A clinical study of haploidentical hematopoietic stem cell transplantation in the treatment of pediatric patients with acquired severe aplastic anemia: single center experience / 中华血液学杂志

Objective: To investigate the efficacy of haplotype hematopoietic stem cell transplantation in the treatment of acquired severe aplastic anemia (SAA) in children. Methods: The clinical characteristics of 59 pediatric patients with SAA, including 26 cases VSAA, 37males and 22 females, 47 cases typeⅠ and 12 cases typeⅡ, undrerwent haplo-HSCT in our hospital between December 1st, 2011 and December 1st, 2017 were retrospectively analyzed. Among 59 patients, 56 patients with a median age of 4.5 (1.2-14.8) years and median weight of 43 (12-80) kg underwent their first HSCT and 3 patients underwent their second HSCT. All patients received the following conditioning regimen: busulfan, cyclophosphamide, and rabbit ATG or Bu (-, CTX) , fludarabineand rabbit ATG. The prophylaxis of acute graft versus host disease (aGVHD) was cyclosporine (CsA) , MMF and methotrexate. All patients received bone marrow transfusion on day 01 and peripheral stem cell transfusion on day 02 from haploid donor. The median dose of donor mononuclear cell counts was 15.60 (7.74-21.04) ×108/kg of recipient weight and CD34+ cell counts was 4.86 (3.74-7.14) ×106/kg of recipient weight. Results: Neutrophils and platelets of all 59 children were implanted. The median implantation time of granulocytes and platelets were 13 (10-19) d, 19 (9-62) d, respectively. The incidence of grade Ⅰ-Ⅱ aGVHD was 45.76% (27 cases) and grade Ⅲ/Ⅳ 13.56% (8 cases) , The incidence of chronic GVHD was 8.47% (5 cases) , The incidences of CMV and EBV viremia were 59.32% (35 cases) and 28.81% (17 cases) , respectively. The median follow-up was 30 (8-80) months, 57 patients survived with disease free, 2 patients died of GVHD. Both of the estimated 5-year OS and DFS rates were (96.4±2.5) %. Conclusion: Haplo-HSCT could improve the outcomes of SAA children.

A clinical study of haploid hematopoietic stem cells combined with third-party umbilical cord blood transplantation in the treatment of chronic granulomatous disease / 中国当代儿科杂志

OBJECTIVE@#To investigate the clinical efficacy of haploid hematopoietic stem cells (haplo-HSC) combined with third-party umbilical cord blood (tpCB) transplantation in the treatment of X-linked chronic granulomatous disease (X-CGD).@*METHODS@#The clinical data of 26 boys with X-CGD were retrospectively analyzed who were admitted to the Sixth Medical Center of PLA General Hospital between April 2014 and March 2018. All the patients were treated with haplo-HSC combined with tpCB transplantation. The median age of the patients was 3.5 years. The donor was the father in 25 cases and an aunt in 1 case. Transplantation was 5/6 HLA-matched in 9 cases, 4/6 in 12 cases, and 3/6 in 5 cases. The patients received busulfan, cyclophosphamide, fludarabine, or anti-thymocyte globulin for myeloablative preconditioning. Cyclosporine A and mycophenolate mofetil were used for prevention of acute graft-versus-host disease (aGVHD). Then the patients were treated with haploid bone marrow hematopoietic stem cells combined with tpCB transplantation on day 1 and haploid peripheral hematopoietic stem cells on day 2. The counts of median donor total nucleated cells, CD34 cells, and CD3 cells were 14.6×10/kg, 5.86×10/kg, and 2.13×10/kg respectively.@*RESULTS@#The median time to neutrophil and platelet engraftment was 12 and 23 days after transplantation respectively. Full donor hematopoietic chimerism was observed on day 30. Twenty-five cases were from haplo-HSC and 1 was from cord blood. No primary implant failure and implant dysfunction occurred, and secondary implant failure occurred in one case. The NADPH oxidase activity returned to normal one month after transplantation. The incidence of grade I-II aGVHD and grade III-IV aGVHD was 35% and 15% respectively. Chronic GVHD (cGVHD) of the skin occurred in one case, and no progression was observed after steroid administration. During the follow-up period of 6-51 months, 25 patients survived, of whom 24 were disease-free (23 patients without cGVHD and 1 with cGVHD of the skin) and NADPH oxidase activity returned to normal; one patient developed secondary implant failure but survived; one patient died of viral interstitial pneumonia 16 months after transplantation. The 5-year event-free survival rate and overall survival rate were 81%±12% and 89%±10% respectively.@*CONCLUSIONS@#Haplo-HSC combined with tpCB transplantation is one of the effective methods for the treatment of X-CGD in children.

