score and CTP criteria during a 3-month observation. Moreover, improvement in physical health status was observed and obtained using the SF-36 questionnaire. This study confirmed the safety of using intraportal therapy of autologous bone marrow MNC in patients on the waiting list for liver transplantation. Herein, we presented the technique of target delivery of autologous bone marrow MNC to the liver to minimize their loss during intraportal infusion. Prolonged follow-up time and improvement of the present technique are needed for further evaluation of the efficiency of autologous bone marrow MNC.

Repeat infusion of autologous bone marrow cells in multiple sclerosis: protocol for a phase I extension study (SIAMMS-II)

IntroductionThe ‘Study of Intravenous Autologous Marrow in Multiple Sclerosis (SIAMMS)’ trial was a safety and feasibility study which examined the effect of intravenous infusion of autologous bone marrow without myeloablative...

Bringing back the help: autologous bone marrow infusion restores CD4+ T cells in AIDS patients with chronic liver disease

Bringing back the help: autologous bone marrow infusion restores CD4+ T cells in AIDS patients with chronic liver disease

Effect of Autologous Transplantation of Bone Marrow Cells Concentrated With the MarrowXpress System in Patients With Critical Limb Ischemia

Critical limb ischemia (CLI), a vascular disease affecting lower limbs, with high morbidity and mortality, is becoming a challenge due to the aging of the population. Patients without direct revascularization options have the worst outcomes. To date, 25% to 40% of CLI patients are not candidates for surgical or endovascular approaches, facing a major amputation as the ultimate option. This study sought to assess the safety and efficacy of transplantation of autologous bone marrow concentrates in “no-option” patients to restore blood perfusion by collateral flow and limb salvage. We performed a nonrandomized, noncontrolled pilot study for no-option CLI patients using intra-arterial infusion of autologous bone marrow concentrate. Variation of blood perfusion parameters, evaluated by laser doppler flowmetry after 6 and 12 months, was set as primary endpoint. Thirteen enrolled patients showed improvements in objective measurements of perfusion. This uncontrolled study provided evidence that transplantation of autologous bone marrow concentrates was well tolerated by CLI patients without significant adverse effects, demonstrating improved perfusion, confirming the feasibility and safety of the procedure.

Bone marrow cell-based regenerative therapy for liver cirrhosis

Bone marrow cells are capable of differentiation into liver cells. Therefore, transplantation of bone marrow cells has considerable potential as a future therapy for regeneration of damaged liver tissue. Autologous bone marrow infusion therapy has been applied to patients with liver cirrhosis, and improvement of liver function parameters has been demonstrated. In this review, we summarize clinical trials of regenerative therapy using bone marrow cells for advanced liver diseases including cirrhosis, as well as topics pertaining to basic in vitro or in vivo approaches in order to outline the essentials of this novel treatment modality.

A Phase II Trial of Autologous Transplantation of Bone Marrow Stem Cells for Critical Limb Ischemia: Results of the Naples and Pietra Ligure Evaluation of Stem Cells Study

Critical limb ischemia (CLI) is a vascular disease affecting lower limbs, which is going to become a demanding challenge because of the aging of the population. Despite advances in endovascular therapies, CLI is associated with high morbidity and mortality. Patients without direct revascularization options have the worst outcomes. To date, 25%-40% of CLI patients are not candidates for surgical or endovascular approaches, ultimately facing the possibility of a major amputation. This study aimed to assess the safety and efficacy of autologous bone marrow (BM) transplantation performed in "no-option" patients, in terms of restoring blood perfusion by collateral flow and limb salvage. A multicenter, prospective, not-controlled phase II study for no-option CLI patients was performed. Patients were subjected to intra-arterial infusion of autologous bone marrow and followed for 12 months after the treatment. Variation of blood perfusion parameters, evaluated by laser Doppler flowmetry or transcutaneous oximetry, was set as the primary endpoint at 12 months after treatment and amputation-free survival as the secondary endpoint. Sixty patients were enrolled and treated with BM transplantation, showing improvement in objective and subjective measures of perfusion. Furthermore, survival analysis demonstrated improved amputation-free survival rates (75.2%) at 12 months after the treatment. This study provides further evidence that autologous bone marrow transplantation is well tolerated by CLI patients without adverse effects, demonstrating trends toward improvement in perfusion and reduced amputation rate, confirming the feasibility and safety of the procedure.

