Incidence and risk factors of bacterial infections in children following autologous hematopoietic stem cell transplantation: Single-center experience from Jordan.

Bacterial infection is a serious sequela following AHSCT; however, limited data are available regarding pediatric recipients, especially in developing countries. We retrospectively analyzed the incidence and risk factors of bacterial infections during the first 100 days after AHSCT in children at KHCC in Amman, Jordan between January, 2005 and September, 2013. A total of 65 patients were identified, with median age of four yr (1-17). Forty-seven patients (72.3%) had solid tumors and 18 (27.7%) had lymphoma. Bacterial infections were documented in 33 patients (50%), with a total of 63 episodes. Gram-negative infection (57.1%) was more prevalent than Gram-positive infection (38%). The risk of bacterial infections was higher among patients less than five yr of age (p = 0.028) and those who developed hypogammaglobulinemia requiring IVIG replacement (p = 0.001). Patients with solid tumors developed more bacterial infections compared to patients with lymphoma (p = 0.0057). No deaths were attributed to bacterial infection. Bacterial infection rate is high among recipients of AHSCT in Jordan with Gram-negative bacteria being the most common.

Hematopoietic stem cell transplantation from non-sibling matched family donors for patients with thalassemia major in Jordan.

There are limited data on the outcome of patients with thalassemia receiving HSCT from non-sibling matched family donors. Of the 341 patients with thalassemia major that underwent donor search at our center from January 2003 to December 2011, 236 (69.2%) had fully matched family donor of which 28 patients (8.2%) had non-sibling matched family donors identified. We report on seven patients with a median age of eight yr (4-21) who underwent myeloablative (n = 4) or RIC (n = 3) HSCT. The median age of the donors was 33 yr (4-47), three were parents, two first cousins, one paternal uncle, and one paternal aunt. All patients achieved primary neutrophil and platelet engraftment at a median of 18 (13-20) and 16 days (11-20), respectively. One patient developed grade II acute GVHD, and two patients developed limited chronic GVHD. One patient experienced secondary GF requiring a second transplant. At a median follow-up of 69 months (7-110), all patients are alive and thalassemia free. Our data emphasize the need for extended family HLA typing for patients with thalassemia major in regions where there is high rate of consanguinity. Transplant from non-sibling matched family donor can result in excellent outcome.

Hematopoietic stem cell transplantation in the Eastern Mediterranean Region (EMRO) 2011-2012: A comprehensive report on behalf of the Eastern Mediterranean Blood and Marrow Transplantation group (EMBMT).

OBJECTIVE/BACKGROUND: The Eastern Mediterranean Blood and Marrow Transplantation (EMBMT) group has accumulated over 31 years of data and experience in hematopoietic stem cell transplantation (HSCT), particularly in hemoglobinopathies, severe aplastic anemia, inherited metabolic and immune disorders, in addition to a wide array of hematologic malignancies unique to this region. A regional update in current HSCT trends is highly warranted. We studied the trends of HSCT activities in World Health Organization-Eastern Mediterranean (EMRO) region, surveyed by the EMBMT, between 2011 and 2012. METHODS: Retrospective analysis of the survey data mainly of cumulative number of transplants, types of transplants (autologous vs. allogeneic), types of conditioning such as myeloablative versus reduced intensity was conducted. Also, trends in leukemias, hemoglobinopathies, severe aplastic anemia, inherited bone marrow failure syndromes, amongst others were analyzed. RESULTS: Twenty-one teams from nine EMRO countries reported their data (100% return rate) to the EMBMT for the years 2011-2012, with a total of 3,546 first HSCT (1,670 in 2011; 1,876 in 2012). Allogeneic HSCT (allo-HSCT) represented the majority (62%) in both years. The main indications for allo-HSCT were acute leukemias (988; 46%), bone marrow failure syndromes (421, 20%), hemoglobinopathies (242; 11%), and immune deficiencies (157; 7%). There was a progressive increase in the proportions of chronic myeloid leukemia cases transplanted beyond first chronic phase (37 [7%] of all chronic myeloid leukemia cases in 2011 vs. 39 [29%] in 2012). The main indications for autologous transplants were multiple myeloma/plasma cell disorders (510; 39%), Hodgkin lymphoma (311; 24%), non-Hodgkin lymphoma (259; 20%), and solid tumors (163; 12%). Reduced intensity conditioning continued to show a progressive decrease over years (9.5% in 2011 vs. 7.9% in 2012), yet remained relatively low compared with contemporary practices in Europe published by EBMT. The vast majority (91%) of allo-HSCT source was from sibling donors with continued dominance of peripheral blood (64%) followed by bone marrow (33%).While umbilical cord blood transplants increased to 4% of allo-HSCT, matched unrelated donor remained underutilized and there was no haplo-identical transplant reported. Large centers with >50 HSCT/year, showed a continued increase in the total number of allo-HSCT over the past 2years that may be related to capacity building issues and require further studies. CONCLUSION: There is a discernable increase of HSCT rate in the EMRO region with a significant expansion in utilization of cord blood transplants and allogeneic peripheral blood-HSCT as a valuable source. However, further research of outcome data and the development of regional donor banks (cord blood and matched unrelated donors) may help to facilitate future planning to satisfy the escalating regional needs and augment collaboration within the EMBMT and globally.

