Caphosol Mouth Rinse Diminishes Oral Mucositis In Allo-HSCT Recipients.

Blood ◽  
2010 ◽  
Vol 116 (21) ◽  
pp. 3498-3498 ◽  
Author(s):  
Miroslaw Markiewicz ◽  
Dzierzak Mietla Monika ◽  
Patrycja Zielinska ◽  
Tomasz Kruzel ◽  
Tomasz Czerw ◽  
...  
Keyword(s):  
Oral Mucositis ◽  
Acute Gvhd ◽  
Conditioning Regimen ◽  
Control Group ◽  
Cell Transplant ◽  
Mouth Rinse ◽  
Oral Toxicity ◽  
Hematopoietic Stem ◽  
Conditioning Treatment ◽  
Swallowing Problems

Abstract Abstract 3498 Introduction: Oral mucositis is a complication of conditioning treatment that produces pain and morbidity in allogeneic hematopoietic stem cell transplant (allo-HSCT) recipients. The rationale of this study was to evaluate the efficacy of a calcium phosphate mouth rinse (Caphosol) vs standard regimen in 40 adult patients undergoing allo-HSCT. Patients and Methods: 40 patients treated with allo-HSCT (31 from unrelated donors, 9 from siblings) performed in Hematology and BMT center in Katowice in 2009 were randomized and stratified by the conditioning regimen (busulfan-, treosulfan- or TBI- based), type of transplant (unrelated or related) and age into two equal groups. Treatment group received Caphosol washes 4 times daily from first day of conditioning until reaching ANC 0.2 G/l. Control group received standard topical mouth care with salvia, antibacterial and antifungal solutions. During the trial patients subjectively assessed level of pain in mouth and in pharynx using 0–10 scale and swallowing problems using 0–5 scale. Mucositis was judged by experienced physician. Nonparametric Mann-Whitney U-tests were used for statistical analysis. Results: Average oral toxicity in WHO scale in Caphosol vs control group was 0.9 vs 1.8 (p=0.02), duration of mucositis was 3.2 vs 7.1 days (p=0.02). Total parenteral nutrition (TPN) due to mucosits was required in Caphosol vs control group in 0 vs 6 pts, average duration of TPN was 0 vs 1.9 days (p=0.009). Analgetics were required, respectively, in 3 vs 9 pts and analgesy lasted for 1.1 vs 3.4 days (p=0.047). Average subjective peak pain in mouth was 0.85 vs 1.75 (p=0.005) and in pharynx 1.95 vs 2.2 (NS) in Caphosol vs control group, average pain intensity was lower in Caphosol group throughout the whole period of mucositis. Intensity of swallowing problems tended to be lower in Caphosol group (NS). Acute GVHD was observed in 7 vs 9 pts in Caphosol vs control group and its average degree was 0.5 vs 0.9 (NS). Conclusions: Caphosol mouth rinse in the allo-HSCT recipients is associated with decrease of oral toxicity, lower peak pain due to mucositis and its shorter duration. In consequence, comfort of life is improved and the incidence of acute GVHD is reduced, as well as the requirement of TPN and analgetics. Disclosures: No relevant conflicts of interest to declare.

Immunosuppression medication adherence after allogeneic hematopoietic stem cell transplant: Impact of a specialized clinical pharmacy program

2021 ◽  
pp. 107815522110001
Author(s):  
Florent Charra ◽  
Michael Philippe ◽  
Chloé Herledan ◽  
Anne-Gaëlle Caffin ◽  
Virginie Larbre ◽  
...  
Keyword(s):  
Clinical Pharmacy ◽  
Hematopoietic Stem Cell Transplant ◽  
Acute Gvhd ◽  
Intervention Group ◽  
Serum Levels ◽  
Control Group ◽  
Cell Transplant ◽  
Drug Adherence ◽  
Hematopoietic Stem ◽  
Discharge Prescription