Clinical effect of umbilical cord blood transplantation in 37 pediatric patients with hematologic malignancies: a single-center experience / 中国当代儿科杂志

<p><b>OBJECTIVE</b>To evaluate the clinical effect of umbilical cord blood transplantation (UCBT) in children with hematologic malignancies.</p><p><b>METHODS</b>A retrospective analysis was performed on the clinical data of 37 pediatric patients with hematologic malignancies that consisted of 14 cases of acute lymphocyte leukemia, 9 cases of acute myeloid leukemia, 5 cases of juvenile myelomonocytic leukemia, 3 cases of chronic myeloid leukemia, 2 cases of acute mixed leukemia, 3 cases of myelodysplastic syndrome, and 1 case of lymphosarcomatous leukemia. Thirty-seven children with hematologic malignancies received UCBT from unrelated donors (34 cases) and related donors (3 cases). Grafts were 6/6 HLA-matched in 5 cases, 5/6 HLA-matched in 12 cases, 4/6 HLA-matched in 11 cases, and 3/6 HLA-matched in 9 cases. Before transplantation, these patients received rabbit antithymocyte globulin-containing conditioning regimen. The myeloablative conditioning regimen was given in 36 cases and the reduced-intensity conditioning regimen in one case. The median age of transplantation was 5.7 years, and the median weight was 20 kg. The grafts that contained a median of 6.2×10(7) total nucleated cells (TNC)/kg and 2.7×10(5) CD34(+) cells/kg were infused.</p><p><b>RESULTS</b>The median times to neutrophil engraftment and platelet engraftment were 12 days and 25 days, respectively, and the rates of neutrophil engraftment and platelet engraftment were 95% and 78%, respectively. The rate of neutrophil engraftment was positively correlated with the number of CD34(+) cells (P=0.011), while the rate of platelet engraftment was correlated with the numbers of CD34(+) cells and TNC (P=0.001; P=0.014). The incidence rates of acute and chronic graft-versus-host disease were 49% and 11%, respectively. The median follow-up was 54 months. The 5-year transplant-related mortality, overall survival, and disease-free survival were 27%, 57.4% and 41%, respectively.</p><p><b>CONCLUSIONS</b>UCBT is an alternative source of hematopoietic stem cells for patients with hematologic malignancies.</p>

Morphologic diagnosis and onset characteristics of acute leukemia: a retrospective analysis of 233 cases in 10 years / 中国实验血液学杂志

The objective of this study was to evaluate the value of morphologic diagnosis for acute leukemia (AL), to explore the relation of morphologic diagnosis with immunology, cytogenetics and molecular biology diagnosis of AL and to analyze the onset characteristics of AL in 10 years. The samples of bone marrow and peripheral blood from 233 newly diagnosed cases of AL were collected during 2001-2011 years; the morphologic examination and immunologic, cytogenetic and molecular biologic examination (ICM) were carried out, the consistency of morphologic diagnosis with ICM diagnosis was compared, the onset characteristics of AL was analyzed. The results showed that: (1) the consistent rate of immunology, cytogenetics, molecular biology diagnosis with morphologic diagnosis was 84.3%. The order of consistent rat was AUL, M0 < M1 < HAL < M4 < M2 < M3 < M5 < ALL < M6, M7, AP; (2) Misdiagnosis always occurred among AUL, M0, M1, ALL and HAL or among M2a, M3v, M4 and M5. (3) In 233 cases, the highest ratio of blast was observed in M1 (92.5%), while the lowest ratio of blast was observed in M2 (49.5%). (4) AL occurred more frequently in males than that in female (147:86). (5) AL occurred in patients aged from 1 to 88 years. The median age was 41.5 for AUL, 40.8 for M0, 43.4 for M1, 46.3 for M2, 33.8 for M3, 42.6 for M4, 48.8 for M5, 77.3 for M6, 2.5 for M7, 65.0 for AP, 29.1 for ALL and 40.3 for HAL. (6) The number of patients in the later five years (139 cases) was significantly greater than that in the first five years (94 cases), especially the patients with M1, M2, M3, M4, and M5. It is concluded that morphologic diagnosis has important clinical value in the MICM diagnosis of AL. Attaching importance to the confusing cell morphology and onset characteristics of AL can improve the diagnostic accuracy.