Spectrum of Atypical Clinical Presentations in Patients with Biallelic PRF1 Missense Mutations.

BACKGROUND: Perforin, encoded by PRF1, is a pore-forming protein crucial for lymphocyte cytotoxicity. Biallelic PRF1 nonsense mutations invariably result in early-onset hemophagocytic lymphohistiocytosis (HLH), termed familial HLH type 2 (FHL2). In contrast, biallelic PRF1 missense mutations may give rise to later-onset disease and more variable manifestations. PROCEDURE: We retrospectively searched our database for patients from families with siblings carrying biallelic PRF1 missense mutations where at least one sibling did not develop HLH, and for patients with biallelic PRF1 missense mutations and an atypical presentation of disease. We reviewed their clinical, genetic, and immunological characteristics. RESULTS: In all, we identified 10 such patients, including three sibling pairs with discordant manifestations. Interestingly, in two families, siblings of late-onset HLH patients developed Hodgkin lymphoma but no HLH. In a third family, one sibling presented with recurrent HLH episodes, whereas the other remains healthy. Of note, the affected sibling also suffered from systemic lupus erythematosus. Additional unrelated patients with biallelic PRF1 missense mutations were affected by neurological disease without classical signs of HLH, gastrointestinal inflammation as initial presentation of disease, as well as a hematological malignancy. Compared to early-onset FHL2 patients, the patients with an atypical presentation displayed a partial recovery of NK cell cytotoxicity upon IL-2 stimulation in vitro. CONCLUSIONS: Our findings substantiate and expand the spectrum of clinical presentations of perforin deficiency, linking PRF1 missense mutations to lymphoma susceptibility and highlighting clinical variability within families. PRF1 mutations should, therefore, be considered as a cause of several diseases disparate to HLH.

Outcome of aplastic anaemia in children. A study by the severe aplastic anaemia and paediatric disease working parties of the European group blood and bone marrow transplant.

This study analysed the outcome of 563 Aplastic Anaemia (AA) children aged 0-12 years reported to the Severe Aplastic Anaemia Working Party database of the European Society for Blood and Marrow Transplantation, according to treatment received. Overall survival (OS) after upfront human leucocyte antigen-matched family donor (MFD) haematopoietic stem cell transplantation (HSCT) or immunosuppressive treatment (IST) was 91% vs. 87% (P 0·18). Event-free survival (EFS) after upfront MFD HSCT or IST was 87% vs. 33% (P 0·001). Ninety-one of 167 patients (55%) failed front-line IST and underwent rescue HSCT. The OS of this rescue group was 83% compared with 91% for upfront MFD HSCT patients and 97% for those who did not fail IST up-front (P 0·017). Rejection was 2% for MFD HSCT and HSCT post-IST failure (P 0·73). Acute graft-versus-host disease (GVHD) grade II-IV was 8% in MFD graft vs. 25% for HSCT post-IST failure (P < 0·0001). Chronic GVHD was 6% in MFD HSCT vs. 20% in HSCT post-IST failure (P < 0·0001). MFD HSCT is an excellent therapy for children with AA. IST has a high failure rate, but remains a reasonable first-line choice if MFD HSCT is not available because high OS enables access to HSCT, which is a very good rescue option.

Incidence and risk factors for cytomegalovirus (CMV) reactivation following autologous hematopoietic stem cell transplantation in children.