This study aims to evaluate the impact of implementing a specialized clinical pharmacy program in patients with allogeneic hematopoietic stem cell transplant (HSCT) on their adherence to the immunosuppression treatment after discharge. A prospective open interventional design using a retrospective control group was used. The intervention was based on pharmaceutical consultations: the first was performed the day before discharge of HSCT unit and the next consultations during day-care follow-up (weeks 2 and 4 after discharge). Proactive medication reconciliation was implemented with a complete list of medications before the discharge prescription. The discharge prescription summarized on a personalized drug schedule was explained to the patient. The importance of optimal adherence and the potential problems related to self-medication were explained to the patient. Immunosuppression drug adherence was assessed by a direct method using serum levels of calcineurin inhibitors. The potential impact on acute GvHD, and infection was investigated. Twenty-six patients were included in the specialized clinical pharmacy program and 35 patients were in the control group. Seventy-nine pharmaceutical consultations were conducted in the intervention group, lasting a mean 25 min and 16 min for the first and following consultations, respectively. Serum levels in the therapeutic target range were higher in the intervention group (61.5% versus 53.0%, p = 0.07), with greater intra-individual variation (p = 0.005). There was no significant intergroup difference in acute GvHD (53.8% versus 50.3%, p = 0.85) or infection (26.9 versus 22.8%, p = 0.72). The implementation of a specialized clinical pharmacy program for patients who have received allogeneic HSCT seems to be beneficial for immunosuppression drug adherence; this now needs to be confirmed in a multicenter study involving a larger number of patients.

Use of a Calcium Phosphate Mouth Rinse for Prevention of Oral Mucositis After Haematopoietic Stem Cell Transplantation- Single Center Experience.

Blood ◽  
2009 ◽  
Vol 114 (22) ◽  
pp. 4311-4311 ◽  
Author(s):  
Piotr Rzepecki ◽  
Anna wasko-Grabowska ◽  
Sylwia Oborska
Keyword(s):  
Stem Cell ◽  
Calcium Phosphate ◽  
Stem Cell Transplantation ◽  
Cell Transplantation ◽  
Hematopoietic Stem Cell ◽  
Oral Mucositis ◽  
Control Group ◽  
High Dose ◽  
Mouth Rinse ◽  
Hematopoietic Stem

Abstract Abstract 4311 Objectives Oral mucositis is a common complication of high-dose chemotherapy and radiotherapy followed by hematopoietic stem-cell support (HSCT). We evaluated the efficacy and safety of calcium phosphate mouth rinse (Caphosol) for prevention and reduction of severity and duration of oral mucositis in patients treated with HSCT. Methods In 2009 23 patients were treated. Three received allogeneic stem cells (ALLO): 2- Hodgkin disease (HD), 1-myeloma multiplex (MM) and twenty autologous transplantation (AUTO): 8-non-Hodgkin lymphomas (NHL); 7- HD; 3-MM. For ALLO conditioning regimens were composed of: fludarabine 150mg/m 2 and melphalan 140mg/m 2. AUTO were treated with: BEAM: carmustine, etoposide, cytarabine and melphalan (HD and NHL) and melphalan 200mg/m 2 (MM). The source of hematopoietic stem cells was peripheral blood. Caphosol was prepared according to the manufacturer‘s instructions and administered 4 times daily, starting from the day before the beginning of chemotherapy till the end of hospitalization. Control group was composed of patients, who had been treated with HSCT previously, before “palifermin and caphosol era”. The groups were comparable according to number of patients, their age, type of disease, transplant and conditioning regimen. Oral mucositis was assessed with the use of the five-grade World Health Organization (WHO) oral-toxicity scale. Oral mucositis was evaluated daily after the beginning of transplantation procedure until discharge from the BMT Unit. The number of days with painkillers or antibiotics were estimated. Total parenteral nutrition was given according to the standards [Martin-Salces M, De Paz R, Canales MA et al (2008) Nutritional recommendations in hematopoietic stem cell transplantation. Nutrition 24: 769-775]. Treatment with antibiotics was started when neutropenic fever occurred. Safety was assessed on the basis of the incidence of adverse events. Statistical analysis was performed using Wilcoxon‘ test for analysis of differences between groups. Results Oral mucositis grade 2-4 was not observed. In patients treated with calcium phosphate mouth rinse oral mucositis grade 2-4 was not observed. Nobody had to receive opioid analgesics or total parenteral nutrition. 30% patients developed the first degree of oral mucositis 4-5 days' duration. In the control group OM was observed in all cases, 50% patients had III- IV degree. Median duration of OM was 10 and 12 days (range 5- 20) for auto- and allogeneic patients, respectively. As compared with control group, treatment with calcium phosphate mouth rinse was associated with significant reduction of the incidence of oral mucositis in II- IV degrees (0 percent versus 50 percent, p < 0.001), duration of oral mucositis (4/ 5 days vs. 10/12 days, p < 0.001), duration of pain-killers‘ treatment (0- 22 days vs. 0- 3 days, p < 0.001) and number of days with antibiotics‘ treatment (0- 7 days vs. 7- 20 days, p= 0.002). These differences were observed in both types of transplantation. No side effects of calcium/phosphate oral rinse were observed. Conclusion In comparison with control group, treatment with Caphosol was associated with the significant reduction of the incidence of oral mucositis in II- IV degrees, duration of oral mucositis, pain-killers‘ treatment and number of days with antibiotics‘ treatment. Calcium phosphate mouth rinse is a very promising medicine for prevention of oral mucositis for patients treated with high dose chemotherapy supported with hematopoietic stem cell transplantation. Disclosures: No relevant conflicts of interest to declare.