Timeline for development of autologous bone marrow infusion (ABMi) therapy and perspective for future stem cell therapy

Liver cirrhosis patients generally progress to liver failure. To cure this progressive disease, we developed a novel cell therapy using bone marrow cells; autologous bone marrow cell infusion (ABMi) therapy. We previously described the possible action mechanism of ABMi therapy in the cirrhotic liver, and showed the timeline and results of clinical studies of ABMi therapy. We have also carried out other clinical studies using bone marrow cells and granulocyte colony-stimulating factor. Here, we report a new randomized clinical trial to evaluate the effects of ABMi therapy. However, ABMi therapy may not be possible in patients who are unable to undergo general anesthesia; therefore, we have started to develop a next-generation stem cell therapy using cultured mesenchymal stem cells.

Terapia celular da pseudoartose

ObjectiveTo assess the safety and efficiency of cell therapy for pseudarthrosis. Implant of the bone marrow aspirate was compared to mononuclear cells purified extemporaneously using the Sepax® equipment.MethodsSix patients with nonunion of the tibia or femur were treated. Four received a percutaneous infusion of autologous bone marrow aspirated from the iliac crest, and two received autologous bone marrow mononuclear cells separated from the aspirate with the Sepax®. The primary fixation method was unchanged, and the nonunion focus was not exposed. Physical examination and radiographies were performed 2, 4 and 6 months after the treatment by the same physician. After consolidation of the fracture the satisfaction of the patients was estimated using the adapted QALY scale.ResultsNo complications occurred as a result of the referred procedures. Bone consolidation was obtained in all cases within 3 to 24 weeks. The degree of patient satisfaction before and after bone consolidation was assessed, with the average value increasing from two to nine (p=0.0156).ConclusionWe conclude that the proposed method is effective and safe for the treatment of nonunion of long bones regardless of the stabilization method used. Level of Evidence II, Prospective Comparative Study

Potential Therapeutic Application of Intravenous Autologous Bone Marrow Infusion in Patients with Alcoholic Liver Cirrhosis

The present study was conducted to evaluate the application and efficacy of autologous bone marrow infusion (ABMi) for improvement of liver function in patients with alcoholic liver cirrhosis (ALC). Five subjects and 5 control patients with ALC who had abstained from alcohol intake for 24 weeks before the study were enrolled. Autologous bone marrow cells were washed and injected intravenously, and the changes in serum liver function parameters, and the level of the type IV collagen 7S domain as a marker of fibrosis, were monitored for 24 weeks. The distribution of activated bone marrow was assessed by indium-111-chloride bone marrow scintigraphy. The number of cells infused was 8.0±7.3×10(9) (mean±standard error). The serum levels of albumin and total protein and the prothrombin time were significantly higher during the follow-up period after ABMi than during the observation period in treated patients, whereas no such changes were observed in the controls. In the patients who received ABMi, the Child-Pugh score decreased in all 3 who were classified as class B; the serum levels of type IV collagen 7S domain improved in 4 of the 5 patients; and bone marrow scintigraphy demonstrated an increase of indium-111-chloride uptake in 3 of the 4 patients tested. ABMi for patients with ALC helps improve liver function parameters in comparison with observation during abstinence and ameliorates the degree of fibrosis in terms of serum markers and bone marrow activation in most cases.

Safety and efficacy of bone marrow-derived autologous CD133 stem cell therapy

The Phase I clinical study was designed to assess the safety and feasibility of a dose escalating intracoronary infusion of autologous bone marrow (BM)-derived CD133+ stem cell therapy to the patients with chronic total occlusion (CTO) and ischemia. Nine patients were received CD133+ cells into epicardial vessels supplying collateral flow to areas of viable ischemic myocardium in the distribution of the CTO. There were no major adverse cardiac events (MACE), revascularization, re-admission to the hospital secondary to angina, or acute myocardial infarction (AMI) for the 24-month period following cellular infusion. In addition, there were no periprocedural infusion-related complications including malignant arrhythmias, loss of normal coronary blood flow or acute neurologic events. Cardiac enzymes were negative in all patients. There was an improvement in the degree of ischemic myocardium, which was accompanied by a trend towards reduction in anginal symptoms. Intracoronary infusion of autologous CD133+ marrow-derived cells is safe and feasible. Cellular therapy with CD133+ cells to reduce anginal symptoms and to improve ischemia in patients with CTO awaits clinical investigation in Phase II/III trials.