There are limited data on the incidence of CMV reactivation following autologous HSCT (AHSCT) in children. We retrospectively reviewed the incidence and risk factors for CMV reactivation in 72 children who received AHSCT. Twenty-two patients (31%) had positive CMV antigenemia at a median of 23 days (12-31) following transplant. Four patients (6%) required preemptive therapy and all episodes resolved. None of the patients developed CMV disease. Only being CMV seropositivity prior to transplant was significantly associated with CMV reactivation (P < 0.001). The incidence of CMV reactivation following pediatric AHSCT is low, and surveillance beyond 30 days is not needed.

Hematopoietic stem cell transplantation of an adolescent with neurological manifestations of homozygous missense PRF1 mutation.

Individuals with biallelic truncating PRF1 mutations typically present with fulminant early-onset familial hemophagocytic lymphohistiocytosis (FHL). We report a 19-year-old male with a 5-year history of recurrent fever and headaches progressing to refractory seizures. Brain imaging revealed multiple ring enhancing lesions. Laboratory investigations demonstrated that the patient displayed defective lymphocyte cytotoxicity and carried a homozygous missense PRF1 mutation, c.394G > A (p.Gly132Arg). The patient was successfully treated with chemo-immunotherapy followed by matched related allogeneic hematopoietic stem cell transplantation (HSCT). Our findings demonstrate that prompt HSCT of late-onset FHL with primarily neurological manifestation can reverse central nervous system symptoms and improve long-term outcome.

Safety and feasibility of granulocyte colony-stimulating factor (G-CSF) primed bone marrow (BM) using three days of G-CSF priming as stem cell source for pediatric allogeneic BM transplantation.

There are limited data on the optimal dosing and schedule of G-CSF priming prior to BM harvest. We evaluated the safety and efficacy of three days of G-CSF of primed BM from related pediatric donors. Forty-five children were treated. All donors received 5 µg/kg per day of G-CSF as a single subcutaneous injection for three consecutive days prior to the BM harvest. The median age of the donors was seven yr (range, 0.8-18) and no donor experienced major adverse events related to G-CSF administration. The median age for the recipients was five yr (0.3-16 yr). Thirty-five patients had non-malignant disorders. The median dose of nucleated (TNC) and CD34+, CD3 cells infused per recipient weight was 5.4 × 10(8) /kg (range, 0.61-17), 4.7 × 10(6) /kg (range, 1.6-19), and 43.8 × 10(6) /kg (range, 1.8-95), respectively. All patients achieved neutrophil and platelets engraftment, at a median of 15 (range, 10-22) and 23 days (range, 13-111), respectively. At a median follow up of 60 months (range 12-100), the estimated five yr overall and EFS was 91% and 80%, respectively. Collection of BM following three days of G-CSF priming from pediatric donors is safe and results in high TNC and CD34+ cell yield.

Outcome of allogeneic stem cell transplantation for patients transformed to myelodysplastic syndrome or leukemia from severe aplastic anemia: a report from the MDS Subcommittee of the Chronic Malignancies Working Party and the Severe Aplastic Anemia Working Party of the European Group for Blood and Marrow Transplantation.

One hundred and forty patients who had undergone hematopoietic stem cell transplantation (HSCT) for myelodysplastic syndrome (MDS) or acute myelogenous leukemia (AML) transformation after treatment of severe aplastic anemia (SAA) were identified in the European Group for Blood and Marrow Transplantation (EBMT) database. The median age at HSCT was 29 years (range, 1 to 66 years). The transplant donor was related in 49% cases and unrelated in 51% cases. The 5-year probability of relapse was 17%, and that of nonrelapse mortality was 41%. The 5-year overall survival was 45% ± 9%, better for patients untreated and patients in remission compared with patients with refractory disease. Our data indicate that allogeneic HSCT leads to prolonged survival in close to one-half of the patients transforming to MDS or AML from SAA.

Feasibility of collecting umbilical cord blood in jordan and the effect of maternal and neonatal factors on hematopoietic stem cell content.