Comparison of reduced intensity and myeloablative conditioning regimens for stem cell transplantation in patients with malignancies: a meta-analysis.

OBJECTIVE: The reduced intensity conditioning (RIC) stem cell transplantation is widely employed for the treatment of many hematologic malignancies, but the survival effectiveness is still unclear. This study conducted an updated meta-analysis to determine whether any significant difference could be found by using RIC vs. myeloablative conditioning (MAC) regimen for transplantation in patients with malignancies. METHODS: We electronically searched the Cochrane Central Register of Controlled Trials (CENTRAL), MEDLINE, Embase, and relevant articles (1987.01-2009.12). Comparative studies were carried out on clinical therapeutic effect of RIC and MAC on the survival outcomes and the transplantation-related complications. RESULTS: We obtained 1776 records, and 29 studies totaling 6235 patients have been assessed. Compared with MAC regimen, the RIC regimen had a higher overall survival (OS) at one-yr and no difference at two-yr later after transplantation. RIC regimen had significantly lower rates of disease-free survival (DFS) after two-yr follow-up, lower incidences of ≥ II degree acute graft-versus-host disease (aGVHD), and lower TRM [OR, 0.61, 95% CI (0.53, 0.69)], but with a higher relapse rate [OR, 1.88(1.41, 2.51)]. No significant difference was found in rates of cytomegalovirus (CMV) infection and chronic GVHD between the regimens. CONCLUSIONS: This meta-analysis confirmed that compared with MAC condition regimen, the RIC regimen had a consistently equivalent or even better rate in OS, but with lower DFS at longer follow-up.

Total body irradiation plus cyclophosphamide versus busulphan with cyclophosphamide as conditioning regimen for patients with leukemia undergoing allogeneic stem cell transplantation: a meta-analysis.

The aim of the study was to compare the therapeutic efficacy of total body irradiation (TBI)/cyclophosphamide (CY) versus BU/CY as conditioning regimen for leukemia. We electronically searched the Cochrane Central Register of Controlled Trials, Medline, Embase, CIBMTR and critically appraised all relevant articles (1990.01-2009.04). Comparative studies were evaluated on clinical therapeutic effects of TBI/CY and busulphan BU/CY regimens with assessement of engraftment, relapse, complications, and disease-free survival (DFS). Eighteen trials totaling 3172 patients have been assessed. Pooled comparisons of studies indicated that for patients with acute leukemia (ALL and AML), the TBI/CY regimen lead to lower rates of leukemia relapse, lower transplant-related mortality (TRM), and higher DFS, while for chronic myeloid leukemia (CML), the TBI/CY regimen had a higher rate of leukemia relapse, lower TRM, and similar DFS. The TBI/CY regimen was associated with similar occurrence of engraftment, acute and chronic graft-versus-host disease (GVHD), but with higher rates of cataract [odds ratio (OR) 12.69, p = 0.01], interstitial pneumonitis, later growth or development problems [OR 5.04, p = 0.008]. BU/CY regimen was associated with higher rates of complications like liver veno-occlusive disease [OR 0.43, p < 0.00001], hemorrhagic cystitis, and TRM. Our meta-analysis confirmed that different regimens and type of leukemia may affect the complications and outcome. An analysis of the effects of other regimens need to be carried out by large sample and well-designed clinical trials.

Meta-analysis of HLA matching and the outcome of unrelated umbilical cord blood transplantation (CBT).

OBJECTIVE: The aim of this meta-analysis is to compare the HLA disparities and the outcome of UCBT, i.e. the disease-free survival (DFS), engraftment, graft-versus-host disease, (GVHD), and transplantation related mortality (TRM). METHODS: We electronically searched the Cochrane Central Register of Controlled Trials (CENTRAL), MEDLINE, Pubmed, IBMTR and critically appraised all relevant articles (1989.01-2008.12). Comparative studies are carried on HLA typing and cord blood transplantation with research on stem cells engraftment, GVHD, TRM, and DFS. A meta-analysis is performed using Review Manager 5.0 software and adopted funnel plot regression assessed the publication bias. RESULTS: We got 882 records, and 10 trials totaling 1589 patients assessed. Pooled comparisons of studies of outcomes found that the incidence of neutrophil and platelet engraftment failure increased with HLA-mismatched antigen increased, >or=2-Ag mismatched group had a higher risk of >or=II degree GVHD and a lower DFS rate than the HLA matched group. CONCLUSIONS: Our meta-analysis confirmed that with the HLA-mismatched antigen increased, the rate of graft failure, severe GVHD and TRM increased, and the DFS decreased. We cannot fully exclude the possibility of center biases in treatment and selection of patients and well-designed trials need to carry out.