Autologous Bone Marrow Infusion Activates the Progenitor Cell Compartment in Patients with Advanced Liver Cirrhosis

Several clinical trials of bone marrow cell infusion in patients with liver cirrhosis (LC) have shown clinical improvement, despite conflicting results from animal models. We investigated serial pathological features and the clinical impact after autologous bone marrow infusion (ABMI) in patients with advanced LC. Ten patients with advanced LC due to chronic hepatitis B virus infection underwent ABMI. Serological tests, MRI, and liver biopsies were performed, and quality of life was assessed by a questionnaire. Median serum albumin and hemoglobin levels increased significantly after ABMI. All patients showed an improvement in quality of life, with no serious adverse events. Liver volume, measured by MRI, increased in 80% of the patients, and ascites decreased after ABMI. Child-Pugh scores were also significantly improved at 6 months after ABMI. In the serially biopsied livers, a gradually increasing activation of the hepatic progenitor cell (HPC) compartment, including HPC activation (ductular reaction) and HPC differentiation (intermediate hepatocyte), reached a peak after 3 months, with continued proliferation of hepatocytes, and returned to baseline levels after 6 months. There was no significant change in grade or stage of liver fibrosis or stellate cell activation after ABMI. ABMI is suggested to improve liver function and to activate the progenitor cell compartment. Although clinical improvement was sustained for more than 6 months, histological changes in the liver returned to baseline 6 months after ABMI. Further comparative studies are warranted.

Regeneration of insulin production by autologous bone marrow blood autotransplantation in patients with type 1 diabetes

Keywords Autotransplantation.Betacellfunction.Bonemarrow.Type1diabetesTo the Editor: Among the possible cellular therapies toreplace insulin secretion in diabetes, bone marrow-derivedstem cells have aroused interest because of their plasticityand ease of procurement. Indeed, some experimentalreports suggest that the bone marrow harbours cells capableof differentiation into beta cells [1–3], the transplantation ofwhich can correct hyperglycaemia [1, 4]. However, thesefindings have not been corroborated in other studies [5].Nonetheless, the experimental data are not conclusive andpoint to three potential roles of bone marrow cells in thetreatment of diabetes, namely as a source of stem cells forbeta cells, as a facilitator of beta cell regeneration and as animmune modulator.The infusion of autologous bone marrow was recentlyreported to restore insulin secretion in type 1 diabetes [6].To reproduce these results, we evaluated the effect of anintrapancreatic infusion of autologous bone marrow bloodon insulin production in patients with type 1 diabetes.The present pilot study was designed to include tenpatients with type 1 diabetes of more than 5 years durationand the following characteristics: inadequate glycaemiccontrol (HbA

Cell therapy for ischaemic heart disease: focus on the role of resident cardiac stem cells.

Myocardial infarction results in loss of cardiomyocytes, scar formation, ventricular remodelling, and eventually heart failure. In recent years, cell therapy has emerged as a potential new strategy for patients with ischaemic heart disease. This includes embryonic and bone marrow derived stem cells. Recent clinical studies showed ostensibly conflicting results of intracoronary infusion of autologous bone marrow derived stem cells in patients with acute or chronic myocardial infarction. Anyway, these results have stimulated additional clinical and pre-clinical studies to further enhance the beneficial effects of stem cell therapy. Recently, the existence of cardiac stem cells that reside in the heart itself was demonstrated. Their discovery has sparked intense hope for myocardial regeneration with cells that are obtained from the heart itself and are thereby inherently programmed to reconstitute cardiac tissue. These cells can be detected by several surface markers (e.g. c-kit, Sca-1, MDR1, Isl-1). Both in vitro and in vivo differentiation into cardiomyocytes, endothelial cells and vascular smooth muscle cells has been demonstrated, and animal studies showed promising results on improvement of left ventricular function. This review will discuss current views regarding the feasibility of cardiac repair, and focus on the potential role of the resident cardiac stem and progenitor cells. (Neth Heart J 2009;17:199-207.).

What is the status of gene therapy for primary immunodeficiency?