BACKGROUND: Cord blood transplant is an accepted treatment for many malignant and non-malignant diseases. We sought to determine the feasibility of collecting cord blood in Jordan and the effect of maternal and fetal factors on the quality of the cord blood units. METHODS: A total of 124 cord blood units were collected, and 75 (60%) cord blood units were included in this analysis. Cord blood volume, total nucleated cell (TNC) count, cell viability and CD34(+) content were measured, and clonogenic assay was performed. RESULTS: The mean volume of the collected units was 68.9 ml (range 40-115) with mean nucleated cell count of 6.5 x 10(8) (range 1-23.0). Our results showed a positive correlation between the volume of cord blood and TNC count (p=0.008), cell viability (p=0.001), CD34(+) content (p=0.034) and the length of the umbilical cord (p=0.011). In addition, our results showed an inverse relation between the Colony Forming Unit-Granulocyte Macrophage (CFU-GM) concentration and the gestation duration (p=0.038). CONCLUSION: We conclude that it is feasible to collect cord blood units in Jordan with excellent TNC and CD34(+) cell content. The volume of cord blood collected was associated with higher TNC count and CD34(+) count. Efforts toward establishing public cord blood banks in our area are warranted.

Allogeneic hematopoietic stem cell transplantation for infants with idiopathic myelofibrosis.

IMF is a rare disease in children that can present during infancy and has a protracted course. The only known curative approach for this disease in adult patients is allogeneic HSCT. There are very few reports describing the long-term outcome of young children following stem cell transplantation for IMF. We report on eight patients less than two yr of age with IMF that did not resolve with supportive care measures. All patients underwent myeloablative conditioning regimen with busulfan and cyclophosphamide ± ATG followed by HSCT from matched related (n = 6) or unrelated donor (n = 2). All patients achieved neutrophil and platelet engraftment. Four patients had grade II-III acute GVHD, and chronic GVHD developed in five patients (three mild and two severe). At a median follow-up of eight and a half yr (0.7-9), all patients are alive with complete resolution of their hematologic manifestations. At the last follow-up, all patients had normal endocrine function except for one patient who developed hypothyroidism. To date, this is the largest cohort of young children with IMF treated successfully with HSCT, with the longest duration of follow-up. In conclusion, our study showed that HSCT is a curative option for infants with IMF.

Hematopoietic stem cell transplantation for children with primary immunodeficiency diseases: single center experience in Jordan.

HSCT can be curative for many PID. Little is known about the outcome of HSCT for patients with PID in the developing countries. We retrospectively reviewed all children with PID who received HSCT at KHCC in Jordan between August 2003 and October 2011. Twenty-eight patients were identified. The median age was 16 months (3 months-17 yr). Patients' diagnoses were SCID (n = 16), CHS (n = 3), HLH (n = 3), WAS (n = 2), Griscelli syndrome (n = 1), ALPS (n = 1), Omenn's syndrome (n = 1), and DiGeorge syndrome (n = 1). Seventeen patients received HLA-matched related HSCT, eight received maternal un-manipulated haploidentical HSCT, and three received unrelated cord blood transplantation. Nine patients (32%) developed BCGosis secondary to reactivation of pretransplant vaccination. Three died while still receiving anti-tuberculosis drugs, one still on treatment, and all others have recovered. Six patients had graft failure; four of them received no conditioning regimens. At a median follow up of 32 months (range 1-67), 21 patients are alive, with overall survival of 72%. We conclude that HSCT for PID patients can be performed with a good outcome in developing countries; however, delayed diagnosis or referral and BCG reactivation are unique challenges.

Hematopoietic stem cell transplant versus chemotherapy plus tyrosine kinase inhibitor in the treatment of pediatric Philadelphia chromosome-positive acute lymphoblastic leukemia (ALL).

BACKGROUND: Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ ALL) remained until recently the molecular genetic abnormality associated with the worst outcome. Hematopoietic stem cell transplant (HSCT) was considered the treatment of choice, however, recent data have indicated that chemotherapy plus tyrosine kinase inhibitor (TKI) maybe an alternative effective therapy. METHODS: We conducted a retrospective analysis of children (<18 years) with Ph+ ALL who were treated at King Hussein Cancer Center (KHCC) from January 2003 till December 2011. RESULTS: Over a 9 year period, 411 children were diagnosed and treated for ALL at KHCC. Twenty three (6.6%) had Ph+ ALL; 16 males and 7 females. Median age at diagnosis was 9.5 years (range 1.67-17). The median white blood cell count was 58.6×10(3)/µL (range 1.6-459). Twelve patients underwent HSCT from a full matched related donor; and 10 were treated with intensive chemotherapy plus TKI (imatinib). Those who underwent HSCT were significantly older (P=0.004) and had a higher leukocyte count at diagnosis (P=0.53). After a median follow up of 42.2 months (range 12.7-107), the estimated 5 year event free survival (EFS) and overall survival (OS) were 75% and 91.6%, respectively, for those who underwent HSCT as primary therapy and 49.3% and 83.3%, respectively, for those treated with chemotherapy plus imatinib. There was no significant difference in EFS (P=0.98) or OS (P=1) between the two treatment modalities. CONCLUSIONS: Our results indicate that chemotherapy plus TKI may be a reasonable treatment option for some children with Ph+ ALL.