Final analysis of the phase II, randomized, double-blind, placebo-controlled trial of single dose velafermin (CG53135–05) for the prevention of oral mucositis

2006 ◽  
Vol 24 (18_suppl) ◽  
pp. 6537-6537 ◽  
Author(s):  
M. W. Schuster ◽  
E. Anaissie ◽  
D. Hurd ◽  
W. Bensinger ◽  
J. Mason ◽  
...  
Keyword(s):  
Stem Cell ◽  
Single Dose ◽  
Phase Ii ◽  
Oral Mucositis ◽  
Conditioning Regimen ◽  
Study Drug ◽  
High Dose ◽  
Cell Transplant ◽  
Hematopoietic Stem ◽  
Grade 3

6537 Background: Oral mucositis (OM) is a commonly occurring side effect of high-dose chemotherapy (HDCT) in patients (pts) undergoing autologous hematopoietic stem cell transplant (AHSCT). Velafermin, recombinant human fibroblast growth factor 20, is being investigated for prevention of OM. Velafermin promotes epithelial and mesenchymal cell proliferation in vitro and in vivo. Methods: A phase II trial was conducted to evaluate safety, efficacy, and pharmacokinetics (PK) of velafermin. Inclusion criteria: pts undergoing HDCT and AHSCT with or without total body irradiation (TBI) were enrolled. Velafermin at 0.03, 0.1, or 0.2 mg/kg or placebo was administered as single dose IV at 24h after stem cell infusion. Safety and PK were assessed. Pts were scored daily for presence of OM using the WHO grading scale. The primary endpoint was the incidence of Grade 3/4 OM. Results: A total of 212 pts were randomized to either placebo (n=51) or velafermin at 0.03 (n=50), 0.1 (n=56), or 0.2 (n=55) mg/kg (intent-to-treat or ITT sample). 206 pts (97%) received study drug or placebo. Pt diagnoses included multiple myeloma (57%), non-Hodgkin’s (25%), or Hodgkin’s (11%) lymphoma and 13 pts (6%) received TBI as part of the conditioning regimen. The Grade 3/4 OM incidence rates (%) in the placebo or velafermin arms (0.03, 0.1, and 0.2 mg/kg) were 37, 18, 38, and 36, respectively. The primary analysis of dose dependent reduction of severe OM was not statistically significant (p = 0.549). However, velafermin at 0.03 mg/kg did reduce the incidence of Grade 3/4 OM when compared to placebo alone (p = 0.031). Duration of Grade 3/4 OM was reduced significantly in the 0.03 mg/kg when every pt was evaluated or in the 0.1 mg/kg dose when only pts with Grade 3/4 OM were included in the analysis (p = 0.037 and 0.014, respectively). A total of 5 related SAEs (3 in 0.1 mg/kg, 1 in 0.03 mg/kg, and 1 in placebo cohort) occurred within 4hr of study drug infusion. All symptoms were transient. Conclusion: Single dose velafermin at 0.03mg/kg is may be active in reducing CT induced severe OM in AHSCT pts. Safety profile supports continuing study to define the optimal dose for prevention of severe OM. A new Phase II study will be conducted to confirm velafermin activity at 0.03mg/kg dose. [Table: see text]

scholarly journals Clinical Efficacy of Prebiotics on Transplant-Related Complications