The efforts to find satisfactory treatments for seriously ill patients with primary immunodeficiency have resulted in the development of important new therapeutic procedures with benefits reaching far beyond the relatively small number of patients affected with these rare disorders. Allogeneic bone marrow transplantation, immunoglobulin and enzyme replacement treatments and more recently gene therapy have all been introduced into clinical medicine as treatments for one or more of the primary immunodeficiency diseases. Beginning in 1990, gene-corrected T cells were first used to treat ADA deficiency SCID. With this demonstration that the gene-transfer procedure could be safely used to introduce functional transgenes into patient cells, clinical trials for a broad range of inherited disorders and cancer were started in the mid 90s. Of all these early clinical experiments, those addressing primary immunodeficiency have also been the most successful. Both ADA and X-SCID have now been cured using gene insertion into autologous bone marrow stem cells. In addition some patients with chronic granulomatous disease (CGD) have shown an unexpectedly high level of functionally corrected granulocytes in their blood following infusion of autologous gene-corrected bone marrow. There remain however a great many significant challenges to be overcome before gene therapy becomes the treatment of choice for these and other disorders. The use of genes as medicines is the most complex therapeutic system ever attempted and it may rake several more decades of work before its real potential as a treatment for both inherited and sporadic disorders if finally realized.

1088. Stem Cell Gene Therapy with No Pre-Conditioning for the ADA-Deficiency Patients Leads to Generalized Detoxification and Delayed, but Steady Hematological Reconstitution

In the previous meeting, we reported the initial follow-up of the two patients with adenosine deaminase (ADA)-deficiency whom we had treated in a clinical trial of retroviral-mediated stem cell gene therapy (SCGT). Our protocol is unique in that patients do not receive any kinds of marrow-preparative conditioning prior to infusion of gene-corrected autologous bone marrow (BM) cells. Since the successful cases of ADA gene therapy reported from Italy used low-dose busulfan to enhance the engraftment of gene-transduced BM cells, it is intriguing to see how the lack of conditioning affects the hematological and immunological reconstitution in our patients. Two patients (pt 1, a 4 year-old girl; pt 2, a 13 year-old boy) were given i.v. BM CD34+ cells transduced with the retroviral vector GCsapM-ADA following the cessation of ADA-enzyme (PEG- ADA) replacement. The first evidence of clinical benefits emerged in a form of systemic detoxification as we previously reported. Steady decrease of toxic metabolites, once accumulated shortly after PEG-ADA discontinuation, is still ongoing at present, 2 years after SCGT. Slow, but gradual increase of lymphocyte counts has followed as the second, reaching the recent values that rather surpass the pre- GT levels. T cells have been the most abundant population among the emerging lymphocytes in both patients, exhibiting relatively rapid development. Most striking is the appearance of peripheral B cells in pt 1 after long latency; B cells had never appeared during the first year, but then emerged and has shown a steady increase thereafter. NK cells showed similar reconstitution kinetics, with the evidence of 100% positive for the presence of ADA-transgene. Consistent with these observations, the proviral copy numbers in peripheral blood lymphocytes (PBL) of pt 1 have now reached 0.7 copies/cell and the latest value of ADA enzyme activity in PBL was shown to be near normal. Although pt 2 has shown much slower kinetics of lymphocyte development, he maintains healthy status with the recent evidence of improvement in T cell function. No serious adverse events, including clonal expansion of certain cell types, have been observed. Although some prophylactic measures are still necessary, both patients have been completely off PEG-ADA for longer than 2 years and now are ready for their new lives; pt 1 entering an elementary school and pt 2 proceeding to a high school. These results suggest that SCGT for ADA-deficiency without pre-conditioning can lead to meaningful hematological reconstitution and be a safe and effective treatment option for the patient who needs SCGT, especially when the conditioning may not be tolerated.

Outcome of the Rotterdam-84 CNS-ALL Chemotherapy Protocol without Radiotherapy for Isolated Central Nervous System Relapsed Acute Lymphoblastic Leukemia.