Risk adopted allogeneic hematopoietic stem cell transplantation using a reduced intensity regimen for children with thalassemia major.

BACKGROUND: Patients with thalassemia in developing countries have limited access to safe transfusions, regular medical care and chelation therapy. Although allogeneic hematopoietic stem cell transplantation (HSCT) can offer a curative approach, there are limited data on the use of this procedure in developing countries. PROCEDURE: Forty-four patients underwent a risk adopted HSCT from matched related family donor in Jordan. Thirty-one patients (7 Class 1 and 24 Class 2) underwent myeloablative conditioning (MAC) with busulfan (16 mg/kg), cyclophosphamide (200 mg/kg) and antithymocyte globulin (ATG). Thirteen patients all with Class 3, seven with hepatitis C received reduced intensity conditioning (RIC) with busulfan (8 mg/kg), fludarabine (175 mg/m(2)), total lymphoid irradiation (500 cGy) and ATG. RESULTS: All patients had initial neutrophil and platelet engraftment. Secondary graft failure was observed in 2 (6%) patients receiving myeloablative HSCT and 3 (23%) patients receiving RIC. At a median follow up of 64 months (13-108), 43 of 44 patients are alive. The 5-year probability of overall survival (OS) was 97.8% for all patients, 96.8% for patients received MAC and 100% for patients received RIC. The 5-year probability of thalassemia-free survival was 86.4% for all patients, 90.3% and 77% for patients who received MAC and RIC, respectively. CONCLUSION: Implementing a risk-adopted therapy in patient with thalassemia in Jordan can result in an excellent thalassemia free and OS, especially in those at highest risk.

Probability of finding an HLA-matched donor in immediate and extended families: the Jordanian experience.

Information regarding the probability of finding HLA-matched related donor for a patient awaiting hematopoietic stem cell transplantation (HSCT) in developing countries is scanty. We performed a retrospective review of HLA genotypes and related data for 1254 consecutive patients and their families at King Hussein Cancer Center in Amman, Jordan, between 2003 and 2011 to evaluate the chance of finding HLA-matched donor. The median family size was 5 for all patients in the study (range, 1-14), and the average number of donors was 1.4 ± 0.9 for pediatric patients and 1.6 ± 0.9 for adults. Overall, the probability of finding an HLA-matched related donor at our center was 65.5% (60.6% in pediatric patients and 74% in adults). Of the total identified donors, 18% were nonsibling donors after an immediate and/or extended family search in the pediatric group, and 6% were nonsibling donors in the adult group. Overall, 13% of donors were nonsibling donors. We conclude that the probability of finding a matched related donor for HSCT in Jordan is much higher than that reported in Western countries and Asia (65% versus 25%). We expect a similar trend in other developing and Arab countries. We recommend integrating an extended family search before or concomitantly with an unrelated donor search.

New autoimmune diseases after cord blood transplantation: a retrospective study of EUROCORD and the Autoimmune Disease Working Party of the European Group for Blood and Marrow Transplantation.

To describe the incidence, risk factors, and treatment of autoimmune diseases (ADs) occurring after cord blood transplantation (CBT), we analyzed both CBT recipients reported to EUROCORD who had developed at least 1 new AD and those who had not. Fifty-two of 726 reported patients developed at least 1 AD within 212 days (range, 27-4267) after CBT. Cumulative incidence of ADs after CBT was 5.0% +/- 1% at 1 year and 6.6% +/- 1% at 5 years. Patients developing ADs were younger and had more nonmalignant diseases (P < .001). ADs target hematopoietic (autoimmune hemolytic anemia, n = 20; Evans syndrome, n = 9; autoimmune thrombocytopenia, n = 11; and immune neutropenia, n = 1) and other tissues (thyroiditis, n = 3; psoriasis, n = 2; Graves disease, n 1; membranous glomerulonephritis, n = 2; rheumatoid arthritis, n = 1; ulcerative colitis, n = 1; and systemic lupus erythematosus, n = 1). Four patients developed 2 ADs (3 cases of immune thrombocytopenia followed by autoimmune hemolytic anemia and 1 Evans syndrome with rheumatoid arthritis). By multivariate analysis, the main risk factor for developing an AD was nonmalignant disease as an indication for CBT (P = .0001). Hematologic ADs were most often treated with steroids, rituximab, and cyclosporine. With a median follow-up of 26 months (range, 2-91), 6 of 52 patients died as a consequence of ADs. We conclude that CBT may be followed by potentially life-threatening, mainly hematologic ADs.