Unrelated umbilical cord blood transplantation and unrelated bone marrow transplantation in children with hematological disease: a meta-analysis.

UCB has been used as an alternative source of HSC. Both unrelated donor BM and UCB are available as potential options for transplantation. However, there have been limited comparisons of the outcomes of unrelated donor UCBT vs. UBMT in the unrelated setting. Our aim is to observe the therapeutic efficacy of UCBT and UBMT for treatment of pediatric hematological diseases. We electronically searched the Cochrane Central Register of Controlled Trials (CENTRAL), MEDLINE, and critically appraised all relevant articles (1989.1-2008.5). Comparative studies were carried out on clinical therapeutic effect of UCBT and UBMT with research on stem cells engraftment, complications, earlier mortality, and survival rate. We performed a meta-analysis using review manager 5.0 software (RevMan, The Nordic Cochrane Center, The Cochrane Collaboration) and adopted funnel plot regression to assess the publication bias. We obtained 324 records. Seven trials totaling 1453 patients have been assessed. Pooled comparisons of studies of UCBT and UBMT in children found that the incidence of engraftment failure and earlier transplantation-related mortality were higher with UCBT because of its delay of hematological recovery [OR = 4.96, 95% CI (3.25, 7.59), p < 0.00001 and OR = 2.36, 95% CI (1.79, 3.11), p < 0.00001 respectively], but CMV infection didn't increase obviously. There was no difference in long disease-free survival rate [OR = 0.85, 95% CI (0.65, 1.01), p = 0.06] between UCBT and UBMT due to the decrease of GVHD in UCBT [OR = 0.45, 95% CI (0.34, 0.60), p < 0.0001]. Our meta-analysis confirmed that UCBT in children is also an effective way to treat children with hematological disease and has equivalent survival outcomes compared with UBMT.

Unrelated umbilical cord blood transplantation as a treatment for children with malignant leukemia / 中国当代儿科杂志

<p><b>OBJECTIVE</b>Unrelated umbilical cord blood has the clear benefits of rapid availability and a reduced stringency of requirement for HLA match. The aim of this study was to investigate the efficacy of unrelated umbilical cord blood transplantation (UCBT) in the treatment of malignant leukemia in children.</p><p><b>METHODS</b>Six children with malignant leukemia, including three cases of acute lymphocyte leukemia [two high-risk patients and one standard-risk patient in complete remission (CR)], two juvenile myelomonocytic leukemia (one in CR and one in the accelerating stage), and one acute myeloblastic leukaemia (in CR), received a UCBT. The umbilical cord blood grafts were HLA-matched (n=1) or HLA-mismatched at 1 (n=1) or 2 (n=1) or 3 (n=3) loci. Busulfan/cyclophosphamide/antithymocyte globulin (ATG) or total body irradiation (TBI)/cyclophosphamide/ATG was involved in the myeloablative pretreatment regimen. The median infused donor nucleated cell was 8.51 x 10(7)/kg of recipient weight, and the CD34+ cell was 1.81 x 10(5)/kg of recipient weight. Cyclosporin, corticoid, mycophenolate mofetil and daclizumab were used for prophylaxis of acute graft versus host disease (GVHD).</p><p><b>RESULTS</b>The time to reach an absolute neutrophil count of 0.5 x 10(9)/L ranged from 11 to 35 days (median: 13 days) and the time to reach a platelet count of 20 x 10(9)/L ranged from 27 to 68 days (median: 30 days) after transplantation, and the donors' hematopoietic stem cells were shown in these patients. Four patients developed grade I to III acute GVHD but responded to steroids and daclizumab. Chronic GVHD was not found during a 3-16-month follow-up. Four patients survived and did not relapse during the follow-up.</p><p><b>CONCLUSIONS</b>Unrelated umbilical cord blood is an alternative source of hematopoietic stem cells for patients with leukemia. UCBT can tolerate 1-2 HLA mismatches. The incidence of acute GVHD is high in UCBT recipients.</p>

Comparison of different cryopreservation systems for peripheral blood stem cells / 中国应用生理学杂志