Blood ◽  
2018 ◽  
Vol 132 (Supplement 1) ◽  
pp. 4573-4573
Author(s):  
Kota Yoshifuji ◽  
Kyoko Inamoto ◽  
Kazuhiko Kakihana ◽  
Megumi Akiyama ◽  
Takashi Toya ◽  
...  
Keyword(s):  
Gut Microbiota ◽  
Oral Mucositis ◽  
Intestinal Inflammation ◽  
Conflicts Of Interest ◽  
Conditioning Regimen ◽  
Early Period ◽  
Control Group ◽  
Fermentation Products ◽  
Hematopoietic Stem ◽  
Grade Ii

Abstract Introduction Although allogeneic hematopoietic stem cell transplantation (allo-HSCT) is a potentially curable treatment for various hematological malignancies, some serious comorbidities are still problematic, and acute graft-versus-host disease (aGVHD) is a major cause of morbidity and mortality. The gut microbiota and its metabolites have been reported to play pivotal roles in both intestinal inflammation and the immune system. Also in allo-HSCT, it is increasingly evident that imbalance in the gut microbiota (dysbiosis) related to the development of aGVHD. Short chain fatty acids (SCFAs), which are produced as fermentation products by gut microbiota, are among the most studied metabolites. Of them, butyrate has been reported to strengthen the mucosal barrier, induce the differentiation of colonic regulatory T cells (Natue. 2013; 504: 446-450). Actually, both butyrate and butyrate high-producing bacteria mitigate aGVHD in a mouse model (Nat Immunol. 2016; 17: 505-513) and furthermore, some dietary ingredients are known to increase SCFAs in the colon. Resistant starch (RS) is the starch portion that is not absorbed in the small intestine but is fermented in the large intestine, and converted into butyrate. GFO is an enteral supplement comprising glutamine, fiber, and oligosaccharides, and has been reported to be alleviate the mucosal injury in HSCT (Case Rep Oncol. 2014; 7: 692-699). Based on these results, we conduct a prospective study to evaluate the clinical impact of these prebiotics on transplant outcomes. Methods Forty-nine patients who underwent allo-HSCT in our hospital were enrolled in this study (prebiotics group). As a control group, 142 patients were selected because both the clinical and micorobial data were available. We used commercially available RS (Amylofiber SH®, J-Oil Mills Inc., Tokyo, Japan) and GFO (GFO®, Otsuka Pharmaceutical Factory Inc., Tokushima, Japan). Amylofiber SH® is corn starch which contains 70% of RS. Eight grams of RS were provided for patients at lunch and dinner, and one pack of GFO®, which contains 3 g of glutamine, 4.7 g of polydextrose , 1.45 g of oligosaccharides, was provided at breakfast. Patients ingested RS and GFO® from the start of conditioning regimen to +day 28. To evaluate the efficacy of prebiotics, the incidence of aGVHD, and severity of oral mucositis and diarrhea were compared. Moreover, sequencing of 16s ribosomal RNA gene was performed using feces samples before and day 28 after allo-SCT in both groups. Results The patient characteristics of the two groups were no significant differences except for the rate of female to male transplantation (4 % vs 17 % p=0.028). Median intake of prebiotics was 51%; 45% in RS and 68% in GFO, respectively. There was clear positive correlation between RS and GFO intakes. No adverse events obviously attributable to intake of prebiotics were observed. The cumulative incidence of all grade (63.3% vs. 75.4%, p=0.02) and Grade II-IV aGVHD (32.9% vs 47.9% p=0.03) was significantly lower in the prebiotics group compared with the control group. Interestingly, the incidence of skin aGVHD was significantly lower in the prebiotics group compared with the control group (53.8% vs 64.8% p=0.04 in all grade; 12.7% vs. 26.9% p=0.03 in ≥stage 3).In a multivariate analysis, prebiotics intake was associated with a decreased risk of grade II-IVaGVHD (p=0.04, HR: 0.58[95% CI: 0.35-0.97]).Oral mucositis was significantly less severe in prebiotics group in the early period (day 0 to +9) of allo-HSCT. With regard to diarrhea, the duration of diarrhea in the prebiotics group was shorter than that in the control group (median 7 days vs. 9 days, p=0.049). In microbial analysis, the diversity of the gut microbiota was well conserved, or improved in some cases, in the prebiotics group compared to that in the control group. Conclusions Our results indicate that prebiotics treatment could reduce post-transplant complications, including GVHD by conserving the intestinal microbial diversity in transplant patients. Further evaluation will be needed to confirm them. Disclosures No relevant conflicts of interest to declare.