Hematopoietic stem cell transplantation in the Eastern Mediterranean Region (EMRO) 2008-2009: report on behalf of the Eastern Mediterranean Bone Marrow Transplantation (EMBMT) Group.

BACKGROUND: The Eastern Mediterranean Bone Marrow Transplantation (EMBMT) Group has accumulated over 25 years of data and experience in hematopoietic stem cell transplantation (HSCT), most particularly in hemoglobinopathies, severe aplastic anemia (SAA), and inherited metabolic and immune disorders, in addition to hematologic malignancies peculiar to the region and where recent updates in trends in activities are warranted. OBJECTIVES: To study trends in HSCT activities in the World Health Organization-Eastern Mediterranean (EM) region surveyed by EMBMT between 2008 and 2009. STUDY DESIGN: Retrospective analysis of the survey data, mainly of the cumulative number of transplants, types of transplants (autologous vs. allogeneic), types of conditioning as myeloablative (MAC) vs. reduced intensity conditioning (RIC) and trends in leukemias, hemoglobinopathies, SAA, inherited bone marrow failure syndromes amongst others. RESULTS AND DISCUSSION: Fourteen teams from ten Eastern Mediterranean Region Organization (EMRO) countries reported their data (100% return rate) to the EMBMT for the years 2008-2009 with a total of 2608 first HSCT (1286 in 2008; 1322 in 2009). Allogeneic HSCT represented the majority (63%) in both years. The main indications for allogeneic HSCT were acute leukemias (732; 44%), bone marrow failure syndromes (331, 20%), hemoglobinopathies (255; 15%) and immune deficiencies (90; 5%). There was a progressive increase in the proportions of chronic myeloid leukemia (CML) cases transplanted beyond the first chronic phase (3; 7% of all CML cases in 2008 vs 13; 29% in 2009). The main indications for autologous transplants were plasma cell disorders (345; 36%) Hodgkin disease (256; 27%), non-Hodgkin lymphoma (207; 22%) and solid tumors (83; 9%). RIC continued to show a progressive increase over the years (7% in 2007, 11% in 2008 and 13% in 2009), yet remained relatively low compared to contemporary practices in Europe published by EBMT. The vast majority (95%) of allo-HSCT sources were from sibling donors with a continued dominance of peripheral blood (PB) (1076; 63%), while cord blood transplant (CBT) increased to 83 (5% of allo-HSCT), matched unrelated donor (MUD) remained underutilized (1; 0%) and there were no haploidentical transplants reported. Large centers with >50 HSCT/year showed a plateau of the total number of allo-HSCT over the last 5 years that may be related to capacity issues and needs further study. CONCLUSIONS AND RECOMMENDATIONS: There is an overall increased rate of HSCT in the EMRO region with a significant increase in utilization of CBT and allogeneic PB-HSCT as a valuable source. However, further research on outcome data and development of regional donor banks (CB and MUD) may help facilitate future planning to satisfy the regional needs and increase collaboration within the group and globally.

Haploidentical stem cell transplantation as a salvage therapy for cord blood engraftment failure in a patient with Fanconi anemia.

A 7-year-old male with Fanconi Anemia who developed primary graft failure following one antigen-mismatched unrelated cord blood transplantation and a nonradiation-based conditioning, underwent a second hematopoietic stem cell transplantation (HSCT) from his 2-loci mismatched haploidentical father, using a nonradiation-based regimen, 79 days after the first HSCT. A sustained hematological engraftment was achieved at 9 days post-second HSCT. At 15 months post-second HSCT; the patient demonstrated normal blood counts, sustained donor chimerism, and no evidence of GVHD. Haploidentical HSCTs as primary or secondary sources of stem cells, with appropriate T-cell depletion, may be a readily available option in the absence of HLA-matched related or unrelated donors.