<p><b>AIM</b>To explore proper cryopreservative systems for hematopoietic stem cells.</p><p><b>METHODS</b>Peripheral blood mononuclear cells from 20 persons were mixed with different cryopreservative agent, dimethyl suflfoxide (DMSO) or combination of DMSO and hydroxyethyl starch (HES), then cooled in -80 degrees C low temperature refrigerator (Refr) or autocontrolled programmed cryogenic system (PCS), preserved in Refr or in liquid nitrogen. GM-CFU, LTC-IC, CD34+ cells and typeran blue resistance (TBR) were assayed after different period of cryopreservation.</p><p><b>RESULTS</b>The recovery rates of CFU-GM, LTC-IC, CD34+ cells and TBR in peripheral blood mononuclear cells which were cooled and preserved in Refr with 5% DMSO-6% HES were 82.2% +/- 14.7%, 83.0% +/- 12.2%, 94.2% +/- 4.3% and 97.7% +/- 3.9% respectively, significantly higher than that in Refr with 10% DMSO (P < 0.05). When cells were cryopreservated with the same cryopreservatives, there was no significantly difference of recovery rate in group of Refr and group of Refr with PCS. Meanwhile, there was not significantly difference of recovery rate among all three groups, preserved in Refr ahead of liquid nitrogen, in Refr merely, in liquid nitrogen with PCS within one year (p > 0.05). However, the recovery rate of CFU-GM, LTC- IC, CD34+ cells and TBR decreased dramatically if cells were cooled and preserved in Refr for two years. After cells were thawed, the cell activity declined gradually at room temperature if the cryopreservatives were not removed or diluted. The cell activity of 10% DMSO group was affected more than that of 5% DMSO-6% HES group.</p><p><b>CONCLUSION</b>5% DMSO-6% HES is better than 10% DMSO as cryopreservatives for hematopoietic stem cells. Refr cryopreservation is a simple and effective method if cells would be cryopreserved for less than one year. If cells would be cryopreserved for more than one year, liquid nitrogen cryopreservation should be recommended. The cryopreservatives should be diluted or removed immediately after cells were thawed.</p>

Comparison of total body irradiation-cyclophosphamide versus busulphan-cyclophosphamide as conditioning regimens for myelogenous leukemia: a meta-analysis / 中国实验血液学杂志

Total body irradiation combined with cyclophosphamide (TBI/CY) and busulphan combined with cyclophosphamide (BU/CY) are standard conditioning regimens in hematological stem cell transplantation for patients with myelogenous leukemia. This study was aimed to compare the therapeutic efficacy of TBI/CY and BU/CY as conditioning regiment for acute or chronic myelogenous leukemia. The Cochrane Central Register of Controlled Trials (CENTRAL), MEDLINE, CNKI, CBM (Chinese Bio-medicine Database) had been searched for all relevant articles (1999-2007). Comparative studies were carried out on clinical therapeutic effects of TBI/CY and BU/CY including stem cell engraftment, relapse, complications, transplant-related mortality, and disease-free survival. A meta-analysis was performed using Review Manager 4.2 software and funnel plot regression was adopted to assess the publication bias. The results indicated that 2149 articles in English and 46 articles in Chinese were got, and finally 9 clinical trials with total 3039 patients have been assessed. No significantly difference was found in engraftment failure and transplant-related mortality resulting from TBI/CY and BU/CY conditioning regimens, but the incidence of veno-occlusion of liver and hemorrhagic cystitis obviously increased in BU/CY group after transplantation, the acute GVHD, interstitial pneumonia and cataract significantly increased in TBI/CY group. The relapse rate of AML in TBI/CY group was lower than that in BU/CY group, and the rate of long-term disease-free survival of AML patients in TBI/CY group also significantly lower than that in BU/CY group, but the relapse rate of CML in TBI/CY group after transplantation was obviously higher than that in BU/CY group, but there was no difference in longterm disease-free survival rate between the two conditioning regimens mentioned above. It is concluded that the meta-analysis confirms different effects of TBI/CY and BU/CY regimens on myelogenous leukemia transplantation. This result is useful for physicians to select treatment regimens.

