Evidence map›Paper›PMID 39836841›Full record

Observational studyBiomedica : revista del Instituto Nacional de Salud2024

Haploidentical hematopoietic stem cell transplantation using post-transplant cyclophosphamide in patients with inborn errors of immunity: Experience in a reference center in Colombia

Diego Medina, Jhonier Orlando Castro, David Esteban Castro, Estefanía Beltrán, Eliana Manzi, Alexis Antonio Franco, Manuela Olaya

Abstract readObservational Study
In one paragraph

Observational study in Biomedica : revista del Instituto Nacional de Salud, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 1 paper.

0numbers the graph read from it
0cells of the map it votes in
1citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

1 citing paper in PubMed.

  1. Article
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

7 authors.

Diego MedinaUnidad de Trasplante de Progenitores Hematopoyéticos, Servicio de Hemato-Oncología Pediátrica, Departamento Materno Infantil, Fundación Valle del Lili, Cali, Colombia; Facultad de Ciencias de la Salud, Universidad ICESI, Cali, Colombia.ORCID 0000-0002-1465-7957
Jhonier Orlando CastroFacultad de Ciencias de la Salud, Universidad ICESI, Cali, Colombia.ORCID 0000-0002-8155-5985
David Esteban CastroFacultad de Ciencias de la Salud, Universidad ICESI, Cali, Colombia.ORCID 0000-0001-9177-3698
Estefanía BeltránCentro de Investigaciones Clínicas, Fundación Valle del Lili, Cali, Colombia.ORCID 0000-0003-2438-8356
Eliana ManziFacultad de Ciencias de la Salud, Universidad ICESI, Cali, Colombia; Centro de Investigaciones Clínicas, Fundación Valle del Lili, Cali, Colombia.ORCID 0000-0001-5577-970X
Alexis Antonio FrancoUnidad de Trasplante de Progenitores Hematopoyéticos, Servicio de Hemato-Oncología Pediátrica, Departamento Materno Infantil, Fundación Valle del Lili, Cali, Colombia; Facultad de Ciencias de la Salud, Universidad ICESI, Cali, Colombia.ORCID 0000-0002-3173-6059
Manuela Olayaacultad de Ciencias de la Salud, Universidad ICESI, Cali, Colombia; Servicio de Alergología e Inmunología Pediátrica, Departamento Materno-Infantil, Fundación Valle del Lili, Cali, Colombia.ORCID 0000-0002-3239-8928

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

introductionInborn errors of immunity is a diverse group of rare diseases caused by over 400 genetic mutations affecting the immune system and increasing infection susceptibility, autoimmunity, and malignancy. Hematopoietic stem cell transplantation offers a curative option for some inborn errors of immunity, with haploidentical donors providing a viable alternative when identical donors are unavailable.

objectiveTo determine survival, usefulness of weekly chimerism monitoring, immune reconstitution, and complications in patients with inborn errors of immunity who underwent haploidentical hematopoietic stem cell transplantation at a reference center in Colombia. MATERIALS AND

methodsWe conducted a retrospective and observational study of a case series of pediatric patients who underwent haploidentical hematopoietic stem cell transplantation with post-transplant cyclophosphamide and follow-up with weekly chimerism. Survival analysis was performed using the Kaplan-Meier method.

resultsSixteen patients with haploidentical familial donor transplantation were included. The most frequent diagnosis was severe combined immunodeficiency (n=5). Eleven out of seventeen patients received a non-myeloablative conditioning regimen. Twelve out of sixteen patients developed acute graft-versus-host disease. Out of these, 3 corresponded to grades III-IV. Post-transplant infections affected 14 of the subjects, predominating bacterial agents. Median T-cell chimerism was greater than 80% during the follow-up. Reconstitution of B and T lymphocytes was achieved in more than 80%. Overall survival at five years was 81%. Survival at 100 days was 94%.

conclusionHaploidentical hematopoietic stem cell transplantation using post-transplant cyclophosphamide is a viable alternative for inborn errors of immunity when an identical donor is unavailable. Serial chimerism monitoring is useful for graft follow-up.

Indexed as

CyclophosphamideHematopoietic Stem Cell TransplantationImmunosuppressive AgentsTransplantation, HaploidenticalChildChild, PreschoolColombiaFemaleGraft vs Host DiseaseHumansInfantMaleRetrospective StudiesSevere Combined ImmunodeficiencyTransplantation ChimeraTransplantation ConditioningCyclophosphamideImmunosuppressive Agentshematopoietic stem cell transplantationimmune reconstitution

Identifiers

PMID39836841
PMCPMC12014220

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Registered trials

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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.