5-Day Decitabine with Mini Fludarabine and Busulfan Yields Comparable Outcomes to Mini Flu/Bu but with Increased Incidence of Severe Acute GvHD

Blood ◽  
2016 ◽  
Vol 128 (22) ◽  
pp. 3422-3422
Author(s):  
Melissa Baker ◽  
Tracy Andrews ◽  
Scott D. Rowley ◽  
Andrew L. Pecora ◽  
Alan P Skarbnik ◽  
...  
Keyword(s):  
Board Of Directors ◽  
Research Funding ◽  
Acute Gvhd ◽  
Conditioning Regimen ◽  
Control Group ◽  
Cell Transplant ◽  
Hematopoietic Cell Transplant ◽  
Advisory Committees ◽  
Disease Response

Abstract Background: Studies have shown that hypomethylating agents (HMAs), including 5-AZA and decitabine (Dac) are well-tolerated antileukemic agents (Kantarjian et al, JCO, 2012). Despite its myelosuppressive effect, Dac has low extramedullary toxicities, making it an attractive drug for allogeneic hematopoietic cell transplant (HCT). Reports suggest that HMAs selectively upregulate tumor associated antigens (TAAs) on malignant cells without expression in healthy tissue (Cruijsen, 2016). We previously reported on a series of 20 patients (pts) in a phase I study of 5-day Dac plus mini fludarabine and busulfan (DacMiniFluBu) in elderly or medically infirm pts (Baker et al, Blood, 2012). In the current analysis, we compared updated results from our DacFluBu study with a historical MiniFluBu control group in pts with MDS or AML. Methods: Pts were evaluated to assess engraftment, toxicity, disease response, PFS and OS. Pts received Dac 20 mg/m2/day on days (d) -15 to -11, Flu 30 mg/m2/day, on d -7 to -3 and Bu 130 mg/m2 on d -4 and -3. The control group received Flu 30 mg/m2 on d -6 to -2 and Bu 130 mg/m2 on d -3 and -2. Both groups received thymoglobulin 2 mg/kg IV on d -3, -2 and -1, followed by infusion of donor stem cells on d 0. Immunosuppression consisted of tacrolimus starting on d -2 and MTX 5 mg/m2 IV on d +1, 3, 6, and 11. Results: 107 pts were analyzed between 5/2009 and 8/2015; 36 pts received DacMiniFluBu; 17 with MDS, and 19 with AML. 23 (64%) had unrelated donors (URD); 13 (36%) had sibling donors. 71 pts were included in the MiniFluBu control group for comparison; 33 with MDS, and 38 with AML. 53 (75%) had URD; 18 (25%) had sibling donors. Median age was 68.5 yrs compared to 66 yrs, respectively. Cohorts were comparable for gender, disease and graft source. The incidence of severe (gr III/IV) acute GVHD (aGvHD) was 22% compared to the control group of 6% (p=0.0195). Moderate or severe cGVHD was seen in 7 pts vs 22 in the control group (p=0.2535). The median follow-up in the DacMiniFluBu group was 262 d, OS was 35%, relapse incidence was 28%, and NRM at 6 mos was 22%. In the control group, the median follow-up was 424 d (p=0.2213), OS was 34%, relapse was 41%, and NRM was 15%. Median time to relapse in the study vs control group was 142 and 149 d (p=0.8722). There were 22 deaths after DacMiniFluBu and 43 after MiniFluBu (p=0.7382). 6 pts in the study group received DLI at a median of 170 d post HCT for either relapse (n=3) or falling chimerism (n=3) compared to 16 pts in the control group at a median of 183 d. Multivariate analysis was performed to estimate the cumulative incidence of severe aGvHD by regimen. Results showed that conditioning regimen (HR=3.98, 95% CI, p=0.0197), degree of match (HR=1.365, p=0.039) and non-hematologic (heme) gr IV events (HR= 4.266, p=0.029) were all significant independent factors predicting a higher incidence of severe aGvHD. Conclusions: There were no significant differences in the cumulative incidences of relapse or survival between pts receiving DacMiniFluBu and MiniFluBu. However, the risk of severe aGvHD was 4 times greater in DacMiniFluBu recipients when controlling for infections, degree of match, and non-heme gr IV events. Findings were confirmed in univariate and multivariate analyses. This may be explained by the increased expression of TAAs in healthy tissues in response to Dac, which evoke T cell responses. This is the first report showing that adding Dac to the MiniFluBu regimen was an independent risk factor for severe aGvHD. Other findings in our analysis linking age, risk stratification, and degree of match to GvHD are consistent with prior reports. The differences between our results and those of other studies warrant larger validation analyses. Dac as part of a conditioning regimen should only be used in context of a clinical trial. Table Table. Figure 1 Figure 1. Figure 2 Figure 2. Disclosures Skarbnik: Genentech: Consultancy, Speakers Bureau; Pharmacyclics: Consultancy, Speakers Bureau; Abbvie: Consultancy, Speakers Bureau. Vesole:Amgen: Speakers Bureau; Novartis: Speakers Bureau; Celgene: Speakers Bureau; Janssen: Speakers Bureau; Takeda: Speakers Bureau. Goy:Pharmacyclics LLC, an AbbVie Company: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees, Research Funding, Speakers Bureau; Celgene: Consultancy, Honoraria, Research Funding; Acerta: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees; Genentech: Research Funding; Johnson & Johnson: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees, Research Funding, Speakers Bureau; infinity: Consultancy, Membership on an entity's Board of Directors or advisory committees; Takeda: Consultancy, Honoraria, Other: Writing support, Speakers Bureau.