Introduction Current protocols use radiotherapy (craniospinal irradiation) as the treatment of choice to cure isolated central nervous system (CNS) acute lymphoblastic leukemia (ALL) relapses. The severe toxicity of this treatment encouraged us to develop a new CNS-ALL protocol without radiotherapy.Patients and methods From Jan 1987 till Aug 2004, 13 children were diagnosed in our centre with an isolated CNS relapse after initial treatment according to standard DCOG-ALL protocols. Treatment of CNS relapse consisted of induction by weekly intrathecal Methotrexate (MTX). At remission, an Ommaya reservoir was implanted and CNS-directed intraventricular sandwich therapy, consisting of MTX day 1; ARA-C day 2 and MTX day 3 (dosage according to age) was given every four weeks for one year. At the same time systemic treatment, based on the ALL-6 protocol (JCO 1996; 14:911–8), was started in which at week 23, 44 and 65 intensification courses of 6 weeks duration with Teniposide, HD-ARA-C and HD-MTX were inserted. The total duration of treatment is 95 weeks. Six of 13 patients could be assessed for behavior, intelligence, memory, visual-spatial and visual-motor skills before and after treatment using the CBCL and WISC-RN tests among others.Results All 13 patients, 3 girls and 10 boys aged 2.3 till 14.8 years (9 precursor B-ALL and 4 T-ALL), had an early isolated CNS relapse after a median first remission duration of 16 months (range 2–30 months). Nine of them were high risk according to BFM relapse criteria (male, age < 6 years, T-ALL phenotype, relapse < 18 months from diagnosis). At present, eight patients are alive in 2nd complete remission (CR) with a median follow up of 82 months (range 7–189 months). Five patients relapsed, all high risk, of which three died. One died after a secondary AML, one after a bone marrow (BM) relapse in 2nd CR due to fungal sepsis and one after a combined BM and CNS relapse due to streptococcal meningitis/encephalitis during neutropenia. The fourth patient had a second CNS relapse after 42 months in second remission. He is still in 3rd CR for 86 months after an autologous BM infusion. The fifth patient had recently an isolated BM relapse after 11 months in 2nd CR and started systemic reinduction therapy. The 5 years EFS of this study is 57% ± 15% and the 5 years OS 73% ± 14%. Before start of chemotherapy no significant differences in psychological testing were found in comparison with the normal population. After stop chemotherapy significant lower scores were obtained on the domains of perceptual organization and behavior similar to those found in other patients treated for cancer. Furthermore, our treatment protocol has no significant effect on neurocognitive functioning in comparison with craniospinal radiotherapy.Conclusion Sandwich intraventricular therapy together with systemic anti-leukemia therapy without radiotherapy seems to be an effective treatment with minimal neurocognitive disfunctioning for isolated CNS-ALL relapse. Further investigations in a larger group of patients are essential with special emphasis on comparing late effects of this therapy with radiotherapy.

400 Four-month outcome and safety of intracoronary autologous bone marrow infusion for the treatment of sever ischemic cardiomyopathy with evidence of hibernating myocardium

400 Four-month outcome and safety of intracoronary autologous bone marrow infusion for the treatment of sever ischemic cardiomyopathy with evidence of hibernating myocardium

Transplante de medula óssea e transplante de sangue de cordão umbilical em pediatria

OBJETIVO: descrever as indicações, tipos, principais etapas e complicações do transplante de medula óssea, na forma de uma revisão, com a finalidade de facilitar a compreensão do processo pelo pediatra. FONTES DE DADOS: revisão bibliográfica sobre o tema, utilizando-se como base de dados o Medline. SÍNTESE DOS DADOS: são abordados os tipos de transplante de medula óssea autogênico, singênico e alogênico com suas indicações clínicas, os procedimentos de seleção de doadores, de coleta e infusão das células que irão reconstituir os sistemas hematopoético e imunológico. Expõem-se as alternativas para condicionamento pré-transplante e as novas fontes de células, como o sangue de cordão umbilical, descrevendo-se os resultados e as principais complicações relacionadas ao procedimento, tais como infecções oportunistas, doença enxerto contra hospedeiro, complicações hepáticas, gastrintestinais, gênito-urinárias e cárdio-respiratórias. Aborda-se o risco de complicações tardias e seu impacto na qualidade de vida pós-transplante. CONCLUSÕES: o transplante de medula óssea não proporciona a todos os pacientes uma sobrevida absolutamente normal, mas é um progresso considerável no tratamento das doenças que há pouco tempo não apresentavam alternativas terapêuticas satisfatórias. Conhecendo as diversas etapas do procedimento e sua morbidade, o pediatra pode representar uma fonte de informações aos pacientes com indicação de transplante de medula óssea e seus familiares.