Treatment of refractory rheumatism among preschool children with autologous peripheral blood hematopoietic stem cell transplantation / 中华儿科杂志

<p><b>OBJECTIVE</b>To investigate the feasibility and safety of autologous peripheral blood hematopoietic stem cell transplantation (auto-PBHSCT) and its therapeutic effect on refractory rheumatism among preschool children.</p><p><b>METHODS</b>Three boys with juvenile rheumatoid arthritis (JRA), juvenile systemic lupus erythematosus (JSLE) and juvenile dermatomyositis (JDM) respectively, 3 to 6 years old with the mean age of 5 years with 3.5 to 22 months course of disease with 14 months on average, received auto-PBHSCT. Their conditions were so severe that conventional therapy failed to control the diseases. The changes of both clinical manifestations and immunologic indexes were observed before and after transplantation with long term following up at specialty clinic of rheumatism.</p><p><b>RESULT</b>The time when neutrophil count >or= 0.5 x 10(9)/L in the 3 children was days +9, +13 and +11 respectively, that of platelet count >or= 20 x 10(9)/L was days +14, +18 and +13 respectively. The cellular immune function remained abnormal with CD4 cells at a low level and CD4/CD8 being inverted. As to the JDM child, the skin rash had disappeared and his muscle tone was improved to grade 5 within one month after the transplantation. The EMG and serum creatase level returned to normal and muscle MRI findings were improved greatly within 2 months after the transplantation. As to the JSLE child, skin rash and proteinuria had disappeared, MRI of brain showed that the pathological changes had been absorbed and EEG returned to normal 3 months after the transplantation, all the autoantibodies turned to negative within 8 months after transplantation. As to the JRA child, the arthritis had been improved remarkably within 3 weeks after auto-PBHSCT. There was no swelling of joints nor movement limitation 3 months post transplantation. The steroids and immunosuppressive drugs were discontinued post transplantation. Cushing syndrome disappeared. Their body heights increased by 10 to 15 cm in the past 18 months, and they all returned to school. There was no relapse during follow-up periods of 25 - 27 months.</p><p><b>CONCLUSION</b>The therapy with auto-PBHSCT for refractory rheumatism among preschool children was remarkably effective in a short-term, yet the safety and long-term effect still need to be further studied.</p>

Study Progress of Hematopioetic Stem Cell Transplantation Therapy on Acute Leukemia in Children / 实用儿科临床杂志

Hematopoietic stem cell transplantation(HSCT) is one of the measures therapy on acute leukemia in children.Very-high-risk children with acute lymphocytic leukemia (ALL),such as Philadelphia chromosome-positive(Ph+),T cell immunophenotype,achieved the first complete remission(CR1),and then receive allogeneic HSCT(allo-HSCT).The transplantation group had significantly improved disease-free survival(DFS)and overall survival(OS) compared with chemotherapy group. ALL children in the second complete remission(CR2) were recommended to receive allo-HSCT,especially early-relapse group. All children in the third complete remission (CR3) were recommended to receive an allo-HSCT,and reduce replapse rate through inducing graft versus leukemia.Total body irradiation-based conditioning regimens in children with ALL has advantage over chemotherapy conditioning regimens.Children with acute myeloblastic leukemia (AML) achieved CR1 and then undergo all-HSCT.This group had significantly improved DFS and OS compared with chemotherapy group.The children with relapsed AML,if having suitable donor,were recommended to receive allo-HSCT. The children with AML who underwent transplantation relapse,and can receive the second transplantation.There was no advantage in children patients with AML using TBI-based conditioning regimens.Conditioning regimens consisted of high-dose cytarabine,in combination with granulocyte colony-stimulating factor has advantage,and improve DFS. Unrelated umbilical cord blood has emerged as a potential option.Compared with unrelated bone marrow transplantation,there was no different in DFS and relapse.For the extent,it is one of the donor hematopoietic stem cell sources.

Collection of Peripheral Blood Stem Cells from Low-Weight Infants with Osteopetrosis and Its Clinical Signi-ficance / 实用儿科临床杂志

Objective To explore the safety of collection of peripheral blood stem cells(PBSCs) from low-weight infants with osteopetrosis(OP) and its clinical significance. Methods One case of low-weight infants with OP received PBSCs collection using a continuous-flow blood cell separator,and the safety of collection process was observed.The amount of monocyte cell(MNC) and CD34+ cell were noted and its clinical significance was analyzed.Results Low-weight infants with OP could tolerate collection process,the number of collection MNC and CD34+ cells were 10.06?108/kg,2.74?106/kg.Conclusion Adequate PBSCs can be collected from OP who need not be mobilized,thus can offer backup for graft failure.PBSCs collection from low-weight infants is safe.