Reduced-Intensity Allogeneic Hematopoietic Stem Cell Transplant For GATA2 Deficiency

Blood ◽  
2013 ◽  
Vol 122 (21) ◽  
pp. 2113-2113
Author(s):  
Jennifer Cuellar-Rodriguez ◽  
Juan Gea-Banacloche ◽  
Christa Zerbe ◽  
Terry J Fry ◽  
Kristin Baird ◽  
...  
Keyword(s):  
Hematopoietic Stem Cell Transplant ◽  
Acute Gvhd ◽  
Stem Cell Transplant ◽  
Conditioning Regimen ◽  
Early Death ◽  
Cell Transplant ◽  
Hematopoietic Stem ◽  
Post Transplant ◽  
Infection Susceptibility ◽  
Gata2 Deficiency

Abstract Background Heterozygous sporadic or inherited mutations in the GATA2 gene result in a syndrome known variably as: “MonoMAC” for monocytopenia with nontuberculous mycobacterial (NTM) infections; DCML, dendritic cell, monocyte and lymphoid cell deficiency; Emberger's syndrome, lymphedema and MDS with monosomy 7; and familial myelodysplastic syndrome (MDS)/acute myelogenous leukemia (AML). Life-threatening opportunistic infections and myeloid transformation constitute the rationale for allogeneic hematopoietic stem cell transplant (HSCT) for GATA2 deficiency. Methods We treated 14 patients with GATA2 deficiency using a nonmyeloablative-conditioning regimen and matched related donors (MRD) (n=4), matched unrelated donors (URD) (n=4), umbilical cord blood (UCB) (n=4), and haploidentical related donor (HD) (n=2) sources. There was considerable pre-transplant morbidity in this cohort of patients. MRD and URD recipients received 200 cGy of total body irradiation (TBI) and 3 days of fludarabine; UCB recipients received 200cGy TBI, cyclophosphamide 50 mg/kg on day -6, and 5 days of fludarabine; HD recipients received 200cGy TBI, cyclophosphamide 14.5 mg/kg on days -6 and -5, and fludarabine for 5 days. MRD, URD, and UCB recipients received tacrolimus and sirolimus for GVHD prophylaxis. HD recipients received cyclophosphamide 50 mg/kg on days + 3 and +4 followed by tacrolimus and mycophenolate mofetil. MRD and URD donor recipients received peripheral blood stem cells (PBSC) and HD donor recipients received bone marrow stem cells. Results (table 1): The median follow-up in the MRD cohort was 32 months, excluding one early death in a patient on a ventilator at the time of transplant. One patient relapsed one year post-transplant and required re-transplant using a myeloablative regimen. All 4 patients in this cohort developed acute GVHD. In the URD cohort, all patients are alive, however one patient rejected the PBSC graft and required a second URD transplant from a different donor. Two patients developed acute GVHD. In the UCB cohort, there was one early death from sepsis, one graft rejection, and one donor cell leukemia that occurred 2.5 years post-transplant. The remaining patient had a complicated course, but at 3 years post-transplant he is fully engrafted, on no medications, and has no GVHD. In the two patients who received HD transplants, one had progressed to proliferative CMML prior to transplant and died in the immediate post-transplant period. The second patient engrafted and is doing well two months post-transplant with resolution of an EBV-driven T cell lymphoma. All patients who engrafted had complete reconstitution of the monocyte, NK, and B lymphocyte compartments; all had correction of the underlying myeloid malignancy, and reversal of the infection susceptibility phenotype, characteristic of the disease. In particular, there were no recurrences of NTM. Conclusions Nonmyeloablative HSCT in GATA2 deficiency results in reconstitution of the severely deficient monocyte, B and NK cell populations and correction of the infection susceptibility phenotype. However, 1 of 4 MRD recipients developed a relapse of the original clone, and 1 of 4 URD recipients rejected the donor PBSC. The incidence of relapse and rejection, as well as the unfavorable cytogenetics in many patients, suggests that a more intense conditioning regimen is required to treat these patients. In this regard, we are now using a myeloablative regimen with busulfan and fludarabine. The poor outcome with UCB in this cohort of immunocompromised patients supports the use of HD transplants when a MRD or URD is not available. We anticipate that with increasing use of genetic testing for GATA2 mutations, patients will be transplanted earlier in the course of disease, before significant organ damage or clonal evolution of MDS to AML and CMML occurs, and that the outcome of allogeneic HSCT in these patients will continue to improve with these modifications in the transplant approach. Disclosures: No relevant conflicts of interest to declare.

scholarly journals Oral mucositis in patients with acute myeloid leukemia treated with allogeneic hematopoietic stem cell transplantation in relation to the conditioning used prior to transplantation

2021 ◽  
Author(s):  
Aleksandra Wysocka-Słowik ◽  
Lidia Gil ◽  
Zuzanna Ślebioda ◽  
Agnieszka Kręgielczak ◽  
Barbara Dorocka-Bobkowska
Keyword(s):  
Acute Myeloid Leukemia ◽  
Cell Transplantation ◽  
Oral Mucositis ◽  
Myeloid Leukemia ◽  
Hematopoietic Cell Transplantation ◽  
Conditioning Regimen ◽  
World Health ◽  
Post Transplantation ◽  
Hematopoietic Stem ◽  
Acute Myeloid

AbstractThis study was designed to investigate the frequency and severity of oral mucositis in patients with acute myeloid leukemia after allogeneic hematopoietic cell transplantation, in relation to the type of conditioning used. Eighty patients diagnosed with acute myeloid leukemia were assigned to two groups based on the conditioning regimen used before transplantation. The intensity of oral inflammatory lesions induced by chemotherapy (oral mucositis) was evaluated according to a 5-point scale recommended by World Health Organization. Oral mucosa was investigated in all patients before the transplantation and during two subsequent stages of the post-transplantation procedure in relation to the conditioning regimen used. Mucositis in the oral cavity was observed in the majority of patients (66%) in the first week after transplantation, whereas the largest percentage of patients suffering oral lesions (74%) occurred in the second week after transplantation. A significantly higher percentage of patients with mucositis was observed in the group which underwent myeloablation therapy (74% of MAC and 50% of RIC patients in the first week; 83% of MAC and 53% of RIC patients in the second examination).The severity of mucositis after transplantation was higher in the MAC patients compared to the RIC patients. The highest mean value of the mucositis index was recorded in the second week in the MAC group (1.59). In AML sufferers receiving allo-HSCT, oral mucositis is a significant complication of the transplantation. This condition is more frequent and more severe in patients after treatment with myeloablation therapy.

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